Biologics

FDA Lifts Hold on Rocket’s Gene Therapy

FDA has lifted a clinical hold on Rocket Pharmaceuticals’ pivotal Phase 2 trial of RP-A501, an investigational gene therapy for Danon disease, allowing the study to resume less than three months after being paused. The hold followed the death of a participant who experienced serious complications during the study.

The agency gave the company the green light to proceed after approving Rocket’s proposed protocol adjustments, including a recalibrated starting dose in three sequentially treated patients, each with a minimum four-week interval between dosing, according to the company. The modified dose reflects the lower end of the ranges shown to be effective in an earlier Phase 1 study while optimizing the safety profile, it says.

RP-A501 is described as a one-time gene therapy candidate that uses an AAV9 vector to deliver a healthy version of the LAMP2B gene, which is mutated in Danon disease, a rare and often fatal inherited disorder that primarily affects the heart and is estimated to impact 15,000 to 30,000 people across the U.S. and Europe. Currently, heart transplantation is the only available treatment.

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