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.
Boston-based Proteostasis Therapeutics announced positive preliminary data from the ongoing Phase I clinical trial of PTI-808 and PTI-801 in cystic fibrosis. Despite being an early-stage trial and preliminary data, company shares exploded upward by 301 percent, with more than 44 million shares trading hands.
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.
Vertex Pharmaceuticals reportedly terminated a long-acting trial combining a recently acquired therapy with the company’s cystic fibrosis drug Kalydeco.
