REGENXBIO
NEWS
The FDA’s recently altered outlook on the evidence required for approval of rare disease drugs could have immediate benefits for companies including Skyhawk Therapeutics, Capricor Therapeutics and Biohaven.
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.
The resubmission for RGX-121, expected in the third quarter, comes as the FDA has deemed REGENXBIO’s existing data “sufficient” to support an accelerated filing. It immediately follows a similar reversal of position regarding uniQure’s embattled Huntington’s disease gene therapy.
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.
Fallout from the resignation of FDA Commissioner Marty Makary continues as several other senior regulators are removed from their posts; pharma’s top paid CEOs make up to 358 times more than their employees; Revolution Medicine’s pancreatic cancer results highlight movement in the deadly disease space; more.
Shares of REGENXBIO declined 37% on a mixed data readout and other updates from the company’s first quarter earnings call Thursday.
Comprehending the spate of recent rejections in the cell and gene therapy space may require looking no further than early-stage clinical trials of candidates from REGENXBIO, Excision BioTherapeutics and Intellia Therapeutics.
Avlayah is the first Hunter syndrome therapy approved to address the condition’s neurologic complications, according to Tracy Beth Høeg, acting director of the Center for Drug Evaluation and Research.
The Hunter syndrome space suffered a setback in February when the FDA turned down REGENXBIO’s investigational gene therapy, raising urgent questions about whether competitor Denali Therapeutics can clear the agency’s bar next month.
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