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FDA approves Celgene drug for multiple myeloma

By Toni Clarke

(Reuters) - U.S. regulators have approved a new drug from Celgene Corp for patients with multiple myeloma whose disease has worsened after being treated with other cancer drugs.

The U.S. Food and Drug Administration said on Friday it approved the drug, Pomalyst, also known as pomalidomide, for the most difficult-to-treat patients.

Celgene, in an emailed statement, said the drug was priced at about $10,500 per cycle, and clinical trials have shown that the typical course of treatment is about five cycles.

The company filed its application based on a mid-stage, or Phase II, trial in which patients either took pomalidomide, plus a low dose of the standard treatment dexamethasone, or pomalidomide alone.

Of 191 patients evaluated, 34 percent treated with the two-drug combination responded to treatment, compared with 13 percent of patients treated with pomalidomide alone. The analysis of whether patients in the treatment group lived longer has not yet been completed.

Data from the trial, known as MM-002, showed that median progression-free survival, or the length of time patients lived before relapsing, was 4.7 months in the pomalidomide plus low-dose dexamethasone arm, versus 2.7 months in the pomalidomide-alone arm.

The FDA will require the Pomalyst label to carry a "boxed warning" alerting patients and healthcare professionals that the drug should not be given to pregnant women because it can cause life-threatening birth defects, and that it can cause blood clots.

Celgene is also required to institute a risk management program for Pomalyst, which is designed to work by enlisting the body's immune system to attack cancer cells.

The new drug comes from the same family as Celgene's existing drug Revlimid but appears to help patients who have become resistant to current therapies, including Revlimid and its predecessor drug Thalomid.

Multiple myeloma is a cancer that starts in plasma cells in bone marrow and ultimately disrupts the production of normal blood cells. The American Cancer Society estimates there will be about 22,300 new cases diagnosed in 2013 and 10,700 deaths.

A variety of new drugs have entered the market during the last few years, often allowing patients to live years longer. Last July, for example, the FDA approved Kyprolis, a drug made by Onyx Pharmaceuticals Inc for patients with multiple myeloma who had received at least two prior therapies.

"Pomalyst marks the second treatment to receive accelerated approval in the last seven months, which is unparalleled in any other cancer," the Multiple Myeloma Research Foundation said in a statement.

In addition to MM-002, Celgene conducted a late-stage trial known as MM-003 in Europe that tested pomalidomide plus a low dose of dexamethasone versus a high dose of dexamethasone alone. Results of that trial have been submitted to European regulators and the company expects to hear whether the drug has been approved there later this year. They were not included in the package submitted for FDA approval.

Data from the European trial showed patients who had failed an average of five previous therapies who took pomalidomide and low-dose dexamethasone survived significantly longer than those who took high-dose dexamethasone.

The median overall survival when the data was reported in November had not yet been reached in the pomalidomide group, while the median overall survival in the control arm was 34 weeks. The results show that no matter what occurs during the remainder of the trial, the improvement in overall survival will be highly statistically significant.

Geoff Meacham, an analyst at J.P. Morgan, has estimated pomalidomide could generate sales of around $450 million by 2015. Celgene's chief executive has said he expects the drug to ultimately generate sale of more than $1 billion.

(Reporting By Toni Clarke. Additional reporting by Deena Beasley; Editing by Tim Dobbyn and M.D. Golan)

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