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BioMarin Reports Stable and Durable Annualized Bleed Control for Roctavian,

January 9, 2023

BioMarin Pharmaceutical reported more than three years of follow up from its ongoing global phase 3 study of its experimental hemophilia A one-and-done gene therapy Roctavian (valoctocogene roxaparvovec) showed it remained effective.

Photo: Hank Fuchs, president of worldwide research and development at BioMarin

The company said at the end of year three, 92 percent of patients remained off prophylaxis. Those patients who returned to factor VIII or emicizumab prophylaxis did so safely. The company said it expects to share addition data from the study at upcoming medical meetings.

“The three-year data reinforce our belief that Roctavian has the potential to fundamentally transform the treatment of severe hemophilia A for patients and eliminate the burden of prophylaxis,” said Hank Fuchs, president of worldwide research and development at BioMarin.

BioMarin said the FDA completed a Pre-License Inspection of the manufacturing facility in early December. The company has provided responses to the comments and observations received at the close of the inspection, and the company believes all are addressable.

BioMarin said the new data will be shared with the FDA as part of the agency’s ongoing review of the Biologics License application of Roctavian. The agency is expected to act on the application by March 31, 2023.

Hemophilia A 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 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have hemophilia A. People with the most severe form of hemophilia A (factor VIII levels <1 percent) often experience painful, spontaneous bleeds into their muscles or joints. Individuals with the most severe form of hemophilia A make up approximately 50 percent of the hemophilia A population.

“We continue to learn more about the durability, safety and efficacy of valoctocogene roxaparvovec,” said Steven Pipe, professor of pediatrics and pathology at the University of Michigan and an investigator in the phase 3 study. “I am encouraged to see the consistent clinical response and the significant number of study participants who remain off prophylaxis after three years. This shows the potential transformative impact of this single treatment event for people with severe hemophilia A.”

The European Commission granted conditional marketing authorization to Roctavian on August 24, 2022. As previously announced, BioMarin is targeting outcomes-based agreements with the three largest health insurance groups that represent about 80 percent of German lives. The company has executed an agreement with one of the three.

The company expects to sign additional agreements in the coming weeks in Germany and continues to progress the European launch of Roctavian on a country-by-country basis, including meetings with authorities in France and the submission of the reimbursement dossier in Italy.

For covered patients, the agreements provide for companion diagnostic testing and reimbursement of ROCTAVIAN, allowing physicians to prescribe and patients to be treated with therapy.

The agreements in Germany are multiyear agreements that cover payer risk of patients potentially returning to prophylaxis through direct BioMarin financial commitment in return for substantial and full upfront payment.

“We continue to see a high level of interest from physicians in Germany. Treatment centers are ready to go, and our market research indicates that there are approximately 40 patients queued up for pre-treatment screening,” said Jeff Ajer, executive vice president and chief commercial officer of BioMarin. “We are pleased that these outcomes-based agreements will enable access for individuals with severe hemophilia A.”

Author: Rare Daily Staff

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