Federal regulators on Friday approved an Ariad Pharmaceuticals Inc. drug to treat two types of leukemia, giving the Cambridge 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 biotechnology company said. It was approved to treat chronic myeloid leukemia, as well as Philadelphia chromosome positive acute lymphoblastic leukemia, for patients who can’t tolerate or have developed a resistance to existing therapies.
“This is our first branded medicine,” chief executive Harvey J. Berger said. “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 patients.”
Ariad will charge $115,000 a year per patient for Iclusig, a premium over the approximately $100,000 charged by rival drug makers for existing treatments, Berger said.
Iclusig acts by blocking mutations that develop with the current class of drugs, called tyrosine kinase inhibitors, from Novartis AG and Bristol-Myers Squibb Co.
Iclusig is 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 that doctors could prescribe it before trying other drugs.
Shares of Ariad, which had nearly doubled in value this year as the drug candidate moved toward approval, fell $4.93 Friday to close at $18.95, a decline of 20.6 percent.
Analysts said the stock may have been overbought, but also blamed a Food and Drug Administration-required “black box” warning label for Iclusig. It will alert doctors and patients that the drug 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 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 for Iclusig, 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. 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.
AVEO Pharmaceuticals Inc., also of Cambridge, has a kidney- cancer drug application before the FDA and expects a decision by the end of July.
Ariad’s approval came 3½ months before March 27, when regulators were scheduled to complete their review. The fast action underscored the importance of Iclusig to oncologists and the roughly 5,000 Americans diagnosed each year with chronic myeloid leukemia, or CML, a cancer of 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 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.
Analysts had projected that the drug could become a blockbuster therapy — one that generates more than $1 billion a year in revenue — within five years. But on Friday they said it was too soon to predict how the warning label could affect sales.
Regulators are expected to rule by mid-2013 on Ariad’s application to sell the drug in Europe. The company has set up a base in Lausanne, Switzerland.
The warning label will also alert patients the drug can trigger common side effects such as high blood pressure, rash, abdominal pain, fatigue, headache, dry skin, fever, joint pain, and nausea.Robert Weisman can be reached at firstname.lastname@example.org.