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FDA Assigns Goal Date for Mavorixafor for Rare Immunodeficiency Disease
October 31st 2023If approved, mavorixafor would be the first therapy for WHIM syndrome, an ultra-rare disease that can cause recurrent lung infections, papillomavirus-related warts, and an increased risk of developing certain types of cancer. The PDUFA date is April 30, 2024.
FDA Issues Complete Response Letter for Onpattro in Heart Failure Indication
October 9th 2023Alnylam Pharmaceuticals will no longer pursue this indication of Onpattro and will instead on focus on a label expansion for Amvuttra, which is in phase 3 development to treat patients with cardiomyopathy of ATTR amyloidosis.