Biologics

BioMarin Asked to Submit New Data on Gene Therapy

As expected, BioMarin Pharmaceutical says FDA has requested results from an upcoming three-year data analysis from the ongoing Phase 3 GENEr8-1 study on its resubmitted valoctocogene roxaparvovec, a gene therapy for adults with severe Hemophilia A that is currently under BLA review. The agency also told the company that it has scheduled a pre-licensure inspection of BioMarin’s gene therapy manufacturing facility in Novato, CA.

Last month, FDA accepted for review BioMarin’s BLA resubmission and set a user fee review action target date of 3/31. Previously, BioMarin said it expects an additional three months may be added to the review “based on the number of data read-outs that will emerge during the procedure.” It now notes that the three-year data analysis just requested may result in a three-month review extension.

The resubmission includes the company’s response to a 2020 complete response letter and subsequent agency feedback, including two-year outcomes data from the global GENEr8-1 Phase 3 study and supportive data from five years of follow-up from the ongoing Phase 1/2 dose escalation study, BioMarin says. It also proposes a long-term extension study following all clinical trial participants for up to 15 years, as well as a post-approval registry study to follow patients dosed in a real-world setting.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome