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.
Cambridge, Massachusetts-based Yumanity Therapeutics and Boston-based Proteostasis Therapeutics announced plans to merge. The combined company will operate after the deal as Yumanity Therapeutics, and will focus on protein misfolding and neurodegenerative diseases.
Chicago-based AbbVie announced plans for a strategic collaboration to develop a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator compound licensed from the Cystic Fibrosis Foundation.
The U.S. FDA approved Symdeko (tezacaftor/ivacaftor and ivacaftor) for use in children with cystic fibrosis ages 6 through 11 years who have two copies of the F508del-CFTR mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator gene that is responsive to the Vertex Pharmaceuticals Inc. medicine.
The FDA approved Vertex Pharmaceuticals’ Orkambi (lumacaftor/ivacaftor) to include use in children ages 2-5 years with cystic fibrosis who have two copies of the F508del-CFTR mutation.
Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium – consisting of Imperial College London and the Universities of Oxford and Edinburgh – Imperial Innovations, and Oxford BioMedica announced a global collaboration to develop a first-in-class, long-term therapy for patients with cystic fibrosis.
