UPDATED: FDA OKs Ariad's blockbuster contender ponatinib for leukemia

Ariad's plan to launch its maiden commercialization effort took hard shape today as the FDA announced that it had approved ponatinib for two types of leukemia. Tapped as a likely blockbuster by a number of analysts, Ariad ($ARIA) gained regulatory approval for the targeted treatment a full three months ahead of its regulatory deadline. 

Ariad will market the drug as Iclusig, now approved for patients with chronic myeloid leukemia (CML) who have stopped responding to other drugs and for patients with Philadelphia chromosome positive acute lymphoblastic leukemia (ALL). Iclusig targets CML cells that have a particular mutation, known as T315I, that makes these cells resistant to currently approved tyrosine kinase inhibitors. 

In a phone call with analysts Friday afternoon, Ariad CEO Harvey Berger said that the drug would be sold at a wholesale acquisition cost of $9,580 a month, or $115,000 a year, with free supplies for the uninsured and "competitive" co-pay support for the insured. But it was the news that the FDA had decided to put a "black box" warning about potential adverse events on the label that attracted the most attention during the conference call with company executives. Clearly taken by surprise, despite assurances that the warning should have no major market consequences, spooked investors drove company shares down 20% on what should have been a red-letter day for the company.

Ponatinib generated impressive results in a pivotal study, which showed that more than half of patients with chronic myeloid leukemia who failed prior treatment had a major cytogenetic response on the experimental BCR-ABL inhibitor. This is important because patients with the disease build resistance to current therapies such as Novartis' ($NVS) blockbuster Gleevec, and patients with resistant disease eventually run low on treatment options. 

"The approval of Iclusig is important because it provides a treatment option to patients with CML who are not responding to other drugs, particularly those with the T315I mutation who have had few therapeutic options," said Richard Pazdur, director of the Office of Hematology and Oncology Products in FDA's Center for Drug Evaluation and Research. "Iclusig is the third drug approved to treat CML and the second drug approved to treat ALL this year, demonstrating FDA's commitment to approving safe and effective drugs for patients with rare diseases."

Berger has been supremely confident of an approval, laying the foundation to commercialize the drug in the U.S. and Europe, where it has also been fast-tracked. Berger has told analysts that he expects the drug can garner $800 million in annual revenue on the initial approval, with $1.5 billion in projected revenue if the company can broaden the label to include its use as an initial treatment for leukemia. A head-to-head study with Gleevec is currently under way.

"Within less than five years, we were able to bring Iclusig from the start of clinical development to U.S. approval, achieving a major milestone in Ariad's history," Berger said in a release. "We have now transformed Ariad into a commercial oncology company addressing major unmet medical needs for cancer patients."

- here's the FDA press release
- read the company release

Special Report: Ponatinib – Top 10 Late-Stage Cancer Drugs – 2012

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