Product approvals in regenerative medicine and rare diseases are among the highlights in our recap of recent FDA regulatory decisions.

The FDA on Fri­day is­sued two si­mul­ta­ne­ous cys­tic fi­bro­sis ap­provals for Ver­tex Phar­ma­ceu­ti­cals but in­clud­ed high-lev­el safe­ty warn­ings for both.

Separately, the FDA expanded the indication for Vertex's Trikafta on Friday to include cystic fibrosis patients with additional genetic variants. The FDA initially approved Trikafta in 2019 for ...

The majority of patients have at least one F508del mutation. TRIKAFTA’s mechanism aims to correct the defective CFTR protein, improving salt and water flow in cells and alleviating symptoms. The FDA ...

Vertex Pharmaceuticals (VRTX) announced the U.S. Food and Drug Administration has approved the expanded use of Trikafta for the treatment of people with cystic fibrosis ages 2 and older who have ...

BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) has approved the expanded use of TRIKAFTA ® (elexacaftor ...

Trikafta, a drug approved for use in Canada in 2021 that dramatically improves the functioning of the lungs, has revolutionized treatment and significantly increased the life expectancy of people ...

It operates via a molecular "cut and paste" mechanism driven by special proteins, known as recombinases. The integron system has been researched a lot. Some bacteria gain new resistance very fast ...

The company’s product portfolio includes Trikafta, Symdeko/Symkevi ... of-the-art technology platforms to discover new disease targets, compounds, delivery mechanisms, and treatment modalities. Vertex ...

KYIV, Dec 15 (Reuters) - Ukrainian President Volodymyr Zelenskiy said on Saturday he had instructed his government to set up supply mechanisms to deliver together with international organisations ...