(This story was updated at 5:40 p.m. ET.)
Federal regulators on Thursday approved a new combination drug treatment for the most common form of cystic fibrosis. But it comes at a steep price — more than $250,000 for a year's treatment.
The Food and Drug Administration granted approval for the sale of Cambridge, Mass.-based Vertex Pharmaceutical's drug Orkambi for use in patients ages 12 and older.
The agency's decision follows recommendations made by an advisory panel in May that voted 12-1 in favor of the drug.
The new treatment is a therapy that combines the already approved medication for cystic fibrosis, Kalydeco, with a newly developed medication called lumacaftor to treat the most common genetic mutation which causes cystic fibrosis that affects roughly 8,500 of the estimated 30,000 patients with CF in the U.S. CF is a life-threatening genetic condition that causes cells to secrete fluids that becomes thick and can block passageways in the lungs and pancreas.
“Today is a remarkable day for science, medicine and the CF community," said Dr. Jeffrey Leiden, CEO of Vertex in a statement released Thursday. “Today, the approval of Orkambi represents a fundamental change in the treatment of the most common form of CF, marking significant progress for us and for the entire CF community.”
The company has priced the new treatment at more than $250,000 a year, which will undoubtedly limit its access to some patients. The cost is less than the $300,000 a year price per patient for Kalydeco, which treats a pool of around 2,000 patients for a rarer form of CF.
Dr. Robert Giusti of New York University's Langone Medical Center noted that half of all U.S. cystic fibrosis patients have the form targeted by Orkambi, which occurs when a child inherits two copies of a certain genetic mutation — one from each parent. He expects the FDA will eventually expand the drug's approval to patients as young as 6, increasing the number of people who could benefit.
"This is really exciting because this is a disease that causes a 1 to 2 % deterioration each year in lung function of patients," said Giusti, who directs the center's cystic fibrosis program. "Now, they have a therapy available to potentially reverse that effect."
But questions over Orkambi's effectiveness were apparent even when the advisory committee recommended its approval. Investigators concluded in a committee report that a clinical trial involving more than 1,000 patients revealed the drug therapy resulted in a modest improvement in lung function. The approval will mark a financial turnaround for Vertex, which had posted a net loss of nearly $200 million for the first quarter of 2015. Analysts have estimated sales of Orkambi could reach as high as $8 billion worldwide.
In response to the FDA approval of Orkambi, Robert Beall, president of the Cystic Fibrosis Foundation praised the agency's decision, adding that it was latest example of research that was accomplished with the help of the organization's support.
Vertex's cystic fibrosis drugs grew out of a long-term partnership with the Cystic Fibrosis Foundation. Vertex Pharmaceuticals Incorporated received roughly $120 million in research and funding from the foundation, culminating in the 2012 approval of Kalydeco. The twice-a-day pill had sales of $464 million last year, according to the Cambridge, Massachusetts company.
“We applaud the FDA for its swift approval of Orkambi,” Beall said in a statement. “It is our hope that everyone who is prescribed this drug will have immediate access to it so they can begin to live longer, healthier lives.”
Access to drugs has been a concern because of skyrocketing prices. Earlier this week, a report showed that states like California are paying billions of dollars for a select few medications.
“No budget – public or private – can sustain drugmakers' exorbitant prices, and this latest report is case in point,” John Rother, President of the National Coalition on Health Care and leader of the Campaign for Sustainable Rx Pricing said in a statement regarding that report. “Improving patient access to these lifesaving medications will require pharmaceutical companies to confront that reality and work with all stakeholders to advance a sustainable pricing solution,” he said.