The CF Foundation has successfully completed a sale of its remaining stake in royalties related to Vertex's CFTR modulators for an upfront payment of $575 million and a potential future payment of $75 million, bringing additional resources to the fight against CF.
Today, the Cystic Fibrosis Foundation announced that it awarded funding to enGene Inc. to develop a customized vehicle to deliver genetic-based therapies, such as gene therapy and gene editing, into the lung cells of people with cystic fibrosis (CF). Delivering genetic-based therapies to the lungs is a key hurdle to developing effective treatments for all people with CF, including individuals with two nonsense and rare mutations.
Roche has acquired a set of potentiator compounds from Enterprise Therapeutics and intends to study them for the treatment of CF. The Cystic Fibrosis Foundation previously provided funding to Enterprise to develop these potential medicines.
Corbus Pharmaceuticals reported today that the Phase 2b clinical trial of its potential anti-inflammatory drug lenabasum did not meet its primary objective of decreasing pulmonary exacerbations in people with cystic fibrosis.
On Aug. 27, leaders from the Cystic Fibrosis Foundation, along with several people from the CF community, participated in a virtual public meeting hosted by the Institute for Clinical and Economic Review to provide insights and expertise on the value of CFTR modulators.
The U.S. Food and Drug Administration has approved the use of Kalydeco® (ivacaftor) for infants with cystic fibrosis as young as four months who have certain mutations.
Today, Vertex Pharmaceuticals Inc. announced positive Phase 3 clinical trial data for Trikafta® (elexacaftor/tezacaftor/ivacaftor) in children 6-11 years old with cystic fibrosis.
Vertex Pharmaceuticals Inc. has submitted three supplemental New Drug Applications to the U.S. Food and Drug Administration (FDA) to expand eligibility for Trikafta® (elexacaftor/tezacaftor/ivacaftor), Symdeko® (tezacaftor/ivacaftor and ivacaftor) and Kalydeco® (ivacaftor) to additional rare CFTR mutations.
The Cystic Fibrosis Foundation has arranged for over 10,000 home spirometers to be provided to care centers to support continued access to high quality, comprehensive care during the COVID-19 pandemic.
Thirty-three new projects are being funded as part of the Foundation's $100 million Infection Research Initiative.