• The European Medicines Agency's CHMP has recommended approval for Alnylam's Onpattro (patisiran), a groundbreaking siRNA therapy for hereditary transthyretin-mediated amyloidosis with polyneuropathy.
• Vertex's Symkevi (tezacaftor/ivacaftor), in combination with Kalydeco, received positive CHMP opinion for treating cystic fibrosis patients with specific F508del mutations.
• The committee also endorsed several other medications, including Pierre Fabre's melanoma drugs and AstraZeneca's Imfinzi for lung cancer, while rejecting BMS's Opdivo-Yervoy combination for first-line kidney cancer.