(Reuters) - Vertex Pharmaceuticals Inc’s unique combination therapy for a rare lung disorder was given the green light by U.S. health regulators, extending the company’s reach to the most common form of genetic mutation responsible for cystic fibrosis.
Orkambi, a combination of Vertex's approved drug Kalydeco and a new compound called lumacaftor, is intended for patients aged 12 and above, the U.S. Food and Drug Administration said on Thursday. (1.usa.gov/1KwebV3)
Orkambi and Kalydeco aim at fixing the underlying condition caused by the misbehaving gene, unlike current drugs which treat related symptoms.
The combination treats the most common F508del mutation, while Kalydeco alone treats cystic fibrosis patients with a different mutation.
Orkambi will carry a wholesale price tag of $259,000 per year in the United States, the company said in a regulatory filing. (1.usa.gov/1HzWyon)
Most of the analysts had expected Orkambi to be priced lower than Kalydeco, which costs about $300,000 per year.
Analyst’s view on the lower pricing reflects the immediate need for a cure to the larger population at an affordable rate.
Cystic fibrosis is caused by a defective gene that disrupts the function of the lungs and digestive system, producing a build-up of thick, sticky mucus leading to inflammation and recurrent bacterial infections. Most patients die in their mid-20s.
William Blair & Co LLC’s Katherine Xu said the “disease modifying” Orkambi is expected to generate peak annual sales of about $5.9 billion worldwide.
Kalydeco sales rose 25 percent to $463.8 million in 2014.
Orkambi is intended to benefit nearly half of the 30,000 Americans affected with cystic fibrosis. Worldwide, about 70,000 people are estimated to have been affected by the disorder.
MorningStar Inc’s Stefan Quenneville said he expects the drug to win European approval by the end of the year.
Vertex Pharma’s shares were trading up 2.2 percent at $128.85 on the Nasdaq on Thursday.
Editing by Savio D'Souza and Don Sebastian