Celgene Corp. moved one step closer to expanding Thalomid's label.

The Warren, N.J.-based company said the FDA accepted for review its supplemental new drug application seeking approval to market the drug for multiple myeloma, an indication for which the product is getting increased use off-label. Thalomid (thalidomide) generated about $224 million in sales during 2003, more than $100 million more than the previous year, and this year Celgene expects it to produce revenue ranging from $280 million to $290 million.

Such sales growth stems from the compound's use for multiple myeloma, which is the second most common blood cancer in the U.S., affecting about 50,000 people.

"For Celgene, to have a label indication for multiple myeloma is certainly a milestone achievement," Brian Gill, Celgene's director of investor and public relations, told BioWorld Today. "We believe that in 2005, there will be some upside opportunity from the commercial side."

The company submitted its application at the end of December, and expects to receive a response from the agency in October.

"Going forward, assuming that there is an approval for Thalomid in multiple myeloma, it certainly gives the immunology [sales] specialists an opportunity to really help oncologists better understand treatment regimens and results," Gill added, noting the use of Celgene's program for prescribers and patients, STEPS (Systems for Thalomid Education and Prescription Safety).

About 14,600 new cases of multiple myeloma are diagnosed each year and about 11,000 Americans are expected to die each year of multiple myeloma. Clearly the chance to further market Thalomid to such patients adds to the drug's positive profile.

The compound, long associated with severe birth defects, has a checkered history as a result. First used in the 1950s in Europe for morning sickness, its harmful side effects became apparent through shortened or absent limbs in children born to mothers who began taking the drug when pregnant.

But Thalomid first received an FDA approval more than five years ago for a condition related to leprosy. More specifically, the product is approved for the acute treatment of cutaneous manifestations of moderate to severe erythema nodosum leprosum and as maintenance therapy for prevention and suppression of cutaneous manifestations of the disease's recurrence.

Though Gill said sales associated with those indications remains nominal, interest in using the drug for multiple myeloma and other cancers remains high. Thalomid has been evaluated in hundreds of investigator-sponsored clinical trials around the world, he added, with various supporting data published in a variety of peer-reviewed publications.

"These are the things that promote the use and uptake of Thalomid in the marketplace," Gill said. "When we go to medical meetings, our people can't believe the feedback we get from investigators who are doing work on thalidomide in some shape or form."

It seems to directly affect multiple myeloma cells by inhibiting their growth and survival, according to findings published in the Nov. 1, 2000, issue of Blood.

He said that for cancer, the drug is used about 77 percent of the time for blood-borne malignancies. About 70 percent of that wedge represents use in treating multiple myeloma, another 5 percent for myelodysplastic syndromes and the remainder in other hematologic cancers. The other 23 percent of Thalomid's use is in treating solid tumors, such as prostate, renal cell, brain, bladder and liver cancers, as well as melanoma and others.

One in five users receives the drug at no cost as part of Celgene's compassionate use program.

"It's a wonderful thing to have Thalomid continuing to do so well, particularly in the oncology community, because its success fuels a very broad and deep pipeline here at Celgene that has a lot of high-potential cancer-fighting drugs," Gill said. "More than 45 percent of our revenues go right back into our R&D programs."

Celgene has a late-stage investigational compound for multiple myeloma in development as well. Called Revimid (CC-5013), the Thalomid analogue is in pivotal trials in multiple myeloma, for which it has been granted fast-track status, and metastatic melanoma.

The company paid about $27 million to gain access to the thalidomide analogue program from Rockville, Md.-based EntreMed Inc. Previously, both parties had begun legal scraps over patent issues surrounding thalidomide, but settled all disagreements through the license agreement. Celgene first licensed thalidomide in 1998, but only for angiogenesis-related uses. (See BioWorld Today, Jan. 3, 2003.)

Celgene's stock (NASDAQ:CELG) gained 21 cents Monday to close at $39.89.