The FDA approved Roctavian for treating hemophilia A. The regulatory decision makes the BioMarin Pharmaceutical product the first gene therapy for this inherited bleeding disorder.
BioMarin Pharmaceutical said the layoffs will improve efficiency across the company as it prepares for the potential U.S. launch of Roctavian, a gene therapy for severe hemophilia A. The one-time treatment was approved in Europe in August.
Munck Wilson Mandala Partner Greg Howison shared his perspective on some of the legal ramifications around AI, IP, connected devices and the data they generate, in response to emailed questions.
BioMarin Pharmaceutical’s Voxzogo is the first FDA approved therapy for achondroplasia, an inherited disorder that causes the most common form of dwarfism. With approvals in Europe and the U.S., and more regulatory decisions expected in 2022, the biotech said the drug has blockbuster potential.
A Phase 1/2 study of BioMarin Pharmaceutical’s gene therapy for phenylketonuria has been placed under a clinical hold after interim results from a preclinical study showed that some mice developed liver tumors. Patients did not receive the same high dose that was given to the mice, and no one in the clinical trial has developed cancer.
The European Commission has approved a BioMarin Pharmaceutical drug that treats achondroplasia, a rare genetic disorder that slows bone growth and is the most common cause of dwarfism. The commission also granted conditional approval to a cancer drug from partners Incyte and MorphoSys.
The biotech company said the FDA had given priority review to valoctocogene roxaparvovec for hemophilia A in adults, with the PDUFA date set at Aug. 21.
At the Abarca Forward conference earlier this year, George Van Antwerp, managing director at Deloitte, discussed how social determinants of health and a personalized member experience can improve medication adherence and health outcomes.
An analyst wrote that while the Phase III data - released alongside Phase I/II data - for valrox could be enough for accelerated FDA approval and appeared to allay concerns about declining Factor VIII levels, it could face commercial challenges.