Economics
FDA Panel Backs Vertex Cystic-Fibrosis Drug Orkambi
U.S. approval of Orkambi is seen by analysts as essential to Vertex’s future success
Analysts view approval of Orkambi as essential to Vertex’s success. PHOTO: GREGORY BULL/ASSOCIATED PRESS
Vertex Pharmaceuticals Inc.’s experimental cystic-fibrosis treatment Orkambi won the backing of a Food and Drug Administration advisory committee, which voted 12-1 on Tuesday to recommend approval of the drug.
The advisory committee included 13 independent doctors, scientists and consumer and patient representatives. The FDA, which is expected to make a final decision on whether to approve Orkambi by July 5, isn’t required to follow the recommendations of such committees, but it usually does.
Analysts view U.S. approval of Orkambi as essential to Vertex’s success in the coming years. The Boston-based company was founded in 1989, and had accumulated losses of $4.9 billion as of March 31. If approved, Orkambi could reach global sales of $1.6 billion next year and $4.2 billion in 2020, according to J.P. Morgan Chase.
Vertex, whose shares were halted during the regular trading session Tuesday, has a market value of more than $30 billion.
In clinical studies, the lung function of patients taking Orkambi improved by a range of 2.6 percentage points to 3 percentage points, compared with that of patients receiving placebo. FDA officials called the drug’s effect “modest,” but said there was a statistically significant improvement compared with the lung-function declines experienced by patients receiving placebos.
Vertex officials, along with nearly 20 patients and doctors, told the advisory committee that Orkambi was a meaningful advance in treating cystic fibrosis, a progressive lung disease that often kills patients in their 20s. Jeff Masters, a cystic-fibrosis patient in his 30s, said the drug had helped him gain weight, reduced his dependence on other medications and improved his quality of life.
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Some committee members who voted to recommend approval said they were concerned that the drug’s benefit was relatively small on average, and that some patients actually experienced worsened lung function while taking the drug. Still, a majority of advisers concluded Orkambi’s potential benefits in treating cystic fibrosis outweighed its risks. “This was a difficult vote for me,” said Elaine H. Morrato, an associate professor at the Colorado School of Public Health, who voted to recommend approval.
The advisory committee voted unanimously that the drug was safe. In clinical studies, a higher percentage of patients receiving Orkambi discontinued treatment or experienced serious liver-related adverse events than patients taking placebos, the FDA said. “The risks are small, or modest, and are all manageable with appropriate monitoring,” said Robert G. Castile, a committee member and a physician at Nationwide Children’s Hospital in Columbus, Ohio.
The “positive recommendation brings the cystic-fibrosis community one step closer to potential approval of the first medicine to treat the underlying cause of this disease for many more people,” said Vertex Chief Medical OfficerJeffrey Chodakewitz, in a statement.
If approved, Orkambi could treat as many as 8,500 patients in the U.S., or 28% of the 30,000 people in the U.S. with cystic fibrosis. Vertex currently has one approved drug, called Kalydeco, which treats a different genetic type of cystic fibrosis and treats roughly 6% of the nation’s cystic fibrosis patients. Kalydeco has an annual wholesale price of $311,000 per patient in the U.S.
Vertex hasn’t said what it will charge for Orkambi, but some pharmacy-benefit managers have voiced concerns that the cost will overwhelm the budgets of health insurers and self-insured employers.
Orkambi combines Kalydeco, also known as ivacaftor, with another compound called lumacaftor, and is designed to treat patients ages 12 and older who have two copies of the F508del genetic mutation.
FDA officials at the meeting, held in Gaithersburg, Md., questioned whether Orkambi was superior to Kalydeco alone. Orkambi’s effect was similar to the effect of ivacaftor alone in earlier studies, raising the question of whether lumacaftor added any benefit, they said.
Some advisory committee members quizzed Vertex officials early in the day on lumacaftor’s effect, but they ultimately decided the issue was secondary to Orkambi’s overall effect in clinical studies.
The advisory committee’s vote was inconclusive on whether lumacaftor contributed positively to Orkambi’s effectiveness. Three advisers voted yes, four voted no, and six said they couldn’t determine an answer.
Write to Joseph Walker at joseph.walker@wsj.com
