Rare diseases
The FDA previously refused to review Biohaven’s candidate in the indication due to a failed late-stage trial. However, the company is now planning to file an NDA in the fourth quarter of 2024.
With the FDA’s approval, Fasenra will compete with GSK’s Nucala, which in December 2017 became the first biologic approved for eosinophilic granulomatosis with polyangiitis.
The FDA has six target action dates ahead to round out September as drugs for gastroparesis, Niemann-Pick disease type C and more await decisions.
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.
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