Vertex to Seek Expanded FDA Approval for Trikafta After Positive Child Safety Data
Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is safe for use in children with cystic fibrosis (CF) as young as age 6, data...
Study Finds Funding Disparities Between Sickle Cell Disease, Cystic Fibrosis
Disparities in funding exist between sickle cell disease (SCD) and cystic fibrosis (CF), according to a new study published in JAMA...
This new treatment helps 90% of cystic fibrosis patients breathe better
Vertex’s Trikafta was the second-fastest FDA approval in history, and could help some 27,000 patients. In October 2019, Vertex...
FDA Grants Fast Track Status to Translate Bio’s RNA-Based Therapy MRT5005
The U.S. Food and Drug Administration (FDA) has granted fast track designation to MRT5005, an investigational RNA-based treatment for...
Raleigh biotech startup Arrevus gets FDA support for its drug treating cystic fibrosis patients
RALEIGH — Cystic fibrosis (CF) is a heredity disease with no cure. However, Raleigh biotech startup Arrevus believes it owns a drug that...
5 FDA approval decisions to watch in the 1st quarter
Credit: Yujin Kim / MedTech Dive, original photo courtesy of U.S. Food and Drug Administration The Food and Drug Administration cleared...
5 New Drug Approvals the FDA Gave Top Priority
The U.S. Food and Drug Administration's made it clear that granting a priority review to an application isn't exactly the same thing as...
CF patients experience improved lung health with lumacaftor-ivacaftor but with caveats
In adolescent and adult patients with cystic fibrosis taking lumacaftor-ivacaftor (ORKAMBI), the combination drug appears to improve lung...
Translate Bio Prioritizes Development of CF Therapy MRT5005, Other Lung Disease Candidates
Translate Bio is going to focus on the development of potential therapies to treat pulmonary diseases, including its lead candidate,...
FDA to Review Elexacaftor, Tezacaftor, Ivacaftor Combo for Cystic Fibrosis
The Food and Drug Administration (FDA) has accepted for priority review the New Drug Application (NDA) for elexacaftor (VX-445),...
