Rare diseases
Despite the added survival benefit for its drug, Alnylam still faces steep competition from Pfizer, whose ATTR-CM therapies have become established treatment options.
MBX Biosciences follows Bicara Therapeutics and Zenas Biopharma, which also announced last week their respective plans to go public in a potential boost to the recent slump in the IPO market.
With the help of third-party investors, the new venture will focus on three genetic and rare diseases: tuberous sclerosis complex, erythropoietic protoporphyria and alpha-A1 antitrypsin deficiency.
The entry of new players and new approaches into the ATTR-CM space could help bring down the cost of treatment, experts say.
Ascendis Pharma’s palopegteriparatide was previously rejected by the regulator due to manufacturing problems, and the review period for its resubmitted application was extended by three months.
In the next two weeks, the FDA will hand down its verdicts for three drug applications, including ones for multiple myeloma and hypoparathyroidism therapies.
The company is projecting that future growth will be driven by geographic and label expansions for its rare disease assets, as well as potential approvals in obesity.
Experts say the time is now to develop and provide widespread access to genetic medicines for the rarest diseases. What’s more, they say it is a moral imperative.
An FDA advisory committee agreed on Friday that Zevra Therapeutics had provided sufficient efficacy data supporting the approval of arimoclomol for Niemann-Pick disease type C.
Poor efficacy data for two early-stage candidates for the rare disease alpha-1 antitrypsin deficiency have convinced Vertex Pharmaceuticals to terminate their development.
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