News

The European Medicines Agency (EMA) has validated Vertex Pharmaceuticals’ application seeking approval for its triple combination — elexacaftor (VX-445), plus tezacaftor, and ivacaftor (Kalydeco) — for cystic fibrosis (CF) patients who cannot use other approved CF disease-modifying treatments or don’t benefit from them as intended.

The University of Queensland (UQ), in Australia, has launched its Queensland Cystic Fibrosis Research Program with a goal of improving the life of people with this disease. This research program is supported by $15 million donation put together by several sources: the Cystic Fibrosis Foundation in the U.S., and in Australia, UQ…

Alaxia has joined the European consortium iABC to accelerate the development of ALX-009, an investigational inhaled antibiotic for multi-drug resistant lung infections in people with cystic fibrosis (CF). Under the partnership, Alaxia will have support for setting up clinical trials and recruiting patients, and will gain access…

Vertex Pharmaceuticals has reached an agreement with England’s National Health Service (NHS), allowing patients there access to all Vertex cystic fibrosis (CF) treatments currently approved in Europe at low or no out-of-pocket cost. The agreement between the NHS and Vertex appears to end a long-standing dispute over the company’s pricing…

A majority of cystic fibrosis patients who took part in an Instagram poll by Bionews Services said they would be interested in moving from current treatments to Trikafta (elexacaftor, tezacaftor, and ivacaftor), the newly approved and “next-generation” combination therapy by Vertex that is expected to treat 90% of all…

People who carry certain genetic mutations associated with cystic fibrosis, (CF) but who do not have the disease, are at risk for some gastrointestinal problems similar to those reported by CF patients, a large-scale study shows. This finding helps better understand the impact of genetic mutations linked to CF, as…

The Cystic Fibrosis Foundation (CFF) is celebrating the United States Food and Drug Administration’s decision to approve Trikafta. The therapy — a triple combo based on elexacaftor, tezacaftor, and ivacaftor, which is being developed by Vertex Pharmaceuticals — was approved on Oct. 21 for people with cystic fibrosis (CF)…

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion regarding the use of Kalydeco (ivacaftor) in infants ages 6 months to a year with cystic fibrosis (CF). The decision was announced by Vertex Pharmaceuticals, the…

Vertex Pharmaceuticals has taken a major step toward treating 90% of all people with cystic fibrosis (CF), its long-stated goal, with the U.S. Food and Drug Administration’s approval of Trikafta (elexacaftor, tezacaftor, and ivacaftor) as a next-generation, triple combination treatment for CF. This triple combination treats people with the most…

Boehringer Ingelheim announced the enrollment of a first patient in its Phase 2 BALANCE-CF 1 trial assessing the safety and efficacy of BI-1265162 to treat cystic fibrosis (CF) regardless of a person’s disease-causing mutations. BI-1265162 is an epithelial sodium channel (ENaC) inhibitor that works to prevent cells lining…