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Alnylam Receives Positive CHMP Opinion for GIVLAARI™ (givosiran) for the Treatment of Acute Hepatic Porphyria in Adults and Adolescents
Jan 31, 2020
− European Commission Decision Expected in March −
− Positive Opinion is Based on Data from the Pivotal ENVISION Phase 3 Study −
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jan. 31, 2020-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending approval of givosiran, an RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older. If approved by the European Commission, givosiran will be commercialized under the brand name GIVLAARI™.
“Today’s CHMP positive opinion is very encouraging news for patients living with acute hepatic porphyria and recognizes the potential of givosiran to address the urgent unmet need that exists for those living with this ultra-rare, inherited disease,” said Barry Greene, President, Alnylam Pharmaceuticals. “We are committed to bringing givosiran to patients in Europe as rapidly as possible.”
“Acute hepatic porphyria is characterized by debilitating, potentially life-threatening attacks and chronic disease manifestations that dramatically impact the lives of patients and their families,” said Bernhard Kaumanns, Head of Medical Affairs, Europe, Middle East, Africa and Canada, at Alnylam. “We have seen in clinical trials that givosiran has the potential to reduce the frequency of attacks, reduce pain and improve patients’ quality of life. Today’s recommendation takes us a significant step closer to making an important new treatment option a reality for AHP patients in Europe.”
The positive opinion is based on efficacy and safety findings from the pivotal ENVISION Phase 3 study, including data on the reduction in the annualized rate of composite porphyria attacks compared with placebo. Findings were presented in April 2019 at the 54th Annual International Liver Congress of the European Association for the Study of the Liver (EASL).
Givosiran was granted Priority Medicines (PRIME) Designation by the EMA as well as Orphan Designation in the European Union. Givosiran was also granted an accelerated assessment by the EMA, which is awarded to medicines deemed to be of major public health interest and therapeutic innovation, and the award is designed to bring new treatments to patients more quickly. This positive CHMP opinion follows the recent approval of GIVLAARI™ (givosiran) by the Food and Drug Administration in November 2019.
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 subcutaneous 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), toward 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.