BioMarin Pharmaceutical CEO Jean-Jacques Bienaimé will retire in December, when he will be succeeded by Genentech CEO Alexander Hardy. The transition comes with BioMarin in the early stages of the commercial launch of Roctavian, the first FDA-approved hemophilia A gene therapy.
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.
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 submission of additional data for patients treated with its hemophilia A gene therapy, Roctavian, mean that the FDA will push out a regulatory decision to early summer. But it could be worth the wait as analysts anticipate FDA approval and project Roctavian becoming one of BioMarin’s biggest products.
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.
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.
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.
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.
In a CRL issued for valoctocogene roxaparvovec, the FDA told BioMarin that differences between its Phase I/II and Phase III studies limited the ability to rely on the earlier trial to support durability of effect, which an analyst said likely referred to a product comparability issue.
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.
Plus, health insurance startup Oscar to build concierge clinic in Arizona, AIDS Healthcare Foundation wants the FDA and Congress to investigate Gilead and eHealth hires its new CEO from Playboy.
The white knight moment has come for a Texas lawyer who has spent the past two months amassing an “army” of virtual supporters in her quest for access to an experimental cancer drug. Only the turnout isn’t exactly as she had imagined. Andrea Sloan, who has gathered nearly 210,000 signatures on a petition to BioMarin […]
We will highlight Build My Health's revenue practice management tools, which could help physician practices add up to $250,000 to their practices.
For the past seven years, Andrea Sloan has been battling ovarian cancer. Now, she’s waging a second battle – this one against BioMarin Pharmaceuticals (NASDAQ:BMRN) – after being denied access to an experimental drug she thinks is her last hope. And in the process, she’s raising some tough questions about the process of clinical trials […]
BioMarin Pharmaceutical Inc. has received orphan drug status for an investigational enzyme replacement therapy to treat Pompe disease that was developed by ZyStor Therapeutics in Milwaukee, Wisconsin. Just two weeks ago, BioMarin bought ZyStor for $22 million in cash.