Rare diseases
With a Friday advisory committee meeting looming, the sole indication for Intercept Pharmaceuticals’ Ocaliva appears to be at risk as the regulator flags issues regarding its post-marketing results.
No patients have received Casgevy, CRISPR Therapeutics and Vertex Pharmeceuticals’ recently approved sickle cell gene therapy. Experts weigh in on the path to profit for the treatment and the therapeutic class in general.
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 agency’s inertia and bureaucratic roadblocks are throttling hope for millions of patients. A new center of excellence would provide a solution.
The FDA has three regulatory milestones in the next two weeks, including a decision on a subcutaneous formulation of an effective multiple sclerosis therapy.
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.
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