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Drug

ENCELTO™ (revakinagene taroretcel-lwey)

Deflazacort

KEBILIDI™ (eladocagene exuparvovec-tneq)

ATTRUBY™ (acoramidis)

YORVIPATH® (palopegteriparatide)

CASGEVY™ (exagamglogene autotemcel)

LAMZEDE® (velmanase alfa-tycv)

SAJAZIR™ (icatibant)

FUROSCIX® (furosemide injection)

MIPLYFFA™ (arimoclomol)

OLPRUVA® (sodium phenylbutyrate)

Livdelzi® (seladelpar)

Miglustat

Vyondys 53® (golodirsen)

Vowst™ (fecal microbiota spores live-brpk)

Vpriv® (velaglucerase alfa)

Vyepti® (eptinezumab-jjmr)

Vyndamax® (tafamidis)

Vyndaqel® (tafamidis meglumine)

WAINUA™ (eplontersen)

Xenpozyme® (olipudase alfa-rpcp)

Yargesa™ (miglustat)

Zolgensma® (onasemnogene abeparvovec-xioi)

Ztalmy® (ganaxolone)

Photrexa® (riboflavin 5’-phosphate ophthalmic solution)

PEDMARK® (sodium thiosulfate injection)

Pirfenidone

Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat)

Pradaxa® (dabigatran etexilate) oral pellets – pediatric

Roctavian™ (valoctocogene roxaparvovec-rvox)

Ruconest® [C1 esterase inhibitor (recombinant)]

Takhzyro® (lanadelumab-flyo)

Tiopronin

Tzield® (teplizumab-mzwv)

Veopoz™ (pozelimab-bbfg)

Vigabatrin

Viltepso® (viltolarsen)

Vimizim® (elosulfase alfa)

Oxlumo® (lumasiran)

Onpattro® (patisiran)

Esbriet® (pirfenidone)

Emflaza® (deflazacort)

Evkeeza® (evinacumab-dgnb)

Exondys 51® (eteplirsen)

Fabrazyme® (agalsidase beta)

Firazyr® (icatibant)

Haegarda® [C1 esterase inhibitor subcutaneous (human)]

Hemgenix® (etranacogene dezaparvovec-drlb)

Icatibant Acetate

Intravenous Immunoglobulin (IVIG)

Ofev® (nintedanib)

Ingrezza® (valbenazine)

Nexviazyme® (avalglucosidase alfa-ngpt)

Naglazyme® (galsulfase)

MACI® (autologous cultured chondrocytes on a porcine collagen membrane)

Lumizyme® (alglucosidase alfa)

Mifepristone

Kalbitor® (ecallantide)

Aldurazyme® (laronidase)

Amondys 45™ (casimersen)

Amvuttra® (vutrisiran)

Arcalyst® (rilonacept)

Austedo® (deutetrabenazine)

Austedo® XR (deutetrabenazine)

Berinert® [C1 esterase inhibitor (human)]

Brineura® (cerliponase alfa)

Cerdelga® (eliglustat)

Cerezyme® (imiglucerase)

Cinryze® [C1 esterase inhibitor (human)]

Crysvita® (burosumab-twza)

Dojolvi® (triheptanoin)

Elaprase® (idursulfase)

Elelyso® (taliglucerase alfa)

Elevidys™ (delandistrogene moxeparvovec-rokl)

Elfabrio® (pegunigalsidase alfa-iwxj)

Adzynma (ADAMTS13, recombinant-krhn)