(Adds treatment price, background)
By Manojna Maddipatla and Carl O'Donnell
Oct 21 (Reuters) - Vertex Pharmaceuticals Inc has priced its three-drug combination for cystic fibrosis (CF) at $311,503 per year, after winning early U.S. approval on Monday.
Shares of the drugmaker, which also raised its 2019 sales forecast for CF products, were up 3% in after-hours trading.
The U.S. Food and Drug Administration approval, which comes five months ahead of the agency's previously announced action date, greatly expands the percentage of cystic fibrosis patients that Vertex can treat.
The drugmaker boosted its guidance range for CF products sales to between $3.70 billion and $3.75 billion, from $3.60 billion to $3.70 billion.
The treatment, Trikafta, is a combination of three drugs that target a defective protein responsible for the life-threatening disease that causes the build up of thick mucus in body parts such as the lungs and digestive tract. (https://bit.ly/2MxqCrJ)
Trikafta is the first approved treatment that is effective for patients 12 years and older, who have a genetic mutation which affects 90% of CF patients or roughly 27,000 people in the United States, the agency said.
The backbone drugs in Trikafta are ivacaftor and tezacaftor, which make up Vertex's previously approved CF drug Symdeko. Ivacaftor is also sold as a standalone treatment, Kalydeco, and is part of another double combination CF product called Orkambi.
Trikafta improved lung function by around 14% in clinical trials, compared with a roughly 3% improvement in patients treated with Orkambi.
Orkambi has a list price of around $272,000 per year, while Symdeko lists at around $292,000.
Analysts expect the early approval to accelerate the launch date for Trikafta, which they expect will make $630 million in 2020.
However, the approval comes at a time when the drugmaker has been receiving push back from healthcare payers for the price of its existing CF therapies.
England's government-run healthcare system has been holding out on agreeing to pay for Vertex's Orkambi on the grounds that the price that Vertex is demanding from the country is too high. The rebates that Vertex offered in the negotiations have not been publicly disclosed.
CF is a chronic, progressive, and frequently fatal genetic disease, primarily affecting the respiratory and digestive systems in children and young adults. (Reporting by Carl O'Donnell in New York and Manojna Maddipatla in Bengaluru; Editing by Shailesh Kuber)