Vertex to file for U.S. approval of CF therapy in 2019
Vertex to file for approval of cystic fibrosis therapy in 2019
May 30 (Reuters) – Vertex Pharmaceuticals Inc said on Thursday it will apply for regulatory approval later this year for its triple combination of drugs to treat life-shortening genetic condition cystic fibrosis.
The company said it would push forward its drug VX-445, in combination with its backbone double drugs tezacaftor and ivacaftor, together named Symdeko, based on data from patients aged 12 and above with a type of cystic fibrosis.
Late-stage studies have shown the cocktail of drugs led to significant lung function improvement in patients.
Cystic fibrosis, which affects about 75,000 people in North America, Europe and Australia, is caused by a defective or missing protein and results in serious lung infections, declining lung function and often leading to early death.
The combination therapy has the potential to address 90% of people with the disease and analysts have said its success would effectively guarantee multi-billion dollar sales for the company.
Vertex said it plans to file for U.S. approval in the third quarter and for European approval in the fourth.
The company had earlier tested another experimental drug, VX-659, with the two-drug combination Symdeko, but delayed filing for U.S. approval to decide which combination to push forward.
Vertex was the first company to market drugs that address the underlying cause of the disease, rather than just symptoms.
(Reporting by Tamara Mathias in Bengaluru; Editing by Shinjini Ganguli)