Celgene Corp. expects to launch its oral immunomodulatory drug, Revlimid, early next month after the product won FDA approval for treating patients with transfusion-dependent anemia due to low- or intermediate-1-risk myelodysplastic syndromes (MDS) associated with a deletion 5q chromosomal abnormality.

"This is really a transforming moment for these patients," said Brian Gill, Celgene's director or public relations, adding that they are "so sick that the only supportive care involves blood transfusions from week to week."

MDS occurs when blood cells don't develop into mature cells, resulting in symptoms such as anemia and fatigue that can develop into life-threatening complications.

"Revlimid doesn't treat the symptoms, but actually alters the disease state," he said. "It has shown unprecedented and very compelling clinical data from a Phase II study."

It was so compelling that Celgene took a risk and submitted a new drug application based on the Phase II results. That strategy seemed validated in September when the FDA's Oncologic Drugs Advisory Committee voted 10-5 in favor of approving the drug. (See BioWorld Today, Sept. 15, 2005.)

In the 148-patient Phase II study, Revlimid (lenalidomide) demonstrated efficacy in patients missing a certain part of the 5q chromosome. Two-thirds of that patient population went into complete remission, meaning they no longer required blood transfusions. After six months, 83 of the 99 patients who achieved transfusion-independence had maintained that response, and 52 of those maintained independence for more than a year. (See BioWorld Today, April 11, 2005.)

During the trial, the most common adverse events were neutropenia and thrombocytopenia, and the label for Revlimid, an analogue of thalidomide, carries a warning of potential birth defects aimed at pregnant women.

Shares of Celgene (NASDAQ:CELG) gained $3.37 Wednesday to close at $60.85.

Celgene has an ongoing Phase III study of Revlimid in MDS patients in Europe, where it filed for marketing authorization in October.

"Assuming all things work out, by this time next year, we'll have approval in Europe, as well," Gill said.

MDS is estimated by the World Health Organization to affect about 300,000 people worldwide, though that figure likely is a low estimate, since the disease often manifests itself as anemia and goes misdiagnosed. It typically strikes the elderly.

"So it's an unmet need today," Gill said, "but this population will be expanding in the future as the baby boomers enter their seventies."

Celegene expects to price Revlimid at $4,500 per month, which is "in line with other disease-altering therapies," Gill said.

The drug is expected to be available for patients early next month. Celgene will rely on its established sales force in hematology and oncology to get information out to physicians. The company also has focused on an educational campaign to inform physicians of the signs and symptoms of MDS.

There are few players in the MDS market. Boulder, Colo.-based Pharmion Corp. gained approval for its subcutaneously administered Vidaza (azacitidine), a pyrimidine nucleoside analogue, in 2004. Dacogen (decitabine), an injectable hypomethylating agent developed by Dublin, Calif.-based SuperGen Inc. and Minneapolis-based MGI Pharma Inc., received an approvable letter from the FDA in September. (See BioWorld Today, Sept. 6, 2005.)

For Celgene, the approval in MDS is "just the beginning" for Revlimid, which has been the subject of more than 50 independent trials in other indications, Gill said, adding that there "is a substantial interest in evaluating Revlimid more broadly in cancer."

This week, Celgene expects to file a supplemental NDA for the drug in multiple myeloma.

"We're hoping for a quick turnaround from the FDA in that," he said. Data there is supported by two large Phase III studies showing statistically significant survival data.

After that, the company might look at chronic lymphocytic leukemia. Data recently presented at the American Society of Hematology meeting in Atlanta showed that 16 of 19 evaluable CLL patients achieved stable disease or better after treatment with Revlimid. Those patients also showed a median decrease of 61 percent in absolute lymphocyte count.

The MDS, multiple myeloma and CLL markets "all are about the same size, so on a worldwide basis, we could be looking at close to a million patients," Gill said. "The future for Celgene looks very bright. If even half of what we see comes to fruition, it means some really great things for us."

The Summit, N.J.-based company is awaiting an FDA decision on another drug, Thalomid (thalidomide) in multiple myeloma. In November, it received a second approvable letter, requesting revised product labeling. Thalidomide has long been used in that indication off-label.

The company reported net income of $10.4 million, or 6 cents per share, for the third quarter. As of Sept. 30, it had cash and marketable securities of $746 million.

With the approval of Revlimid, the company expects an increase in pre-launch expenses of $5 million, resulting in 2005 full-year adjusted earnings per share to be 36 cents to 38 cent, down from the projected 55 cents.

On the same day it announced Revlimid approval, Celgene reported that its board approved a 2-for-1 stock split to increase the number of authorized shares of common stock from 280 million to 580 million. As of Nov. 30, the company had about 170 million shares outstanding.

The company also said its CEO, John Jackson, plans to retire May 1, to be succeeded by Sol Barer, who currently serves as president and chief operating officer. Jackson will continue to serve as chairman.