Drugs

Stealth Submits Elamipretide NDA Over FDA Concerns

Acting on the wishes of the Barth syndrome patient community, Stealth BioTherapeutics says it has submitted an NDA to FDA for elamipretide to treat Barth syndrome. A company statement says it submitted the NDA despite an FDA recommendation that additional controlled data be generated to support NDA review.

“Neither FDA nor the company has identified a feasible trial design due to the ultra-rare nature of this disease,” Stealth says. “In light of FDA’s view that existing clinical data are insufficient to demonstrate substantial evidence of effectiveness and would not support FDA review, there is no assurance that FDA will file the NDA. Stealth believes, however, that the data could support an NDA review, and has accordingly submitted the NDA as requested by the Barth syndrome patient community.”

Barth syndrome is described as an ultra-rare genetic condition characterized by cardiac abnormalities often leading to heart failure and reduced life expectancy, recurrent infections, muscle weakness, and delayed growth. There are no FDA-approved treatments available.

The company says the NDA submission is based on results from the SPIBA-001 Phase 3 retrospective natural history control trial that compared data from the open-label portion of the TAZPOWER Phase 2/3 clinical trial to matched natural history controls. SPIBA-001 met its primary and most secondary endpoints, Stealth says, demonstrating elamipretide-mediated improvements in assessments of exercise tolerance, strength, and cardiac function that are unexpected in the natural course of the progressively debilitating disease. Stealth also says improvements were observed during the TAZPOWER Phase 2/3 trial and open-label extension in several surrogate and intermediate clinical endpoints that may be reasonably likely to predict clinical benefit for patients suffering from the disease, potentially supporting an accelerated approval pathway.

Elamipretide was previously granted a rare pediatric designation, fast-track designation, and orphan drug designation by FDA.

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

Read more

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