Drugs

Myrtelle Gets 3 FDA Designations for Gene Therapy

FDA has granted Myrtelle a Fast Track, Rare Pediatric Disease, and Orphan Drug designations for its lead clinical-stage gene therapy, rAAV-Olig001-ASPA, for treating patients with Canavan Disease. The condition is a fatal childhood genetic brain disease in which mutations in the Aspartoacylase gene (ASPA) prevent normal Aspartoacylase enzyme expression, a “critical enzyme produced in oligodendrocytes that breaks down the neurochemical N-Acetylaspartate (NAA),” the company says. “When not properly metabolized by oligodendrocytes, NAA accumulates in the brain and negatively affects bioenergetics, myelin production, and brain health.”

An ongoing Phase 1/2 clinical trial utilizes a novel proprietary recombinant adeno-associated virus vector, AAV-Olig, that the company says for the first time directly targets oligodendrocytes in the brain. The oligodendrocyte-targeted gene therapy is intended to restore ASPA function, enable metabolism of the abundant brain chemical NAA, and support myelination.

Myrtelle entered into a licensing agreement with Pfizer Inc. in 2021 to develop and commercialize the gene therapy.

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