BioMarin, Spark Hemophilia Gene Therapies Progress As Accelerated Approval Favors Their Indications
As updated Phase I/II results for BioMarin's hemophilia A and Spark's hemophilia B gene therapies were presented on May 22, it was revealed that FDA plans to make hemophilia the first gene therapy indication eligible for accelerated approval based on a surrogate endpoint.
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During BioMarin’s earnings call Feb. 21, CEO Bienaime indicated the company may be looking to price its valoctocogene roxaparvovec gene therapy at $2m-$3m, as it would represent a savings over the annual cost of infused clotting factor therapy.
The Dutch company's update on AMT-061 and the initiation of a pivotal trial called HOPE-B of around 50 patients could put the product in pole position to be the first gene therapy on the market for the severe orphan blood clotting disorder.
EU approval and coming launch there of Jivi will complement Bayer's two existing Factor VIII treatments, Kogenate and its successor Kovaltry.