Drugs

Amylyx Clinical Hold Lifted on ALS Drug

FDA has lifted a clinical hold on an Amylyx Pharmaceuticals Phase 1 clinical trial of AMX0114, an investigational antisense oligonucleotide targeting calpain-2 for treating amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease). The company plans to initiate the Phase 1 LUMINA clinical trial in Canada early this year. LUMINA is described as a multicenter, randomized, placebo-controlled, multiple ascending dose trial evaluating the safety and biological activity of AMX0114 in 48 patients.

Last year, FDA issued the hold after the company submitted an IND and the agency requested additional information and restricted dosing to an amount that is lower than the company’s proposed starting dose of 12.5 mg. “Toxicology data from studies showed a greater than 10X safety margin at the starting dose of 12.5 mg based on the no observed adverse effect level (NOAEL) determined by independent toxicology firms,” Amylyx says.

LUMINA will also assess ALS biomarkers, the company says, including change from baseline in neurofilament light (NfL) levels. “Based on several preclinical efficacy studies and what is known about the central role of calpain-2 in the process of axonal degeneration, we believe AMX0114 has the potential to be a treatment for ALS and other diseases,” it 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