Rare diseases
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.
The FDA is hoping to repurpose GSK’s Wellcovorin for cerebral folate deficiency; Pfizer acquired fast-moving weight-loss startup Metsera for nearly $5 billion after suffering a hat trick of R&D failures; psychedelics are primed for M&A action and Eli Lilly may be next in line; RFK Jr.’s revamped CDC advisory committee met last week with confounding results; and Stealth secured its Barth approval.
By improving gait stability, Ionis’ zilganersen could be “potentially disease modifying,” according to analysts at William Blair.
The FDA’s proposed Rare Disease Evidence Principles review process is a starting point for getting rare disease therapies across the finish line, but industry leaders say there are more concrete steps the regulator could take to help patients.
A decade-long journey has come to an end for Stealth BioTherapeutics and the Barth syndrome community with the first-ever treatment for this uncommon mitochondrial disease. CEO Reenie McCarthy called it a “pivotal victory” that “offers hope for expedited regulatory attention to other ultra-rare diseases.”
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