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Hat Trick: Pomalyst Joins Celgene’s Multiple Myeloma Arsenal

By Marie Powers
Staff Writer

In October 2012, the FDA cancelled the Oncologic Drugs Advisory Committee meeting scheduled for Nov. 8, which was to include a review of the new drug application for pomalidomide, Celgene Corp.’s immunomodulatory drug (IMiD) candidate in multiple myeloma (MM). Notification came in the aftermath of Hurricane Sandy, which played havoc not only with lives and property up and down the East Coast but also with business and government schedules, including adcom meetings.

Still, analysts saw the move as a tacit nod to the drug’s approval, and the FDA did not disappoint. On Friday afternoon, the agency approved pomalidomide, branded Pomalyst, two days before its Sunday PDUFA date, for use in combination with dexamethasone in relapsed and refractory MM (RRMM) that has progressed following at least two prior therapies. The drug joins Celgene’s IMiD blockbuster Revlimid (lenalidomide), which is approved in nearly 70 countries in combination with dexamethasone to treat MM patients who have received at least one prior therapy, and Thalomid (thalidomide), approved for MM and erythema nodosum leprosum.

Revlimid also is approved in the U.S., Canada and several other countries for transfusion-dependent anemia due to low- or intermediate-1-risk myelodysplastic syndromes associated with a deletion 5q cytogenetic abnormality.

“Pomalyst is an important option for patients who have exhausted most of the standards of care in myeloma,” Celgene spokesman Greg Geissman told BioWorld Today. The treatment of MM patients has shifted over the past seven years as novel therapies were introduced, improving survival but building resistance in many patients. Pomalyst will directly address this medical need, he said.

Approval was supported by the results of MM-002, a randomized, open-label Phase II study evaluating pomalidomide plus low-dose dexamethasone compared to pomalidomide alone in patients with relapsed multiple myeloma who were refractory to their last myeloma therapy and had received Revlimid and bortezomib (Velcade, Millennium: The Takeda Oncology Co.).

Of the 221 patients evaluable for response, 29.2 percent achieved a partial response or better in the pomalidomide plus low-dose dexamethasone arm compared to 7.4 percent for pomalidomide alone. Median duration of response for patients who received pomalidomide plus low-dose dexamethasone was 7.4 months, while the median was not reached for pomalidomide alone.

At the American Society of Hematology meeting in Atlanta in December, Celgene also reported data from its Phase III MM-003 study, which demonstrated that pomalidomide plus low-dose dexamethasone met the primary endpoint of improvement in progression-free survival (PFS) in patients with RRMM who had been treated with Revlimid and Velcade. At the overall survival (OS) interim analysis, the study crossed the superiority boundary for OS – a key secondary endpoint. Improvements in both PFS and OS were both highly statistically significant and clinically meaningful, and the data safety monitoring board recommended that patients who had not yet progressed in the high-dose dexamethasone arm cross over to pomalidomide.

Approval of Pomalyst comes as Celgene seeks to expand Revlimid into new indications, including newly diagnosed multiple myeloma, non-Hodgkin’s lymphoma and chronic lymphocytic myeloma. Although some analysts have questioned whether Pomalyst could cannibalize Revlimid sales, which approached $3.8 billion last year, Celgene officials view the drug as a growth driver that will buttress the company’s MM arsenal and propel overall sales, especially since revenues from Thalomid have declined steeply since the introduction of Revlimid, accounting for just $302 million last year.

Like Revlimid and Thalomid, Pomalyst – an analogue of thalidomide – will be available in the U.S. only through a restricted distribution program. The company expects to launch the drug in about two weeks, according to Geissman.

See Monday's BioWorld Today for More on This Story.