Orsini the Exclusive Specialty Pharmacy Provider for Gene Therapy KEBILIDI (eladocagene exuparvovec-tneq)
ELK GROVE VILLAGE, Ill. , Jan. 14, 2025 /PRNewswire/ – Orsini announced today that it has been chosen by PTC Therapeutics as the exclusive specialty pharmacy provider for the adeno-associated virus vector-based gene therapy KEBILIDITM (eladocagene exuparvovec-tneq). KEBILIDI is approved by the U.S. Food and Drug Administration for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency and is the seventh treatment to join Orsini’s cell and gene therapy portfolio. View the full Prescribing Information here.
AADC deficiency is a rare and fatal genetic disorder that typically causes severe disability starting during the first months of life. Children living with AADC deficiency can experience seizure-like episodes, frequent vomiting, behavioral problems and complications such as infections and severe feeding and trouble breathing.
“As the leading independent specialty pharmacy in cell and gene therapies, Orsini is well-positioned to help those suffering from AADC deficiency gain access to KEBILIDI,” Brandon Tom, Orsini’s CEO, said. “We are always honored to be entrusted with the care of those affected by rare diseases, particularly pediatric patients and their families, and we look forward to partnering with PTC to offer new hope to the AADC community.”
About Orsini
Providing patients with comprehensive and compassionate care since 1987, Orsini is a leader in rare diseases and gene therapies. Orsini partners with biopharma innovators, healthcare providers and payors to support patients and their families in accessing revolutionary treatments for rare diseases. Through integrated rare disease pharmacy solutions including pharmacy distribution, patient services, clinical management and convenient home infusion services, Orsini simplifies how patients connect to advanced therapies. Orsini’s high-touch care model centers on experienced and trained therapy care teams that provide personalized patient care to ensure that No Patient is Left Behind™.
Orsini holds accreditations with the Accreditation Commission for Health Care (ACHC), The Joint Commission, URAC and NABP. Orsini has earned URAC’s Rare Disease Pharmacy Center of Excellence Designation and ACHC’s Distinction in Rare Diseases and Orphan Drugs. For more information, contact Orsini at 847-734-7373 ext. 505, email us at media@orsinihc.com, or visit www.orsini.com.
Orsini Contact
Morgan Olson
molson@orsinihc.com