Rare diseases
BioMarin executives sought to calm an anxious investor base Wednesday with a public address and pledge to achieve a nearly 50% bump in annual revenue by 2027. But analysts were left wanting.
Recursion’s oral drug candidate for cerebral cavernous malformation showed no improvements in patient- or physician-reported outcomes at 12 months. The biotech will engage with the FDA to determine the need for an additional study.
The FDA has three regulatory milestones in the next two weeks, including a decision on a subcutaneous formulation of an effective multiple sclerosis therapy.
The agency’s inertia and bureaucratic roadblocks are throttling hope for millions of patients. A new center of excellence would provide a solution.
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.
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