Drugs

Clene Plans ALS Drug Filing After Input on Accelerated Path

Clene says it plans to submit an NDA in the third quarter for its experimental amyotrophic lateral sclerosis (ALS) therapy, CNM-Au8, following feedback from FDA that indicated the company’s data could support an accelerated approval.

According to the company, FDA officials indicated during a recent Type C meeting that Clene’s dataset “may be capable of supporting” an NDA under the agency’s accelerated approval pathway, which allows earlier authorization of drugs for serious conditions based on surrogate endpoints that are reasonably likely to predict clinical benefit.

Central to the potential filing is the biomarker neurofilament light (NfL), which reflects neuronal damage. FDA said NfL “could potentially serve as a reasonably likely surrogate endpoint,” while emphasizing that Clene’s application would need to demonstrate that reductions in NfL are predictive of meaningful clinical outcomes for ALS patients.

The agency also requested additional analyses linking the magnitude of NfL reduction to clinical benefit, which Clene says it plans to include in its submission. Clene notes it expects to initiate a Phase 3 confirmatory study in early 2027.

CNM-Au8, an oral therapy composed of gold nanocrystals designed to improve cellular energy metabolism and neuronal survival, is being developed as a potential first-in-class treatment for ALS. The planned NDA will be supported by data from multiple mid-stage studies, including the HEALEY ALS Platform Trial and RESCUE-ALS trial, as well as expanded access data. The company says prior analyses showed reductions in NfL were associated with longer survival in an open-label extension study.

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