BioMarin’s hemophilia A drug has been set an Aug. 21 PDUFA date as the California biotech looks to gain a speedy approval for the first-ever gene therapy for the bleeding disease.
The company said Friday morning the FDA had granted its AAV5 gene therapy, valoctocogene roxaparvovec, a quick review and that, at the moment at least, it didn’t see the need for an expert committee to assess the drug, giving it a smoother path to a potential approval.
The FDA has also accepted the premarket approval application for a companion diagnostic test for valoctocogene roxaparvovec, helping identify the patients it can treat. The test is made by ARUP Laboratories, a nonprofit enterprise of the University of Utah and its department of pathology, it said in a statement.
The drug is also under a speedy review in Europe.
It’s not all been smooth sailing: Three-year data on its candidate were reported last May but sparked concerns about the durability of the therapy, also known as "valrox," after factor VIII levels seemed to fall off after 12 to 18 months, raising the possibility that patients might need to be re-dosed to maintain protection against bleeds.
Its main competition could come from Pfizer and Sangamo Therapeutics’ hemophilia A gene therapy SB-525, which reported positive data late last year, with Roche/Spark Therapeutics also in contention with SPK-8011.
Spark, however, suffered a setback after two patients treated with SPK-8011 developed immune reactions, one of which had to be treated in a hospital, but reported encouraging results with its therapy last February. It has since been snapped up in a (protracted) $4.3 billion takeover by Swiss major Roche.
“The hemophilia community has been waiting for decades for gene therapies. The FDA acceptance of the filing and initiation of review for the first gene therapy for hemophilia A builds on years of scientific achievements in improving the standard of care for people with bleeding disorders,” said Doris Quon, M.D., medical director at the Orthopaedic Hemophilia Treatment Center at the Orthopaedic Institute for Children.
“As a treating physician, I look forward to the possibility of having more treatment options for people with hemophilia.”