Shares of gene therapy companies — including Sarepta Therapeutics, Avidity Biosciences and Beam Therapeutics — got a lift Monday after Food and Drug Administration Commissioner Marty Makary laid out plans for a new accelerated approval pathway.
Makary spoke with TV personality Megyn Kelly on Sirius XM in his first big interview since the Senate confirmed his appointment to the FDA.
During the hourlong discussion, Makary laid out a concept for approving drugs based on meeting a "plausible mechanism." This would benefit rare-disease companies where multiple placebo-controlled studies aren't always possible, RBC Capital Markets analysts said in a report.
They noted that a lot of Makary's ideas are already in practice.
"This suggests Makary may not dramatically break from this trend of biomarker-based accelerated approvals followed by confirmatory studies — potentially positive for PTCT, SRPT, BHVN, EWTX, DSGN, RARE, DYN, WVE, MGTX, QURE, REGN, LXEO — though we believe fewer FDA resources could still make this harder to implement in practice," RBC analysts said.
Sarepta Therapeutics, Genetics Stocks Rise
Sarepta Therapeutics stock rose nearly 3% to 55.24, while Beam and Avidity added roughly 7% apiece. Uniqure advanced 2.2%. Dollar stocks Regenxbio and Dyne Therapeutics each jumped by double digits. Intellia Therapeutics, which is working on gene-editing drugs, surged 9.5%.
Makary's suggested pathway closely echoes the FDA's current accelerated approval pathway, which allows the agency to approve a rare-disease treatment based on one Phase 3 study. The drug must show a benefit on an alternative metric. Often this is a biomarker, a measurable biological characteristic. The company then must prove the drug's merit in a confirmatory study.
He also broached the idea of slimming down the average 10-year approval timeline for most drugs. This could lower research and development costs over time — often in the tens of billions of dollars — but closely echoes other efforts under the previous administration.
"We believe he came across as scientifically-grounded level-headed with regards to registrational requirements (slimmed down animal testing), drug approval processes (aim to expedite on pathway plausibility, particularly for orphan diseases), and modernizing data capture, and willing to work with industry appropriately," RBC analysts said.
Rare-Disease Biotechs In Focus
Makary's comments could particularly help the rare-disease guard of biotech. Sarepta Therapeutics has an approved gene therapy for Duchenne muscular dystrophy. Dyne, Avidity and Regenxbio are trailing with their own Duchenne gene therapies.
Uniqure's leading drug is an experimental treatment for Huntington's disease, while Intellia is testing gene-editing approaches to rare diseases like hereditary angioedema, a swelling condition.
Follow Allison Gatlin on X/Twitter at @AGatlin_IBD.
