In a note to investors, SVB Leerink analyst Joseph Schwartz suggested that the 'questionable' approval of Sarepta's Exondys 51 may have played a role in the FDA's decision to turn down golodirsen.
The decision stood in contrast with the agency's highly controversial approval of Sarepta's previous DMD drug, Exondys 51. The company's shares fell sharply in after-hours trading.
The progress in artificial intelligence (AI) is reshaping patient care, across various dimensions, from facilitating faster discharge to curating treatment plans and suggesting lifestyle changes.
Data from the drugmaker's Phase Ib study showed patients responding, but an analyst wrote that it appears less potent and to have higher toxicity than that of its competitor, Sarepta Therapeutics.
An analyst wrote that the therapy's combination of AAV delivery with a validated means of treating DMD could distinguish it from other gene therapies in what is already a crowded space.
After two painfully incremental drug approvals, the Duchenne muscular dystrophy field is still searching for a therapy that can substantially improve patients’ lives and lifespan. Sounds like a job for CRISPR/Cas9.