Search By Condition or Specialty.
| Full Name |
Adzynma (ADAMTS13, recombinant-krhn) |
| Drug |
Adzynma |
| Manufacturer |
Takeda Pharmaceuticals U.S.A., Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Prophylactic or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP) |
| Disease |
Congenital thrombotic thrombocytopenic purpura (c-TTP) |
| Therapeutic Area |
Hematology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-720-0789 |
| Fax Number |
877-251-0709 |
| Product Website |
adzynma.com |
| Full Name |
Aldurazyme® (laronidase) |
| Drug |
Aldurazyme |
| Manufacturer |
Genzyme Corporation |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Adult and pediatric patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms |
| Disease |
Mucopolysaccharidosis I |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
aldurazyme.com/healthcare |
| Full Name |
Amondys 45™ (casimersen) |
| Drug |
Amondys 45 |
| Manufacturer |
Sarepta Therapeutics, Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping |
| Disease |
Duchenne Muscular Dystrophy (DMD) |
| Therapeutic Area |
Neurology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-356-5034 |
| Fax Number |
877-339-4602 |
| Product Website |
amondys45.com |
| Full Name |
Amvuttra® (vutrisiran) |
| Drug |
Amvuttra |
| Manufacturer |
Alnylam Pharmaceuticals, Inc. |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults |
| Disease |
Hereditary ATTR amyloidosis (hATTR) – Polyneuropathy (PN) |
| Therapeutic Area |
Neurology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-372-9581 |
| Fax Number |
877-349-7938 |
| Product Website |
amvuttrahcp.com |
| Full Name |
Arcalyst® (rilonacept) |
| Drug |
Arcalyst |
| Manufacturer |
Kiniksa Pharmaceuticals (UK), Ltd. |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home |
| Approved Indication |
Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS), including Familial Cold Autoinflammatory Syndrome (FCAS), and Muckle-Wells Syndrome (MWS) in adults and children 12 years and older; maintenance of remission of Deficiency of Interleukin-1 Receptor Antagonist (DIRA) in adults and pediatric patients weighing 10 kg or more; and treatment of recurrent pericarditis (RP) and reduction in risk of recurrence in adults and children 12 years and older |
| Disease |
Cryopyrin-Associated Periodic Syndromes (CAPS); Deficiency of Interleukin-1 Receptor Antagonist (DIRA); Recurrent Pericarditis (RP) |
| Therapeutic Area |
Cardiology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-473-3261 |
| Fax Number |
877-576-6745 |
| Product Website |
arcalyst.com/hcp |
| Full Name |
ATTRUBY™ (acoramidis) |
| Drug |
Acoramidis |
| Manufacturer |
BridgeBio Pharma, Inc. |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
ATTRUBY is a transthyretin stabilizer indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. Please see the full Prescribing Information. |
| Disease |
Cardiomyopathy of transthyretin-mediated amyloidosis (ATTR-CM) |
| Therapeutic Area |
Cardiology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
888-331-6574 |
| Fax Number |
877-349-1473 |
| Product Website |
www.attruby.com |
| Full Name |
Austedo® (deutetrabenazine) |
| Drug |
Austedo |
| Manufacturer |
Teva Neuroscience, Inc. |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Indicated in adults for the treatment of Chorea associated with Huntington’s disease and tardive dyskinesia |
| Disease |
Tardive Dyskinesia (TD); Huntington’s Disease Chorea |
| Therapeutic Area |
Psychiatry, Neurology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-691-0718 |
| Fax Number |
877-819-2424 |
| Product Website |
austedohcp.com |
| Full Name |
Austedo® XR (deutetrabenazine) |
| Drug |
Austedo XR Extended Release |
| Manufacturer |
Teva Neuroscience, Inc. |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Indicated in adults for the treatment of Chorea associated with Huntington’s disease and tardive dyskinesia |
| Disease |
Tardive Dyskinesia (TD); Huntington’s Disease Chorea |
| Therapeutic Area |
Psychiatry, Neurology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-691-0718 |
| Fax Number |
877-819-2424 |
| Product Website |
www.austedohcp.com |
| Full Name |
Berinert® [C1 esterase inhibitor (human)] |
| Drug |
Berinert |
| Manufacturer |
CSL Behring |
| Route of Administration |
Intravenous |
| Site of Care |
Home |
| Approved Indication |
Treatment of acute abdominal, facial, or laryngeal hereditary angioedema (HAE) attacks in adult and pediatric patients |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-356-4252 |
| Fax Number |
847-631-6918 |
| Product Website |
berinert.com/professional |
| Full Name |
Brineura® (cerliponase alfa) |
| Drug |
Brineura |
| Manufacturer |
BioMarin Pharmaceutical Inc. |
| Route of Administration |
Intraventricular |
| Site of Care |
Healthcare Facility |
| Approved Indication |
Indicated to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency |
| Disease |
Ceroid Lipofuscinosis (C2LN) |
| Therapeutic Area |
Neurology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
brineura.com/hcp |
| Full Name |
CASGEVY™ (exagamglogene autotemcel) |
| Drug |
Casgevy |
| Manufacturer |
Vertex Pharmaceuticals |
| Route of Administration |
Intravenous |
| Site of Care |
Healthcare Facility |
| Approved Indication |
Treatment of patients aged 12 years and older with:
- Sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs)
- Transfusion-dependent β-thalassemia (TDT)
|
| Disease |
Sickle cell disease (SCD) and Transfusion-dependent β-thalassemia (TDT) |
| Therapeutic Area |
Hematology; Cell & Gene Therapy |
| Phone Number |
1-833-837-8395 |
| Full Name |
Cerdelga® (eliglustat) |
| Drug |
Cerdelga |
| Manufacturer |
Sanofi Genzyme |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Long-term treatment of adult patients with Gaucher disease type 1 who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test |
| Disease |
Gaucher Type I Disease |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
hcp.cerdelga.com |
| Full Name |
Cerezyme® (imiglucerase) |
| Drug |
Cerezyme |
| Manufacturer |
Sanofi Genzyme |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Treatment of adults and pediatric patients 2 years of age and older with Type 1 Gaucher disease that results in one or more of the following conditions: anemia, thrombocytopenia, bone disease, hepatomegaly or splenomegaly |
| Disease |
Gaucher Type I Disease |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
cerezyme.com/hcp |
| Full Name |
Cinryze® [C1 esterase inhibitor (human)] |
| Drug |
Cinryze |
| Manufacturer |
Takeda Pharmaceuticals U.S.A., Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Routine prophylaxis against angioedema attacks in adults, adolescents, and pediatric patients (6 years of age and older) with Hereditary Angioedema (HAE) |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-356-4252 |
| Fax Number |
847-631-6918 |
| Product Website |
cinryze.com/hcp |
| Full Name |
Crysvita® (burosumab-twza) |
| Drug |
Crysvita |
| Manufacturer |
Kyowa Kirin North America |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older and FGF23-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adult and pediatric patients 2 years of age and older |
| Disease |
Tumor-induced Osteomalacia (TIO); X-linked Hypophosphatemia (XLH) |
| Therapeutic Area |
Endocrinology & Bone Specialty |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-240-7614 |
| Fax Number |
877-793-4897 |
| Product Website |
crysvitahcp.com |
| Full Name |
deflazacort |
| Drug |
deflazacort |
| Manufacturer |
Generic |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older |
| Disease |
Duchenne Muscular Dystrophy (DMD) |
| Therapeutic Area |
Neurology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-605-1524 |
| Fax Number |
877-765-6254 |
| Full Name |
Dojolvi® (triheptanoin) |
| Drug |
Dojolvi |
| Manufacturer |
Ultragenyx |
| Route of Administration |
Oral or Gastrointestinal Tube |
| Site of Care |
Home |
| Approved Indication |
The treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD) |
| Disease |
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-546-7102 |
| Fax Number |
877-389-5186 |
| Product Website |
dojolvihcp.com |
| Full Name |
Elaprase® (idursulfase) |
| Drug |
Elaprase |
| Manufacturer |
Takeda Pharmaceuticals U.S.A., Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Patients with Hunter syndrome (Mucopolysaccharidosis II, MPS II) |
| Disease |
Mucopolysaccharidosis II |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
elaprase.com/hcp |
| Full Name |
Elelyso® (taliglucerase alfa) |
| Drug |
Elelyso |
| Manufacturer |
Pfizer |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Long-term enzyme replacement therapy (ERT) for adults with a confirmed diagnosis of Type 1 Gaucher disease |
| Disease |
Gaucher Type I Disease |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
elelyso.pfizerpro.com |
| Full Name |
Elevidys™ (delandistrogene moxeparvovec-rokl) |
| Drug |
Elevidys |
| Manufacturer |
Sarepta Therapeutics, Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Healthcare Facility |
| Approved Indication |
Treatment of adult patients with symptomatic gene mutation confirmed limb-girdle muscular dystrophy type R5 (LGMDR5) |
| Disease |
Limb-Girdle Muscular Dystrophy Type R5 (LGMDR5) |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
elevidyshcp.com |
| Full Name |
Elfabrio® (pegunigalsidase alfa-iwxj) |
| Drug |
Elfabrio |
| Manufacturer |
Chiesi |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of adults with confirmed Fabry disease |
| Disease |
Fabry disease |
| Therapeutic Area |
Neurology, Nephrology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
833-656-1056 |
| Fax Number |
636-355-3610 |
| Product Website |
hcp.elfabrio.com |
| Full Name |
Emflaza® (deflazacort) |
| Drug |
Emflaza |
| Manufacturer |
PTC Therapeutics, Inc. |
| Route of Administration |
Oral |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older |
| Disease |
Duchenne Muscular Dystrophy (DMD) |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
hcp.emflaza.com |
| Full Name |
Esbriet® (pirfenidone) |
| Drug |
Esbriet |
| Manufacturer |
Genentech |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
The treatment of idiopathic pulmonary fibrosis (IPF) |
| Disease |
Idiopathic Pulmonary Fibrosis (IPF) |
| Therapeutic Area |
Pulmonology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-355-9366 |
| Fax Number |
877-358-9246 |
| Product Website |
esbriethcp.com |
| Full Name |
Evkeeza® (evinacumab-dgnb) |
| Drug |
Evkeeza |
| Manufacturer |
Regeneron Pharmaceuticals |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Adjunct to other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adult and pediatric patients, aged 12 years and older, with homozygous familial hypercholesterolemia (HoFH) |
| Disease |
Homozygous Familial Hypercholesterolemia (HoFH) |
| Therapeutic Area |
Cardiology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-645-4142 |
| Fax Number |
877-473-0199 |
| Product Website |
evkeezahcp.com |
| Full Name |
Exondys 51® (eteplirsen) |
| Drug |
Exondys 51 |
| Manufacturer |
Sarepta Therapeutics, Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping |
| Disease |
Duchenne Muscular Dystrophy (DMD) |
| Therapeutic Area |
Neurology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-356-5034 |
| Fax Number |
877-339-4602 |
| Product Website |
exondys51.com |
| Full Name |
Fabrazyme® (agalsidase beta) |
| Drug |
Fabrazyme |
| Manufacturer |
Sanofi Genzyme |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease |
| Disease |
Fabry disease |
| Therapeutic Area |
Neurology, Nephrology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
hcp.fabrazyme.com |
| Full Name |
Firazyr® (icatibant) |
| Drug |
Firazyr |
| Manufacturer |
Takeda Pharmaceuticals U.S.A., Inc. |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Treatment of acute attacks of hereditary angioedema (HAE) in adults 18 years of age and older |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-356-4252 |
| Fax Number |
847-631-6918 |
| Product Website |
firazyr.com/hcp |
| Full Name |
FUROSCIX® (furosemide injection) 80 mg/10 mL for subcutaneous use |
| Drug |
Furosemide |
| Manufacturer |
scPharmaceuticals Inc. |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home |
| Approved Indication |
FUROSCIX is indicated for the treatment of congestion due to fluid overload in adults with chronic heart failure. Please see the full Prescribing Information and Instructions for Use. |
| Disease |
Heart Failure |
| Therapeutic Area |
Cardiology |
| Enrollment Form Link |
www.furoscix.com/hcp/contact-us |
| Phone Number |
855-387-6724 |
| Product Website |
www.furoscix.com/hcp/ |
| Full Name |
Haegarda® [C1 esterase inhibitor subcutaneous (human)] |
| Drug |
Haegarda |
| Manufacturer |
CSL Behring |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home |
| Approved Indication |
Routine prophylaxis to prevent Hereditary Angioedema (HAE) attacks in patients 6 years of age and older |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-356-4252 |
| Fax Number |
847-631-6918 |
| Product Website |
haegarda.com/hcp |
| Full Name |
Hemgenix® (etranacogene dezaparvovec-drlb) |
| Drug |
Hemgenix |
| Manufacturer |
CSL Behring |
| Route of Administration |
Intravenous |
| Site of Care |
Healthcare Facility |
| Approved Indication |
The treatment of adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes |
| Disease |
Hemophilia B |
| Therapeutic Area |
Hematology; Cell & Gene Therapy |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-975-8693 |
| Fax Number |
877-740-7535 |
| Product Website |
hemgenix.com/hcp |
| Full Name |
Icatibant Acetate |
| Drug |
icatibant acetate |
| Manufacturer |
Generic |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of acute attacks of hereditary angioedema (HAE) in adults 18 years of age and older |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-356-4252 |
| Fax Number |
847-631-6918 |
| Full Name |
Ingrezza® (valbenazine) |
| Drug |
Ingrezza |
| Manufacturer |
Neurocrine Biosciences |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
The treatment of adults with tardive dyskinesia or chorea associated with Huntington’s disease |
| Disease |
Tardive Dyskinesia (TD); Huntington’s Disease Chorea |
| Therapeutic Area |
Psychiatry, Neurology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-279-1676 |
| Fax Number |
877-868-1681 |
| Product Website |
ingrezzahcp.com |
| Full Name |
intravenous immunoglobulin (IVIG) |
| Drug |
IVIG |
| Manufacturer |
Generic |
| Route of Administration |
Subcutaneous or Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Treatment of primary humoral immunodeficiency, chronic immune thrombocytopenic purpura (ITP), or chronic inflammatory demyelinating polyneuropathy (CIDP) in adults |
| Disease |
Primary Immune Deficiency |
| Therapeutic Area |
Allergy & Immunology |
| Phone Number |
847-725-8100 |
| Full Name |
Kalbitor® (ecallantide) |
| Drug |
Kalbitor |
| Manufacturer |
Takeda Pharmaceuticals U.S.A., Inc. |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home |
| Approved Indication |
Treatment of acute attacks of hereditary angioedema (HAE) in patients 12 years of age and older |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-356-4252 |
| Fax Number |
847-631-6918 |
| Product Website |
kalbitor.com/hcp |
| Full Name |
Kanuma® (sebelipase alfa) |
| Drug |
Kanuma |
| Manufacturer |
Alexion Pharmaceuticals, a subsidiary of AstraZeneca |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency |
| Disease |
Lysosomal Acid Lipase Deficiency (LAL-D) |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
kanuma.com/hcp |
| Full Name |
KEBILIDI™ (eladocagene exuparvovec-tneq) |
| Drug |
Kebilidi |
| Manufacturer |
PTC Therapeutics, Inc. |
| Route of Administration |
Intraputaminal |
| Site of Care |
Healthcare facility |
| Approved Indication |
Indicated for the treatment of adult and pediatric patients with aromatic 13 L-amino acid decarboxylase (AADC) deficiency |
| Disease |
Aromatic L-amino acid decarboxylase (AADC) deficiency |
| Therapeutic Area |
Neurology, Genetics, Cell & Gene Therapy |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
888-221-3495 |
| Fax Number |
877-810-7806 |
| Full Name |
LAMZEDE® (velmanase alfa-tycv) |
| Drug |
LAMZEDE |
| Manufacturer |
Chiesi Farmaceutici |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric patients |
| Disease |
Alpha-mannosidosis |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
(800) 240-9572 |
| Fax Number |
(847) 427-7976 |
| Product Website |
lamzede.com/hcp |
| Drug Name |
Leqvio® (inclisiran) |
| Manufacturer |
Novartis Pharmaceuticals |
| Route of Administration |
Subcutaneous |
| Approved Indication |
The treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease (ASCVD), who require additional lowering of low-density lipoprotein cholesterol (LDL-C) |
| Disease |
Primary Hyperlipidemia; Heterozygous Familial Hypercholesterolemia (HeFH) |
| Therapeutic Area |
Cardiology |
| Enrollment Form Link |
Enrollment Form |
| Phone Numbers |
800-372-6153 (Enrollment), 877-515-9670 (Support) |
| Product Website |
leqvio.com |
| Full Name |
Livdelzi® (seladelpar) |
| Drug |
Livdelzi |
| Manufacturer |
Gilead Sciences, Inc. |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA |
| Disease |
Primary Biliary Cholangitis (PBC) |
| Therapeutic Area |
Hepatology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
888-263-8004 |
| Fax Number |
877-846-0402 |
| Product Website |
livdelzihcp.com/ |
| Full Name |
Lumizyme® (alglucosidase alfa) |
| Drug |
Lumizyme |
| Manufacturer |
Sanofi Genzyme |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Patients with Pompe disease (GAA deficiency) |
| Disease |
Pompe Disease |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form Link |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
lumizyme.com |
| Full Name |
MACI® (autologous cultured chondrocytes on a porcine collagen membrane) |
| Drug |
MACI |
| Manufacturer |
Vericel Corporation |
| Route of Administration |
Surgery |
| Site of Care |
Healthcare Facility |
| Approved Indication |
The repair of symptomatic, single or multiple full-thickness cartilage defects of the knee with or without bone involvement in adults |
| Disease |
Cartilage Defect Repair |
| Therapeutic Area |
Orthopedics; Cell & Gene Therapy |
| Enrollment Form Link |
MACI Enrollment Form |
| Phone Number |
800-388-1903 |
| Fax Number |
N/A |
| Product Website |
maci.com |
| Full Name |
Mifepristone |
| Drug |
Mifepristone |
| Manufacturer |
Generic |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Control hyperglycemia secondary to hypercortisolism in adult patients with endogenous Cushing’s syndrome who have type 2 diabetes mellitus or glucose intolerance and have failed surgery or are not candidates for surgery |
| Disease |
Cushing’s Syndrome |
| Therapeutic Area |
Endocrinology |
| Enrollment Form Link |
Mifepristone Enrollment Form |
| Phone Number |
(888) 204-7730 |
| Fax Number |
(877) 218-6288 |
| Product Website |
https://www.tevausa.com/products/mifepristonetablets |
| Full Name |
miglustat |
| Drug |
Miglustat |
| Manufacturer |
Generic |
| Route of Administration |
Oral |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Monotherapy for treatment of adult patients with mild/moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option
Use in combination with MIPLYFFA™ (arimoclomol) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older |
| Disease |
Gaucher Type I Disease, Niemann-Pick disease type C (NPC) |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Miglustat Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Full Name |
MIPLYFFA™ (arimoclomol) |
| Drug |
Miplyffa |
| Manufacturer |
Zevra Therapeutics, Inc. |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older |
| Disease |
Niemann-Pick disease type C (NPC) |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
888-668-4198 |
| Fax Number |
888-668-2143 |
| Product Website |
miplyffa.com |
| Full Name |
Naglazyme® (galsulfase) |
| Drug |
Naglazyme |
| Manufacturer |
BioMarin Pharmaceutical Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Patients with Mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy Syndrome) |
| Disease |
Mucopolysaccharidosis VI |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Naglazyme Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
naglazyme.com |
| Full Name |
Nexviazyme® (avalglucosidase alfa-ngpt) |
| Drug |
Nexviazyme |
| Manufacturer |
Sanofi Genzyme |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of patients 1 year of age and older with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) |
| Disease |
Pompe Disease |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Nexviazyme Enrollment Form Link |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
nexviazyme.com |
| Full Name |
Ofev® (nintedanib) |
| Drug |
Ofev |
| Manufacturer |
Boehringer Ingelheim |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Treatment of idiopathic pulmonary fibrosis (IPF) in adults, treatment of chronic fibrosing interstitial lung diseases (ILDs) with a progressive phenotype in adults, or slowing the rate of decline in pulmonary function in adults with systemic sclerosis-associated interstitial lung disease (SSc-ILD) |
| Disease |
Idiopathic Pulmonary Fibrosis (IPF); Systemic Sclerosis Interstitial Lung Disease (SSc-ILD) |
| Therapeutic Area |
Pulmonology |
| Enrollment Form Link |
Ofev Enrollment Form |
| Phone Number |
800-373-1452 |
| Fax Number |
888-975-1456 |
| Product Website |
ofev.com |
| Full Name |
OLPRUVA® (sodium phenylbutyrate) |
| Drug |
Olpruva |
| Manufacturer |
Zevra Therapeutics, Inc. |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Chronic management of adult and pediatric patients weighing 20 kg or greater and with a body surface area (BSA) of 1.2 m2 or greater, with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS) |
| Disease |
Urea Cycle Disorders (UCDs) |
| Therapeutic Area |
Nephrology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-837-8905 |
| Fax Number |
877-369-3806 |
| Product Website |
olpruvahcp.com |
| Full Name |
Onpattro® (patisiran) |
| Drug |
Onpattro |
| Manufacturer |
Alnylam Pharmaceuticals |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults |
| Disease |
Hereditary ATTR amyloidosis (hATTR) – Polyneuropathy (PN) |
| Therapeutic Area |
Neurology |
| Enrollment Form Link |
Onpattro Start Form |
| Phone Number |
800-690-8236 |
| Fax Number |
877-445-8481 |
| Product Website |
onpattro.com |
| Full Name |
Oxlumo® (lumasiran) |
| Drug |
Oxlumo |
| Manufacturer |
Alnylam Pharmaceuticals |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients |
| Disease |
Primary Hyperoxaluria Type 1 (PH1) |
| Therapeutic Area |
Nephrology |
| Enrollment Form Link |
Oxlumo Enrollment Form |
| Phone Number |
800-460-5217 |
| Fax Number |
877-276-8563 |
| Product Website |
oxlumo.com |
| Full Name |
PEDMARK® (sodium thiosulfate injection) |
| Drug |
Pedmark |
| Manufacturer |
Fennec Pharmaceuticals |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Reduce the risk of ototoxicity associated with cisplatin in pediatric patients 1 month of age and older with localized, non-metastatic solid tumors |
| Disease |
Ototoxicity associated with cisplatin |
| Therapeutic Area |
Otolaryngology |
| Enrollment Form Link |
Pedmark Enrollment Form |
| Phone Number |
800-385-8596 |
| Fax Number |
877-694-2545 |
| Product Website |
pedmark.com |
| Full Name |
Photrexa® (riboflavin 5’-phosphate ophthalmic solution) |
| Drug |
Photrexa |
| Manufacturer |
Glaukos |
| Route of Administration |
Intraocular |
| Site of Care |
Healthcare Facility |
| Approved Indication |
The treatment of progressive keratoconus (1.1) and corneal ectasia following refractive surgery |
| Disease |
Progressive Keratoconus; Corneal Ectasia |
| Therapeutic Area |
Ophthalmology |
| Enrollment Form Link |
Photrexa Enrollment Form |
| Phone Number |
800-550-7207 |
| Fax Number |
877-277-3139 |
| Product Website |
|
| Full Name |
pirfenidone |
| Drug |
Pirfenidone |
| Manufacturer |
Generic |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
The treatment of idiopathic pulmonary fibrosis (IPF) |
| Disease |
Idiopathic Pulmonary Fibrosis (IPF) |
| Therapeutic Area |
Pulmonology |
| Enrollment Form Link |
Pirfenidone Enrollment Form |
| Phone Number |
800-355-9366 |
| Fax Number |
877-358-9246 |
| Product Website |
|
| Full Name |
Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) |
| Drug |
Pombiliti & Opfolda |
| Manufacturer |
Amicus Therapeutics |
| Route of Administration |
Intravenous and oral |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT) |
| Disease |
Pompe Disease |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Pombiliti & Opfolda Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
pombilitiopfolda.com |
| Full Name |
Pradaxa® (dabigatran etexilate) oral pellets – pediatric |
| Drug |
Pradaxa Pediatric |
| Manufacturer |
Boehringer Ingelheim |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
To reduce the risk of stroke and systemic embolism in adult patients with non-valvular atrial fibrillation, for the treatment of deep venous thrombosis (DVT) and pulmonary embolism (PE) in adult patients who have been treated with a parenteral anticoagulant for 5-10 days, to reduce the risk of recurrence of DVT and PE in adult patients who have been previously treated, for the prophylaxis of DVT and PE in adult patients who have undergone hip replacement surgery, for the treatment of venous thromboembolic events (VTE) in pediatric patients 8 to less than 18 years of age who have been treated with a parenteral anticoagulant for at least 5 days, or to reduce the risk of recurrence of VTE in pediatric patients 8 to less than 18 years of age who have been previously treated |
| Disease |
Venous Thromboembolism (VTE) |
| Therapeutic Area |
Hematology |
| Enrollment Form Link |
Pradaxa Enrollment Form |
| Phone Number |
800-593-0310 |
| Fax Number |
877-709-9184 |
| Product Website |
https://patientpatient.boehringer-ingelheim.com/us/pradaxa/ |
| Full Name |
Roctavian™ (valoctocogene roxaparvovec-rvox) |
| Drug |
Roctavian |
| Manufacturer |
BioMarin Pharmaceutical Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Healthcare Facility |
| Approved Indication |
The treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity < 1 IU/dL) without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test |
| Disease |
Hemophilia A |
| Therapeutic Area |
Hematology; Cell & Gene Therapy |
| Enrollment Form Link |
Roctavian Enrollment Form |
| Phone Number |
800-358-0013 |
| Fax Number |
877-631-9228 |
| Product Website |
roctavian.com |
| Full Name |
Ruconest® [C1 esterase inhibitor (recombinant)] |
| Drug |
Ruconest |
| Manufacturer |
Pharming Group |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of acute attacks in adult and adolescent patients with hereditary angioedema (HAE) |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Ruconest Patient Enrollment Form |
| Phone Number |
800-356-4252 |
| Fax Number |
847-631-6918 |
| Product Website |
ruconest.com |
| Full Name |
SAJAZIR™ (icatibant) |
| Drug |
Sajazir |
| Manufacturer |
Cycle Pharmaceuticals |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of acute attacks of hereditary angioedema (HAE) in adults 18 years of age and older |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
(800) 356-4252 |
| Fax Number |
(877) 277–7318 |
| Product Website |
sajazir.com/hcp/ |
| Full Name |
Sinuva® (mometasone furoate) |
| Drug |
Sinuva |
| Manufacturer |
Intersect ENT |
| Route of Administration |
Sinus Implant |
| Site of Care |
Healthcare Facility |
| Approved Indication |
The treatment of nasal polyps in patients ≥ 18 years of age who have had ethmoid sinus surgery |
| Disease |
Nasal Polyps |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Sinuva Enrollment Form |
| Phone Number |
800-356-4354 |
| Fax Number |
877-574-0550 |
| Product Website |
sinuva.com |
| Full Name |
Soliris® (eculizumab) |
| Drug |
Soliris |
| Manufacturer |
Alexion Pharmaceuticals, a subsidiary of AstraZeneca |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis |
| Disease |
Paroxysmal Nocturnal Hemoglobinuria (PNH); Atypical Hemolytic Uremic Syndrome (aHUS) |
| Therapeutic Area |
Hematology |
| Enrollment Form Link |
Soliris Enrollment Form |
| Phone Number |
N/A |
| Fax Number |
N/A |
| Product Website |
solirisnmosd.com |
| Full Name |
Takhzyro® (lanadelumab-flyo) |
| Drug |
Takhzyro |
| Manufacturer |
Takeda Pharmaceuticals U.S.A., Inc. |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home |
| Approved Indication |
Prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 2 years and older |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-356-4252 |
| Fax Number |
847-631-6918 |
| Product Website |
takhzyro.com |
| Full Name |
tiopronin |
| Drug |
Tiopronin |
| Manufacturer |
Generic |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
In combination with high fluid intake, alkali, and diet modification, for the prevention of cystine stone formation in adults and pediatric patients 9 years of age and older with severe homozygous cystinuria, who are not responsive to these measures alone |
| Disease |
Homozygous Cystinuria |
| Therapeutic Area |
Nephrology |
| Enrollment Form Link |
Tiopronin Enrollment Form |
| Phone Number |
800-764-0147 |
| Fax Number |
877-848-6579 |
| Product Website |
|
| Full Name |
Tzield® (teplizumab-mzwv) |
| Drug |
Tzield |
| Manufacturer |
Sanofi |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Delay the onset of Stage 3 type 1 diabetes (T1D) in adults and pediatric patients aged 8 years and older with Stage 2 T1D |
| Disease |
Type 1 Diabetes (T1D) |
| Therapeutic Area |
Endocrinology & Bone Specialty |
| Enrollment Form Link |
Tzield Enrollment Form |
| Phone Number |
800-670-5321 |
| Fax Number |
877-655-4364 |
| Product Website |
tzield.com |
| Full Name |
Ultomiris® (ravulizumab-cwvz) |
| Drug |
Ultomiris |
| Manufacturer |
Alexion (purchased by AZ) |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) |
| Disease |
Paroxysmal Nocturnal Hemoglobinuria (PNH); Atypical Hemolytic Uremic Syndrome (aHUS) |
| Therapeutic Area |
Hematology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
N/A |
| Fax Number |
N/A |
| Product Website |
ultomirishcp.com/nmosd |
| Full Name |
Veopoz™ (pozelimab-bbfg) |
| Drug |
Veopoz |
| Manufacturer |
Regeneron Pharmaceuticals |
| Route of Administration |
Intravenous |
| Site of Care |
Healthcare Facility |
| Approved Indication |
The treatment of adult and pediatric patients 1 year of age and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease |
| Disease |
CD55-deficient protein-losing enteropathy (CHAPLE) Disease |
| Therapeutic Area |
Allergy & Immunology, Genetics |
| Enrollment Form Link |
Veopoz Enrollment Form |
| Phone Number |
800-438-2375 |
| Fax Number |
877-440-0891 |
| Product Website |
veopoz.com |
| Full Name |
Vigabatrin |
| Drug |
Generic |
| Manufacturer |
N/A |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Adjunctive therapy for adults and pediatric patients 2 years of age and older with refractory complex partial seizures who have inadequately responded to several alternative treatments and for whom the potential benefits outweigh the risk of vision loss |
| Disease |
Refractory Complex Partial Seizures; Infantile Spasms |
| Therapeutic Area |
Neurology |
| Enrollment Form Link |
Vigabatrin Enrollment Form |
| Phone Number |
800-976-9809 |
| Fax Number |
877-569-6004 |
| Product Website |
N/A |
| Full Name |
Viltepso® (viltolarsen) |
| Drug |
Viltepso |
| Manufacturer |
NS Pharma |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping |
| Disease |
Duchenne Muscular Dystrophy (DMD) |
| Therapeutic Area |
Neurology |
| Enrollment Form Link |
Viltepso Enrollment Form |
| Phone Number |
800-759-0445 |
| Fax Number |
877-286-3620 |
| Product Website |
viltepso.com |
| Full Name |
Vimizim® (elosulfase alfa) |
| Drug |
Vimizim |
| Manufacturer |
BioMarin Pharmaceutical Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome) |
| Disease |
Mucopolysaccharidosis IVA |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Vimizim Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
vimizim.com |
| Full Name |
Vivitrol® (naltrexone) |
| Drug |
Vivitrol |
| Manufacturer |
Alkermes |
| Route of Administration |
Intramuscular |
| Site of Care |
Healthcare Facility |
| Approved Indication |
The treatment of alcohol dependence in patients who can abstain from alcohol in an outpatient setting prior to initiation of treatment with VIVITROL; The prevention of relapse to opioid dependence following opioid detoxification |
| Disease |
Alcohol Dependence; Opioid Dependence |
| Therapeutic Area |
Mental Health & Addiction |
| Enrollment Form Link |
Vivitrol Enrollment Form |
| Phone Number |
800-373-1406 |
| Fax Number |
847-427-7975, 855-677-4641, 877-496-3716 |
| Product Website |
vivitrolhcp.com |
| Full Name |
Vowst™ (fecal microbiota spores live-brpk) |
| Drug |
Vowst |
| Manufacturer |
Aimmune Therapeutics |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Prevent the recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of age and older following antibacterial treatment for recurrent CDI (rCDI) |
| Disease |
Recurrent C. difficile Infection (rCDI) |
| Therapeutic Area |
Infectious Disease; Gastroenterology |
| Enrollment Form Link |
Vowst Enrollment Form |
| Phone Number |
800-485-4885 |
| Fax Number |
877-640-5179 |
| Product Website |
vowsthcp.com |
| Full Name |
Vpriv® (velaglucerase alfa) |
| Drug |
Vpriv |
| Manufacturer |
Takeda Pharmaceuticals U.S.A., Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Long-term enzyme replacement therapy (ERT) for pediatric and adult patients with type 1 Gaucher disease |
| Disease |
Gaucher Type I Disease |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Vpriv Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
hcp.vpriv.com |
| Full Name |
Vyepti® (eptinezumab-jjmr) |
| Drug |
Vyepti |
| Manufacturer |
Lundbeck |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The preventive treatment of migraine in adults |
| Disease |
Migraine |
| Therapeutic Area |
Neurology |
| Enrollment Form Link |
Vyepti Enrollment Form |
| Phone Number |
800-259-7145 |
| Fax Number |
877-892-3019 |
| Product Website |
vyeptihcp.com |
| Full Name |
Vyndamax® (tafamidis) |
| Drug |
Vyndamax |
| Manufacturer |
Pfizer |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
The treatment of the cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization |
| Disease |
Hereditary ATTR amyloidosis (hATTR) – Cardiomyopathy (CM); Wild type transthyretin amyloid cardiomyopathy (wtATTR-CM) |
| Therapeutic Area |
Cardiology |
| Enrollment Form Link |
Vyndamax Enrollment Form |
| Phone Number |
800-930-2043 |
| Fax Number |
877-684-3116 |
| Product Website |
vyndamax.pfizerpro.com |
| Full Name |
Vyndaqel® (tafamidis meglumine) |
| Drug |
Vyndaqel |
| Manufacturer |
Pfizer |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
The treatment of the cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization |
| Disease |
Hereditary ATTR amyloidosis (hATTR) – Cardiomyopathy (CM); Wild type transthyretin amyloid cardiomyopathy (wtATTR-CM) |
| Therapeutic Area |
Cardiology |
| Enrollment Form Link |
Vyndaqel Enrollment Form |
| Phone Number |
800-930-2043 |
| Fax Number |
877-684-3116 |
| Product Website |
vyndamax.pfizerpro.com |
| Full Name |
Vyondys 53® (golodirsen) |
| Drug |
Vyondys 53 |
| Manufacturer |
Sarepta Therapeutics, Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping |
| Disease |
Duchenne Muscular Dystrophy (DMD) |
| Therapeutic Area |
Neurology |
| Enrollment Form Link |
Vyondys 53 Enrollment Form |
| Phone Number |
800-356-5034 |
| Fax Number |
877-339-4602 |
| Product Website |
vyondys53.com |
| Full Name |
Wainua™ (eplontersen) |
| Drug |
Wainua |
| Manufacturer |
AstraZeneca |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home |
| Approved Indication |
The treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults |
| Disease |
Hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) |
| Therapeutic Area |
Neurology |
| Enrollment Form Link |
Wainua Enrollment Form |
| Phone Number |
800-986-4975 |
| Fax Number |
877-369-5207 |
| Product Website |
wainuahcp.com |
| Full Name |
Xenpozyme® (olipudase alfa-rpcp) |
| Drug |
Xenpozyme |
| Manufacturer |
Sanofi Genzyme |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients |
| Disease |
Acid Sphingomyelinase Deficiency (ASMD) |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Xenpozyme Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
xenpozyme.com |
| Full Name |
Yargesa™ (miglustat) |
| Drug |
Yargesa |
| Manufacturer |
Edenbridge Pharmaceuticals |
| Route of Administration |
Oral |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Monotherapy for treatment of adult patients with mild/moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option
Use in combination with MIPLYFFA™ (arimoclomol) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older |
| Disease |
Gaucher Type I Disease, Niemann-Pick disease type C (NPC) |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Yargesa Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Full Name |
YORVIPATH® (palopegteriparatide) |
| Drug |
Yorvipath |
| Manufacturer |
Ascendis Pharma |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home |
| Approved Indication |
The treatment of hypoparathyroidism in adults |
| Disease |
Hypoparathyroidism |
| Therapeutic Area |
Endocrinology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
888-204-7802 |
| Fax Number |
877-471-8175 |
| Product Website |
yorvipathhcp.com |
| Full Name |
Zolgensma® (onasemnogene abeparvovec-xioi) |
| Drug |
Zolgensma |
| Manufacturer |
Novartis Gene Therapy |
| Route of Administration |
Intravenous |
| Site of Care |
Healthcare Facility |
| Approved Indication |
The treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene |
| Disease |
Spinal Muscular Atrophy (SMA) |
| Therapeutic Area |
Neurology; Cell & Gene Therapy |
| Referral Link |
Zolgensma Referral Information |
| Phone Number |
800-697-5048 |
| Fax Number |
877-471-5704 |
| Website |
zolgensma.com |
| Full Name |
Ztalmy® (ganaxolone) |
| Drug |
Ztalmy |
| Manufacturer |
Marinus Pharmaceuticals |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
The treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older |
| Disease |
Cyclin-dependent kinase-like 5 (CDKL5) Deficiency Disorder |
| Therapeutic Area |
Neurology |
| Referral Link |
Ztalmy Enrollment Form |
| Phone Number |
844-ZTALMY1 |
| Fax Number |
844-ZTALMYF |
| Website |
ztalmy.com |
