BioMarin, Spark Hemophilia Gene Therapies Progress As Accelerated Approval Favors Their Indications
Executive Summary
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.
You may also be interested in...
BioMarin CEO Suggests Hemophilia Gene Therapy Pricing In $2m-$3m Range
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.
UniQure Has High HOPE For Hemophilia B Gene 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.
Bayer's Hemophilia A Portfolio Boosted by Jivi's EU Approval
EU approval and coming launch there of Jivi will complement Bayer's two existing Factor VIII treatments, Kogenate and its successor Kovaltry.
Need a specific report? 1000+ reports available
Buy Reports