Biologics

Krystal Biotech BLA for Topical Gene Therapy

Krystal Biotech has submitted a BLA for B-Vec (beremagene geperpavec) and its use in treating patients with dystrophic epidermolysis bullosa (DEB). The topical gene therapy is designed to treat DEB at the molecular level by providing the patient’s skin cells with two copies of the COL7A1 gene to make functional COL7 protein, which addresses the underlying disease-causing mechanism, according to the company.

The submission is supported by data from two placebo controlled clinical trials — the GEM-3 trial (NCT04491604) and the GEM-1/2 trial (NCT03536143). The company says that in the GEM-3 trial, the primary endpoint of complete wound healing at six months was met and so was the secondary endpoint of complete wound healing at three-months. In GEM-1/2 trial, both mechanistic and clinical endpoints were met.

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