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Alnylam’s Givosiran Cuts Pain Attacks From Rare Genetic Disease

March 6, 2019, 3:01 PM

Alnylam Pharmaceuticals Inc.’s experimental drug givosiran significantly reduced painful attacks caused by a rare liver disease in a key study, though patients receiving it were also more likely to develop serious side effects.

Alnylam plans to complete applications for U.S. and European regulatory approval of the medication by mid-2019, potentially clearing the way to launch the company’s second product by this time next year, said Chief Executive Officer John Maraganore. There are few treatments available for the condition known as acute hepatic porphyria, a genetic disease marked by bouts of severe abdominal pain, nausea, vomiting and fatigue.

The study ...