News

The additional funding will support a Phase 2 clinical trial for an inhaled antisense oligonucleotide drug for people with cystic fibrosis who have the splicing mutation 3849+10Kb C-to-T.

The U.S. Food and Drug Administration approved Kalydeco^® (ivacaftor) for infants as young as 1 month who have at least one copy of 97 mutations that have proven to be responsive to Kalydeco.

With this approval, approximately 2,250 children in the U.S. will be eligible for Trikafta® (elexacaftor/tezacaftor/ivacaftor), including more than 900 who will have access to a CFTR modulator for the first time. The Cystic Fibrosis Foundation believes it is beneficial to start people with cystic fibrosis on modulators as early as possible to help prevent lung damage and the onset of complications.

Orkambi^® is now approved for use in children with cystic fibrosis ages 1-2 years who have two F508del mutations. This is the only modulator available to very young children with these mutations.

The Cystic Fibrosis Foundation is awarding up to $15.9 million in additional funding to Eloxx Pharmaceuticals Inc. to expand clinical studies of ELX-02, a potential therapy for people with CF who have nonsense mutations.

The Cystic Fibrosis Foundation awarded up to $3.5 million to Arrevus Inc. to test a potential treatment for pulmonary exacerbations in people with cystic fibrosis in a late phase clinical trial.

The Cystic Fibrosis Foundation awarded up to $4.7 million to EnBiotix Inc. to study the potential use of inhaled colistin as an additional option to treat Pseudomonas infections in people with cystic fibrosis who are not responding to current treatments.

Study reaffirms the Foundation’s commitment to advance solutions to the growing challenge of antibiotic resistance

Today, the Cystic Fibrosis Foundation announced a new award of up to $2 million to Eloxx Pharmaceuticals Inc. to support the global Phase 2 clinical program of ELX-02, a potential therapy to treat people with cystic fibrosis who have nonsense mutations.

The U.S. Food and Drug Administration has approved the use of Trikafta® (elexacaftor/tezacaftor/ivacaftor) for children ages 6 through 11 who have at least one copy of the F508del mutation or certain mutations in the CFTR gene that are responsive based on lab data.