Orsini pharmacy selected to supply Roctavian for hemophilia A patients

Gene therapy by BioMarin won US approval in late June

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Orsini Specialty Pharmacy has been selected to distribute the new gene therapy Roctavian (valoctocogene roxaparvovec-rvox) for use by individuals with hemophilia A, the company announced in a press release.

The one-time treatment from BioMarin Pharmaceutical late last month became the first gene therapy ever approved by the U.S. Food and Drug Administration for eligible adults with hemophilia A.

Now, the specialty pharmacy says its dedicated gene therapy team is ready to start working with patients who want to access the therapy.

“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,” said Brandon Tom, president and CEO of Orsini.

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Most Patients Bleed-free at 3 Years After Roctavian in GENEr8-1 Trial

Roctavian previously approved in EU for hemophilia A

Hemophilia A is caused by mutations that interfere with the production of a clotting protein called factor VIII (FVIII) or disrupt its function. In the most severe form, which affects roughly half of the people with the rare genetic disorder, virtually no FVIII is present. This results in diminished clotting ability and an increased risk of bleeds.

Roctavian is a gene therapy that’s designed to deliver a healthy copy of the gene encoding FVIII to cells in the liver, where most clotting factors are made. That healthy gene copy allows the body to produce a functional version of the FVIII protein. The therapy delivers its genetic payload using an adeno-associated virus called AAV5.

Given via an infusion into the bloodstream, the procedure for administering the therapy should be done under the supervision of a physician with expertise in bleeding disorders.

According to its label, Roctavian should not be given to individuals with antibodies against the FVIII protein or the AAV5 vector. It also should not be used for patients with significant liver damage or ongoing infections.

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.

The therapy’s approval in the U.S. was supported mainly by data from the Phase 3 GENEr8-1 trial (NCT03370913), which tested Roctavian in 134 men with severe hemophilia A. At three years after receiving the therapy’s single dose, most of the men had no clinically relevant bleeds.

Moreover, more than 90% were off standard preventive replacement therapies. Median FVIII activity also remained high, at more than 8% of normal values.

Roctavian also received conditional approval in the European Union, with full approval pending additional clinical data.

“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,” Tom said.