FDA Declines to Approve BioMarin's Gene Therapy for Hemophilia A

By Reuters Staff

August 20, 2020

(Reuters) - The U.S. Food and Drug Administration has declined to approve BioMarin Pharmaceutical Inc's gene therapy for hemophilia A, citing the need for two-year data on the therapy, the drugmaker said on Wednesday.

The company, and Wall Street, had expected the drug to be approved this month after the gene therapy had shown in clinical trials that it reduced the bleeding rate of patients with the disorder, decreasing or eliminating the need for treatment.

BioMarin had said the gene therapy could be priced in a range of $1 million to $3 million.

The medical community and payers have debated whether the price is warranted since it is not yet clear for how long the gene therapy will remain effective in patients.

The company said on Wednesday that the FDA had changed its request, asking for two-year data from an ongoing late-stage study of the therapy for the first time. It received the request in a complete response letter from the agency, which was due to respond to the company this week about its application.

"We are surprised and disappointed that the FDA introduced new expectations for the first time in the Complete Response Letter," Chief Executive Officer Jean-Jacques Bienaimé said in a statement.

The therapy, called valoctocogene roxaparvovec, is one of several gene therapies under development that are expected to cost more than $1 million. The treatment aims to enable patients with hemophilia A to make the blood-clotting protein they otherwise lack.