June 26, 2020 / 11:58 AM / 9 days ago

Vertex treatment for rare genetic disorder wins EU watchdog backing

(Reuters) - A panel of the European health regulator on Friday recommended approving Vertex Pharmaceuticals’ triple-combination treatment for cystic fibrosis (CF), a rare, deadly genetic disorder that causes the build-up of thick mucus in some body parts. The European Medicines Agency (EMA) said its human medicines committee (CHMP) recommended the therapy for patients who are 12 years of age or older and have either of the two types of mutations in the CF transmembrane regulator gene (CFTR), which make up the majority of CF cases.

The European backing comes after U.S. authorities approved a cocktail of elexacaftor, tezacaftor and ivacaftor in October last year as “Trikafta”. In Europe, the drug is expected to be sold under the brand name “Kaftrio”.

CF is caused by a mutation of the CFTR gene, which regulates salt and water content in cells. A protein defect leads to mucus congestion in the lungs and the digestive tract.

Kaftrio has been recommended for two types of CFTR mutations: one where patients have inherited an identical gene with a defective F508del protein from each parent, and another where patients have inherited different forms of genes from each parent.(bit.ly/3dBB7oj)

Vertex’s treatment targets the protein and helps improve the CFTR function. There is no cure for CF and doctors try different regimens aimed at controlling symptoms and preventing or reducing complications.

CF affects around 42,000 people in the European Union (EU), the EMA said, adding that many patients have mutations that make them ineligible for treatments that are currently available.

Final approvals are up to the European Commission, which typically seconds CHMP recommendations. Once formally approved, Kaftrio can be sold in the EU.

Reporting by Pushkala Aripaka in Bengaluru, Editing by Sherry Jacob-Phillips and Aditya Soni

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