With New Data, Alnylam Could Seek Speedy OK For Second RNAi Drug
Summary
Alnylam (NASDAQ: ALNY ), of Cambridge, MA, said this morning that it could soon file for accelerated approval of an RNAi drug, givosiran, that the company is developing for acute hepatic porphyria , a rare genetic disease that causes potentially deadly bouts of abdominal pain.The company isn’t providing details, but said that in the interim look at a 43-patient, Phase 3 study, givosiran led to a statistically significant reduction of aminolevulinic acid, or ALA, in patients’ urine—a marker of the disease—after three months of treatment.As such, Alnylam plans to discuss the data with the FDA and, depending on the outcome of the talks, file an application by the end of the year seeking accelerated approval.The primary goal of Alnylam’s trial isn’t to drop ALA—it’s to show whether givosiran can reduce the rate of the disease’s hallmark attacks after six months of treatment, compared to a placebo.While the side effects disclosed do raise questions, Stifel analyst Paul Matteis wrote, the data “do not suggest that there’d ultimately be a regulatory/commercial risk with the drug” assuming the benefits are similar to what givosiran showed in earlier testing.