Rare diseases
Following up on previous, dimly received issuances, a new set of ideas published by the FDA to streamline regulatory pathways for cell and gene therapies ‘for small populations’ is receiving a warmer welcome—but experts warn it will take more to turn the tide for the fraught therapeutic space.
As with recent rejections for Biogen and Scholar Rock, manufacturing issues stymied a regulatory bid from Fortress Biotech and Sentynl Therapeutics. Fortress said the FDA did not flag problems with the drug’s safety or efficacy.
M&A headlined for a second straight week as Genmab acquired Merus for $8 billion; Pfizer strikes most-favored-nation deal with White House; CDER Director George Tidmarsh caused a stir with a now-deleted LinkedIn post; GSK CEO Emma Walmsley will step down from her role; and uniQure’s gene therapy offers new hope for patients with Huntington’s disease.
Despite tolerability concerns, nomlabofusp’s overall efficacy represents a “large win” for Larimar, according to analysts at William Blair, who lauded the therapy’s functional benefits.
Applied Therapeutics has yet to confirm whether the study, posted on Clinicaltrials.gov on Thursday, means it has indeed aligned with the FDA on govorestat’s development.
The FDA in September issued two rejections for spinal muscular atrophy therapies—both linked to manufacturing problems—and granted approvals in Barth syndrome and for a subcutaneous version of Merck’s Keytruda that could be key to the blockbuster’s future earnings.
From more than 30 target action dates in the last three months of the year, BioSpace has narrowed the list to six regulatory decisions that could have far-reaching implications for biopharma and patients.
The regulatory greenlight was backed by two Phase III trials that showed normalized growth hormone levels in patients with the rare pituitary condition. It’s the first approval for Crinetics Pharmaceuticals and something CEO Scott Struthers predicted “will transform people’s lives.”
While Harmony management has not disclosed future plans for ZYN002, Jefferies analysts expect the asset to be shelved.
Acadia Pharmaceuticals was testing the drug, an intranasal formulation of the oxytocin analogue carbetocin, for its potential to ease hyperphagia in the rare neurological condition.
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