Skip links

Orsini Selected by BioMarin to Dispense ROCTAVIAN™, the First Gene Therapy for the Treatment of Severe Hemophilia A

Orsini Specialty Pharmacy, a leading independent specialty pharmacy focused on rare diseases and gene therapies, announced that BioMarin Pharmaceutical has selected it to dispense ROCTAVIAN™ (valoctocogene roxaparvovec-rvox). ROCTAVIAN is the first gene therapy for the treatment of severe Hemophilia A. For more information about ROCTAVIAN, see the Full Prescribing Information. Orsini’s dedicated Gene Therapy Care team is trained and ready to accept new patients.

Orsini is proud to add this breakthrough gene therapy to our growing list of gene and cell modifying treatments and expand our long-standing partnership with BioMarin,” said Brandon Tom, Orsini’s President and Chief Executive Officer. “Orsini’s highly experienced Hemophilia Gene Therapy Care Team is committed to working closely with payors and healthcare providers to ensure patients obtain access to ROCTAVIAN as quickly as possible.”

Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about one-third of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have Hemophilia A. Individuals with the most severe form of hemophilia A makeup approximately 50% of the hemophilia A population.

About Orsini Specialty Pharmacy
Providing patients with comprehensive and compassionate care since 1987Orsini Specialty Pharmacy 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 pharmacy distribution, patient services, clinical management, and convenient home infusion services, Orsini delivers customized solutions that simplify 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 Specialty Pharmacy 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 about Orsini Specialty Pharmacy, visit

Click here, to discover how Orsini supports patients with Hemophilia.