Rare Mutations Archives

FDA Approves Trikafta, Symdeko and Kalydeco for CF Patients With Certain Rare Mutations

Approvals, Blockbusters, Children, Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), Expanded Approval, FDA, Genetic Diseases, in vitro data, New Indications, Rare Mutations, Therapeutics

The U.S. Food and Drug Administration approved Vertex Pharmaceuticals Inc.’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko (tezacaftor/ivacaftor and ivacaftor) and Kalydeco (ivacaftor) for use in people with cystic fibrosis with certain rare mutations.

December 21, 2020/by Andrew Humphreys

Roche gene testing key to $1.7 billion deal for Blueprint cancer drug

Business, Cancer, Deals, Diagnostics, Drug Pricing, Genetic Testing, Non-Small Cell Lung Cancer, Rare Mutations, RET Gene, Therapeutics, Thyroid Cancer, Tumors

Roche struck a $1.7 billion cancer drug pact with Blueprint Medicines, as advances in genetic testing for rare mutations drive lucrative deals for expensive treatments.

July 14, 2020/by Reuters Health