Duchenne muscular dystrophy
Many of the FDA’s decisions this quarter involve applications that have previously been delayed, declined or outright rejected, including one for an mRNA vaccine that became the center of controversy earlier this year.
Sarepta Therapeutics is seeking to convert the accelerated approval of its therapeutic exon-skippers for Duchenne muscular dystrophy to full despite the drugs’ failure to improve motor function in a confirmatory trial.
The FDA plans to hold an advisory committee meeting to discuss Capricor Therapeutics’ application for deramiocel, which the agency rejected last July. The news surprised CEO Linda Marbán, who told BioSpace the FDA has not communicated any issues of concern with the company’s resubmitted application.
A few short days after announcing an FDA pivot on a separate asset, REGENXBIO is planning to test the agency’s apparent newfound rare disease outlook on another late-stage gene therapy.
Edgewise Therapeutics will now focus on a handful of cardiovascular programs including EDG-7500 for hypertrophic cardiomyopathy thanks to the non-dilutive capital from France’s Servier.
Analysts homed in on Duchenne muscular dystrophy and myotonic dystrophy type 1 assets during first quarter earnings as major players like REGENXBIO and Novartis as well as Dyne, Wave, Solid and Sarepta near the regulatory finish line.
Shares of REGENXBIO declined 37% on a mixed data readout and other updates from the company’s first quarter earnings call Thursday.
Entrada Therapeutics saw a 2.36% dystrophin increase in patients treated with its oligonucleotide asset—a number that fell short of the company’s prior guidance of a double-digit improvement.
Sarepta Therapeutics has put in place several initiatives to help its gene therapy Elevidys return to growth, but recovery will take a long time, according to company executives.
As Q1 earnings arrive, three biotechs have big quarters ahead, with two—Amylyx and Neumora Therapeutics—betting at least partly on novel assets for obesity.
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