Vertex Pharmaceuticals Inc. won US regulatory approval Thursday for a medicine that eventually could treat roughly half of the estimated 30,000 Americans who suffer from cystic fibrosis, the life-threatening lung disease.
Pricing the two-drug therapy called Orkambi at $259,000 per patient annually, the company said it plans to begin shipping the treatment to specialty pharmacies within days.
“This is a big landmark for the company and also for CF patients,” said Phil Nadeau, biotechnology analyst for investment bank Cowen & Co. “Scientifically, this is a breakthrough people didn’t think possible a few years ago.”
Orkambi is expected to generate billions in annual sales and help Vertex become consistently profitable. The company, which moved from Cambridge to a new $800 million campus on Boston’s Fan Pier last year, has become one of the state’s largest biotechs, with a market value of more than $30 billion — despite having lost money in all but one of its 26 years.
The new drug regimen is expected to help provide more than $53 million for a dozen Vertex senior executives who were granted one-time bonuses that would be paid at the end of 2017 at the earliest if the company is profitable for the prior four quarters.
Those so-called retention awards, which Vertex said were meant to keep key leaders at the company at a critical time, have been criticized by consumer activists who say they give the company’s executives an incentive to charge a high price for the new cystic fibrosis treatment.
Cystic fibrosis patients hailed the approval of Orkambi by the Food and Drug Administration.
“I’m overwhelmed with joy,” said Jeff Masters, 37, a medical sales representative from Ann Arbor, Mich., who has taken Orkambi in a clinical trial for two years and said that it improves his breathing and stamina. “The impact of this drug on quality of life is very real.”
Patients with cystic fibrosis, a genetic disease that causes severe damage to the lungs and digestive system, have had few treatment alternatives. Drugs currently on the market alleviate symptoms, such as lung infections and the buildup of mucus, but not the underlying genetic cause of the disease.
Orkambi, while not curing patients, counteracts damage caused by the disease, enabling patients to breath more easily and stay out of the hospital.
“I’m over-the-moon excited,” said Martha Marshall, a resident of York, Maine, whose daughter Kate, a 16-year-old high school soccer and lacrosse player, hopes to begin taking Orkambi this summer. “I started crying when I heard the news.”
Vertex is developing a portfolio of drugs to target specific genetic defects in different patients. But its medicines have drawn criticism for their high costs.
Vertex executives say the prices are necessary because of the small populations they serve and the need to invest in other medicines.
Vertex’s sole drug on the market now, Kalydeco, treats only about 2,000 cystic fibrosis patients, at a cost of more than $300,000 a year per patient.
Orkambi treats a larger set of patients. Its price will be about 16 percent less than the amount Vertex has charged for Kalydeco.
That’s still exorbitant, said John Rother, president of the National Coalition on Health Care, which represents 85 consumer groups, health care providers, and insurers.
“The dollar level here seems out of line and far beyond what’s needed,” Rother said. “Let’s hope this doesn’t establish a new norm for other drugs and other drug companies — because then we’re in trouble as a nation.”
Specialty drug prices are often related to the size of the patient population. Some other drugs treating “ultra-rare” diseases cost more than $300,000 per year and, in one case, as much as $500,000 annually.
Orkambi was initially approved for more than 8,500 US patients age 12 and over, but investors have bought up Vertex shares, betting that market can be rapidly expanded.
The company is projected to become profitable next year and continue to grow through the rest of the decade, said Nadeau, the Cowen & Co. analyst.
Shares of Vertex, which suspended trading just before noon Thursday as the FDA disclosed its decision, finished the day up 4 percent to $131.26. Vertex’s stock has climbed 34 percent in the past year, and more than 300 percent over the past five years.
FDA officials said the approval broadens the availability of targeted treatments for cystic fibrosis. Orkambi represents an advance because it is the first therapy that specifically targets an important genetic mutation, said an FDA spokesman, Eric Pahon.
The agency had given Orkambi an expedited review and issued a decision within six months because the drug promised significant improvement over existing therapies.
In its statement, the FDA acknowledged that Orkambi can cause side effects, such as shortness of breath, upper respiratory tract infection, nausea, diarrhea, and increased menstrual bleeding in women. Those warnings will be included on the drug’s label.
Patients will get access to the medicine once their insurers approve reimbursement.
Between 35 and 40 percent of eligible patients are insured by Medicaid, the government health plan for low-income residents, while most of the rest are privately insured, Vertex executives said.
Vertex said it will launch a patient support program to provide co-pay subsidies to some privately insured patients and make Orkambi available free to qualified uninsured patients.
Jeffrey M. Leiden, Vertex’s chief executive, noted that many Americans with cystic fibrosis still lack medicines to treat the underlying cause of their diseases.
“We’re very committed to developing future medicines for the tens of thousands of people who don’t have a medicine,” he said in a conference call with industry analysts.
Vertex is testing Orkambi — it’s a treatment that combines Kalydeco with another drug called Lumacaftor in a single pill — on other patient groups in the United States and abroad with the most common form of cystic fibrosis.
It’s estimated that about 70,000 people worldwide suffer from the disease.
The Cystic Fibrosis Foundation had helped fund the research that led to Orkambi and last year sold its royalty rights to Royalty Pharma for $3.3 billion.
“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,” foundation president Robert J. Beall said in a prepared statement.