Alnylam Completes Rolling Submission of New Drug Application to U.S. Food and Drug Administration (FDA) for Givosiran for the Treatment of Acute Hepatic Porphyria

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today the completion of the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in development for the treatment of acute hepatic porphyria (AHP). Givosiran previously received Breakthrough Therapy Designation from the FDA and Orphan Drug Designation in the U.S. for AHP.

“We’re very pleased to have completed our NDA filing for givosiran, just 90 days since reporting our positive topline ENVISION Phase 3 study results, consistent with our commitment to bring givosiran to patients as rapidly as possible. ENVISION demonstrated that givosiran substantially reduced the annualized rate of composite porphyria attacks, relative to placebo, with an overall safety and tolerability profile that we believe is encouraging in the context of this high unmet need disease,” said Akin Akinc, Ph.D., Vice President and General Manager, Givosiran Program at Alnylam. “Based on these results, we believe givosiran has the potential to be a life-changing medicine for patients living with AHP. With this submission, we are now one step closer to achieving our goal of providing a new therapeutic option for the treatment of AHP. We look forward to working with the FDA and other global regulatory agencies to bring this medicine to patients.”

Givosiran has also been granted Priority Medicines (PRIME) Designation by the European Medicines Agency (EMA) and Orphan Drug Designation in the EU for AHP. Alnylam intends to file a Marketing Authorisation Application (MAA) in mid-2019.

About Givosiran

Givosiran is an investigational, subcutaneously administered RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in development for the treatment of acute hepatic porphyria (AHP). Monthly administration of givosiran has the potential to significantly lower induced liver ALAS1 levels in a sustained manner and thereby decrease neurotoxic heme intermediates, aminolevulinic acid (ALA) and porphobilinogen (PBG), towards normal levels. By reducing accumulation of these intermediates, givosiran has the potential to prevent or reduce the occurrence of severe and life-threatening attacks, control chronic symptoms, and decrease the burden of the disease. Givosiran utilizes Alnylam’s Enhanced Stabilization Chemistry ESC-GalNAc conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index. The safety and efficacy of givosiran were evaluated in the ENVISION Phase 3 trial with positive results; these results have not been evaluated by the FDA, the EMA or any other health authority.