Rare diseases
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.
The FDA approved the expansion of Casgevy, which had previously been greenlit for patients 12 and up, into a younger pediatric population under the agency’s Commissioner’s National Priority Voucher program.
Ipsen is penning its second acquisition of the week, this time securing Memo Therapeutics and its midstage monoclonal antibody in a deal that could approach $800 million.
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.
Earlier this year, Amgen refused the FDA’s request to withdraw Tavneos from the market. Now, two researchers who participated in the original study to support the drug’s approval claim they did not know the primary endpoint was readjudicated after the study was unblinded.
The star of Ipsen’s acquisition is an MDM2 blocker being proposed as an add-on therapy to ruxolitinib for myelofibrosis. The drug could be available to patients “as early as 2028,” according to Ipsen CEO David Loew.
BridgeBio Pharma plans to file for approval in the third quarter. If granted, the oral drug could present a “highly differentiated” option over current achondroplasia therapies, according to Jefferies.
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.
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.
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