Alnylam announces plan to initiate rolling submission of NDA for givosiran
Alnylam Pharmaceuticals announced that in consultation with the U.S. Food and Drug Administration, the Company plans to pursue a full approval based on the complete results of the ENVISION Phase 3 study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 for the treatment of acute hepatic porphyria, rather than filing based on the interim Phase 3 results. The FDA has also agreed to a rolling submission of a New Drug Application, which will be initiated in 2018 with full clinical sections submitted in mid-2019, assuming positive study results. Alnylam previously reported positive topline results from the interim analysis of the ENVISION Phase 3 study of givosiran demonstrating a statistically significant reduction in urinary ALA levels, a surrogate biomarker that is reasonably likely to predict clinical benefit. As previously reported, serious adverse events were reported in 22 percent of givosiran patients and 10 percent of placebo patients in the interim analysis cohort of 43 patients, with one patient on givosiran discontinuing treatment due to an increase in liver transaminase that resolved. Alnylam continues to dose patients in the ongoing ENVISION study, where enrollment was completed ahead of schedule with 94 AHP patients. The Company expects to report topline full study results of the primary endpoint - the annualized attack rate after six months of treatment - in early 2019. As previously guided, the Company intends to file for marketing authorization in all other markets based on the complete results of the ENVISION Phase 3 study, assuming positive results.