Drugs

LogicBio Clinical Hold Lifted by FDA

FDA has lifted a clinical hold on LogicBio Therapeutics’ Phase 1/2 SUNRISE clinical trial involving LB-001 in pediatric patients with methylmalonic acidemia (MMA), a mutation in a gene called methylmalonyl-CoA mutase that prevents the body from properly processing certain fats and proteins. According to the company, four patients have been dosed in the SUNRISE trial with LB-001, a single-administration, adeno-associated virus (AAV) genome editing therapy. Two of the patients reportedly experienced a drug-related serious adverse event (thrombotic microangiopathy (TMA)), which it says has been previously reported in association with other AAV genetic therapies. LogicBio says the next patient in the trial will be dosed next quarter.

The company says it has amended the SUNRISE protocol and expects to proceed with dosing after it implements the changes, which include enhanced monitoring measures, such as frequent testing for complement activation, a characteristic of TMA, as well as the use of a complement inhibitor in the event there are laboratory findings indicating a potential TMA.

LB-001 is described as a first-in-class, single-administration, genome editing therapy for early intervention in MMA using LogicBio’s proprietary GeneRide drug development platform. “GeneRide technology utilizes a natural DNA repair process called homologous recombination that enables precise editing of the genome without the need for exogenous nucleases and promoters that have been associated with an increased risk of immune response and cancer,” the company says.

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