FDA grants approval for potential ‘blockbuster’ leukemia drug made by Cambridge-based Ariad

Federal regulators Friday approved a drug developed by Ariad Pharmaceuticals Inc. to treat two types of leukemia, giving the Cambridge biotechnology company the green light more than three months ahead of schedule to sell the medicine in the United States.

The once-daily pill, known by its chemical name of ponatinib during years of research and clinical trials, will be marketed under the brand name Iclusig, the company said. It was approved to treat chronic myeloid leukemia and Philadelphia chromosome positive acute lymphoblastic leukemia for patients who can’t tolerate, or have developed resistance to, existing therapies.

“This is our first branded medicine,” Ariad chief executive Harvey J. Berger said Friday. “We will be launching it immediately. It is the first medicine in this area that can be used across the intolerant and resistant population. It’s tremendously important for Ariad. And it’s a breakthrough medicine that will make a difference in the lives of [leukemia] patients.”

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Berger said Ariad would charge $115,000 a year per patient for Iclusig, a premium over the approximately $100,000 charged by rival drug makers for existing treatments. Iclusig acts by blocking mutations that develop with the current class of drugs, called tyrosine kinase inhibitors, made by pharmaceutical giants Novartis AG and Bristol-Myers Squibb Co..

Iclusig is now being taken by newly-diagnosed patients in a clinical trial, and Berger said he hopes it eventually can be approved as a “first-line” therapy—meaning doctors could prescribe it to patients before trying other drugs.

Shares of Ariad, which had nearly doubled this year as the drug candidate moved toward approval, fell $4.93 Friday to close at $18.95, a decline of 20.6 percent on the Nasdaq exchange. Analysts said the stock may have been overbought, but also blamed a Food and Drug Administration-required “black box” warning label for Iclusig alerting doctors and patients that it could cause blood clots and liver toxicity. Such warnings are common for drugs prescribed to severely ill cancer patients.

“Wall Street has a new concern about whether the label and adverse event profile will affect the uptake in healthier patients,” said biopharmaceutical analyst Michael J. Yee, managing director for RBC Capital Markets in San Francisco. “It is a concern that only popped up recently and was not expected, but we think it will be addressed over time as oncologists adopt the drug.”

Ariad has been building a sales force in anticipation of Iclusig’s approval, following a trail blazed by two other Cambridge biotechnology companies that made the transition from research to full-scale commercial companies in the past two years after winning the FDA’s okay to sell their first drugs. The agency signed off on Vertex Pharmaceuticals Inc.’s hepatitis C treatment, while Ironwood Pharmaceuticals Inc. was allowed to sell a drug to treat irritable bowel disease with constipation.

Another Cambridge-based biotechnology company, AVEO Pharmaceuticals Inc., has a kidney- cancer drug application before the FDA and expects a decision by the end of July.

Friday’s Ariad approval came three and a half months before the March 27 date by which federal regulators were scheduled to complete review of the drug application. The fast action underscored the importance of Iclusig to oncologists and the roughly 5,000 Americans diagnosed each year with chronic myeloid leukemia, known as CML, a cancer of the white blood cells that is often fatal. It also works in a smaller number of patients with Philadelphia chromosome positive acute lymphoblastic leukemia. a similar blood and bone marrow cancer.

“It provides a treatment option to patients with CML who are not responding to other drugs,” said Richard Pazdur, director of the Office of Hematology and Oncology Products at the FDA’s Center for Drug Evaluation and Research.

Industry analysts had projected that the leukemia drug, which was granted priority review by the FDA, could become a blockbuster therapy—one that generates more than $1 billion a year in revenue—within five years, but said Friday it was too soon to predict how the warning label could affect sales. Regulators are expected to rule by the middle of next year on Ariad’s application to sell the drug in Europe, where the company has set up a base in Lausanne, Switzerland.

The warning label will alert patients and physicians that the drug, besides causing blood clots and liver toxicity in some cases, can trigger more common side effects such as high blood pressure, rash, abdominal pain, fatigue, headache, dry skin, fever, joint pain, and nausea.