Rare diseases
After a year of controversial regulatory reversals, the FDA has accepted uniQure’s application for ifezuntirgene inilparvovec for priority review, with a May 1 action date. If approved, the therapy—also called AMT-130—would be the first disease-modifying treatment for Huntington’s disease.
Former biologics boss Peter Marks had a vision. With the FDA’s latest draft guidance, it’s coming to fruition. Speaking with BioSpace, current CBER leader Karim Mikhail said the “adapted pathway” is designed to develop personalized gene therapies more quickly.
If approved, BridgeBio Pharma’s BBP-418—which, according to one Jefferies analyst, boasts “pretty darn compelling” data—would be the first therapy for limb-girdle muscular dystrophy. The FDA has an action date of Nov. 27.
Setara Biosciences has formed around a handful of assets from Actio Biosciences, which was acquired by Jazz Pharmaceuticals earlier this year.
After Sanofi paid $600 million in cash to acquire Dren Bio’s bispecific last year, the biotech has secured a hefty financing to fund late-stage development and commercial preparations for its lead asset.
The FDA has outlined a process for allowing a single application to cover multiple variants of a gene therapy. At the same time, the Centers for Medicare & Medicaid Services has revealed plans to expand the Cell and Gene Therapy Access Model to increase patient access to these products.
As both Taysha Gene Therapies and Neurogene warm up for an important year of readouts, the Rett syndrome community is hopeful of seeing a therapy that can truly change the disease’s path.
RNAi specialist Alnylam Pharmaceuticals will advance the Huntington’s disease drug brezosiran into a placebo-controlled study in 2027 once regulatory alignment is achieved.
After cardiac safety signals led to a full clinical hold on Aardvark Therapeutics’ studies of ARD-101, the biotech conducted an additional safety study, in which no cardiac events were detected. Aardvark plans to resume testing the candidate next year.
Troriluzole, in development for spinocerebellar ataxia, was rejected nearly a year ago when the FDA took issue with the design of Biohaven’s registrational study. The agency has now agreed to review a new application and render a decision in the first quarter of 2027.
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