The patient population that can be helped by the drug is small, though, and the cost is high: about $311,000 per patient, per year. The actual or net price of Orkambi will be lower depending on the discounts and rebates offered to insurance companies.
The Orkambi commercial launch will be closely watched for any reimbursement pushback or protests about the Orkambi price from insurance companies and pharmacy benefit managers.
Shares of Vertex closed yesterday at $131.26, up 4.02 percent.
Revenue from Orkambi could reach $3.42 billion in 2018, analysts estimate.
In a statement after submitting their treatment for approval previous year, Vertex’s executive vice president and chief medical officer Jeffrey Chodakewitz said the drug seeks to treat the mutation that is the “most common form of the disease”.
Officials from the US Food and Drug Administration has approved a new combination drug designed to treat a variation of cystic fibrosis that causes the formation of sticky mucus in the lungs and other organs of the patient, leading to digestive problems, infections and early death. About 70,000 people worldwide suffer from the disease, though only about half of those are thought to have the mutation treated by Orkambi.
The new cystic fibrosis drug is a product of Vertex Pharmaceuticals and is administered to patients who are at least 12 years old.
Orkambi improves lung function and significantly reduces the rate of pulmonary exacerbations, which can lead to frequent hospitalizations and accelerated lung disease. In fact, in the study those who took two of the new drug for Cystic Fibrosis every twelve hours showed having better lung function than those who were taking the placebo.
Orkambi has also been submitted for approval in Europe, and a decision by the EMA is expected before the end of the year. This combination drug has been developed by Vertex Pharmaceuticals Inc. Since then, tremendous progress in understanding and treating CF has led to dramatic improvements in the length and quality of life for those with CF. Many people with the disease can now expect to live into their 30s, 40s and beyond. “We congratulate Vertex for their success in developing new CF medicines and are pleased with their continuing commitment to help all eligible patients get access to these medicines”.
The safety and efficacy of Orkambi was studied in two double-blind, placebo-controlled clinical trials of 1,108 participants with CF who were 12 years and older with the F508del mutation.