Rare diseases
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.”
The House Committee on Energy and Commerce has cleared proposed legislation that could bring back the FDA’s rare pediatric priority review voucher program, which allows for expedited drug reviews.
After decades without much movement, a handful of new treatments for this rare autoimmune disease are now approved, and several companies, including argenx and Regeneron, have recently released promising late-stage trial results.
On the FDA’s docket for the back half of September is Merck’s proposed subcutaneous formulation of its blockbuster cancer drug Keytruda.
Ocaliva won accelerated approval for primary biliary cholangitis in 2016, which the FDA refused to upgrade to full approval last year after an advisory committee was unable to verify a favorable risk/benefit profile.
After receiving a complete response letter for its pyruvate dehydrogenase complex deficiency candidate, Saol Therapeutics anticipates it will need “several years” and “significant financial resources” to address the FDA’s concerns.
While the new framework signals continued flexibility at the FDA regarding rare disease approvals, some analysts and advocates question what tangible impacts the new guidelines will have.
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