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By Caroline Humer
(Reuters) – The U.S. Food and Drug Administration has rejected BioMarin Pharmaceutical Inc’s gene therapy for hemophilia A bleeding disorders, citing the need for longer-term data, the drugmaker said on Wednesday, potentially pushing back any approval until 2022.
Shares of BioMarin fell 31%, or $ 37, to $ 81.54 in the morning session.
The company, Wall Street and doctors expected the therapy to be approved this month after it was shown in clinical trials to reduce the rate of bleeding in patients with the disorder, decreasing or eliminating the need for treatment. .
There are approximately 16,000 patients in the United States with hemophilia A who lack the factor VIII coagulation protein. Standard therapy requires patients to infuse factor proteins into their bloodstream several times a week for life, costing hundreds of thousands of dollars a year. It was hoped that gene therapy would allow patients to forgo or reduce these infusions.
BioMarin had said gene therapy could be offered in the range of $ 1 million to $ 3 million, potentially making it the most expensive one-time treatment in the United States. Gene therapy for spinal muscular atrophy from Novartis AG is priced at $ 2.1 million.
The company said on Wednesday that the FDA had changed its data requirements in its application, requesting full two-year data from an ongoing study at the advanced stage of therapy.
He received the request in a full response letter from the agency, which was due to respond to the company this week about his application.
“We are surprised and disappointed that the FDA has introduced new expectations for the first time in the full response letter,” CEO Jean-Jacques Bienaimé said in a statement.
BioMarin submitted its application based on three-year Phase 1/2 data and an interim analysis of Phase 3 data. Phase 1/2 data had shown that treatment effects waned in year two and continued to wane. but more slowly in years three and four. Treatment is aimed at enabling patients with hemophilia A to make the blood clotting protein that they otherwise lack.
The FDA told BioMarin that the differences between the trials limited its ability to rely on phase 1/2 data in its analysis of how long treatment was effective.
The advanced stage trial completed recruitment in November 2019, making data available for re-filing in late 2021 and approval a year after that, Stifel analyst Paul Matteis said in a research note.
The FDA did not comment immediately.
FDA rejection narrows the primary role of BioMarin’s valoctocogene roxaparvovec, or ValRox, over potential competing treatments from Roche Holdings AG and Pfizer Inc / Sangamo Therapeutics Inc. Pfizer and Sangamo are scheduled to start phase 3 this year while Roche is only due not start his trial until next year.
It may be of benefit to the bispecific antibody Hemlibra from Roche which mimics the function of a missing blood clotting protein to restore the clotting process. It is expected to have sales of $ 2 billion this year.
(Reporting by Caroline Humer in New York, John Miller in Zurich and Manas Mishra in Bengaluru; Editing by Maju Samuel, Steve Orlofsky and Andrea Ricci)
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