EU Approves Vertex’s Next-generation Cystic Fibrosis Drug

(Reuters) -Vertex Pharmaceuticals said on Tuesday its next-generation cystic fibrosis treatment has won European Union approval for a rare and progressive genetic disease, expanding the company’s presence beyond the United States.

The approval builds on the company’s dominance in this market and helps protect against future sales declines once its older cystic fibrosis drug, Trikafta, loses patent protection in 2037.

Pharma firms prepare for patent expiry years ahead, as loss of exclusivity can trigger up to a 90% revenue drop due to generic competition.

The new once-daily triple combination therapy, branded Alyftrek, was approved for patients aged six years and older who have at least one F508del mutation or another CFTR gene mutation responsive to the therapy.

Cystic fibrosis is an inherited disorder resulting from the absence of a specific protein, which disrupts salt and water movement in and out of cells in various organs.

In the lungs, this leads to a buildup of sticky, thick mucus, making breathing difficult and causing progressive lung damage that can result in death.

According to the European Cystic Fibrosis Society Patient Registry (ECFSPR) report, more than 50,000 people in Europe have cystic fibrosis.

In late-stage trials, Alyftrek — a combination of vanzacaftor, tezacaftor and deutivacaftor — proved non-inferior to twice-daily Trikafta.

The approval follows a positive recommendation in April from the European Medicines Agency, the region’s drug regulator.

The European Commission, which typically follows the EMA’s guidance, gives final approval for new drugs.

Alyftrek received U.S. approval last year and generated about $53.9 million in sales during the first quarter.

(Reporting by Christy Santhosh in Bengaluru; Editing by Tasim Zahid)