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Alnylam Announces Approval of GIVLAARI® (givosiran) in the European Union for the Treatment of Acute Hepatic Porphyria (AHP) in Adults and Adolescents
− GIVLAARI is the First and Only Therapy Proven to Prevent AHP Attacks, Reduce Chronic Pain and Improve Quality of Life − − GIVLAARI Approved Based on
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n− GIVLAARI is the First and Only Therapy Proven to Prevent AHP Attacks, Reduce Chronic Pain and Improve Quality of Life −\n\n\n− GIVLAARI Approved Based on ENVISION Phase 3 Study Results Showing a 74% Reduction in the Rate of Porphyria Attacks −\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the European Commission (EC) has granted marketing authorization for GIVLAARI® (givosiran), an injection for subcutaneous use targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older. AHP is an ultra-rare condition in which patients can experience debilitating attacks of severe abdominal pain, vomiting and seizures, which can be life-threatening due to the possibility of paralysis and respiratory arrest during attacks. Many patients also experience chronic symptoms, including pain, which continue to be present between attacks.\n\n\n“Today’s approval of GIVLAARI marks a historic moment for patients and families living with this devastating genetic disease, as there are currently no approved medicines in Europe proven to decrease the frequency of attacks and reduce the chronic pain that many patients suffer,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “We are proud to bring GIVLAARI, our second RNAi medicine to be approved in the last 18 months, to patients in Europe and we want to thank the patients, families, investigators and study staff whose support and involvement have made this achievement possible.”\n\n\n“The fear of not knowing when an attack will strike, combined with ongoing symptoms between attacks, affects every aspect of patients’ lives, limiting their ability to work and maintain a social life,” said Dr Eliane Sardh, Head of the Porphyria Centre Sweden, Karolinska University Hospital, Sweden. “In our experience, life is very different for patients since they have been treated with givosiran. In addition to a reduction in the number of porphyria attacks which require hospitalization and urgent healthcare visits, we have seen improvements in how patients report their overall health status and quality of life, so this approval is truly meaningful for patients, their families and the healthcare professionals who treat t...