The U.S. Food and Drug Administration (FDA) is reviewing applications from Vertex Pharmaceuticals to expand the approval for three of the company’s cystic fibrosis (CF) therapies: Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko (tezacaftor/ivacaftor and ivacaftor), and Kalydeco (ivacaftor). The three novel supplemental new drug applications…
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ContraFect will receive funding from the Cystic Fibrosis Foundation to conduct preclinical research about the potential of direct lytic agents, an alternative to conventional antibiotics, to treat lung infections caused by Gram negative antibiotic-resistant bacteria, including Pseudomonas aeruginosa. While antibiotics have had a profound impact on…
People with cystic fibrosis (CF) in the U.K. have grown more numerous, older, and more diverse, according to the U.K. Cystic Fibrosis Registry’s annual data report for 2019. The report, meant to present a snapshot of both the gains as well as the challenges still faced by…
If Trikafta — Vertex Pharmaceuticals’ next-generation triple combination cystic fibrosis (CF) therapy — were to be available in Canada next year, the median age of CF patients could rise by 9.2 years and the number of deaths drop by 15% over the next 10 years, a forecast study reported.
Proteostasis Therapeutics (PTI) has entered into a definitive merger agreement with Yumanity Therapeutics to leverage their common expertise about protein misfolding to advance the development of disease-modifying therapies for several disorders. The merger should be complete before the end of the year. The combined company will operate under the…
The Cystic Fibrosis Foundation (CFF) has awarded nearly $15 million to 33 organizations that are researching how to improve outcomes for cystic fibrosis (CF) patients who are battling infections. The awards fall under the CFF’s Infection Research Initiative, a large-scale effort to provide $100 million in funding…
Poorer oxygen uptake and transport may underlie skeletal muscle differences that affect exercise tolerance in children and adolescents with cystic fibrosis (CF) relative to healthy peers, a recent study suggests. The study, “Tissue oxygenation in peripheral muscles and functional capacity in cystic fibrosis: A cross‐sectional study,”…
The European Commission (EC) approved Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with Kalydeco (ivacaftor) to treat cystic fibrosis (CF) in people 12 and older who have either two F508del mutations or one F508del mutation and one minimal function mutation in the CFTR gene. The decision follows a positive opinion,…
Note: This story was updated Aug. 25, 2020, to clarify that, according to NuvoAir, the regulatory entity in question is the European Medical Device Directive. The NuvoAir Home platform, NuvoAir’s respiratory support system for patients with lung diseases such as cystic fibrosis (CF) and idiopathic pulmonary fibrosis (IPF),…
The U.S. Food and Drug Administration (FDA) has granted the designation of orphan drug to KB407, Krystal Biotech’s potential inhaled gene therapy for the treatment of cystic fibrosis (CF). “We are pleased to receive Orphan Drug Designation for KB407 to treat cystic fibrosis as…
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