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FDA Approval for Thalidomide

Brand name(s): Thalomid®

  • Approved for multiple myeloma

Full prescribing information is available, including clinical trial information, safety, dosing, drug-drug interactions and contraindications.

On May 26, 2006, the U.S. Food and Drug Administration granted accelerated approval for thalidomide (THALOMID®, made by the Celgene Corporation) in combination with dexamethasone for the treatment of newly diagnosed multiple myeloma (MM) patients.

Because of thalidomide’s known teratogenicity, the FDA is controlling thalidomide’s marketing in the United States via the System for Thalidomide Education and Prescribing Safety (S.T.E.P.S.®) program. This mandatory registry includes authorized patients, prescribers and pharmacies, extensive patient education regarding thalidomide’s safety, and is designed to prevent fetal exposure to thalidomide during pregnancy.

Thalidomide was evaluated in 207 patients with newly diagnosed MM in one open-label, multicenter, cooperative group trial. Patients were randomized to four cycles of thalidomide 200 mg daily plus dexamethasone (Thal/Dex) or to dexamethasone (Dex) alone. Response rates based on serum or urine paraprotein measurements were significantly higher in the combination arm than Dex alone (51.5 percent vs. 35.6 percent, respectively, p = 0.025). After completing treatment, patients were encouraged to undergo stem cell transplantation.

The incidence of grade 3 or 4 adverse events was 84.3 percent on the Thal/Dex arm and 73.5 percent on the Dex alone arm. The most common toxicities associated with thalidomide were somnolence, constipation, neuropathy, venous thromboembolism (VTE), and rash.

The incidence of VTE was significantly higher in the Thal/Dex arm than with Dex alone (22.5 percent vs. 4.9 percent, respectively, p = 0.002). Prophylactic antithrombotic therapy prescribed with thalidomide may reduce VTE; however, the decision to prescribe antithrombotic therapy should be made after a careful assessment of the patient’s underlying risk factors.

This approval is based on objective response rates and is approved under accelerated approval regulations (Subpart H) requiring further clinical trials to demonstrate thalidomide’s clinical benefit in the treatment of multiple myeloma.

This summary was provided by Richard Pazdur, M.D., director of the FDA's Division of Oncology Drug Products.

The FDA is the division of the U.S. Department of Health and Human Services charged with ensuring the safety and effectiveness of new drugs and other products. (See "Learn How Drugs and Devices Get Approved.") The FDA's mission is to promote and protect the public health by helping safe and effective products to reach the market in a timely way, and monitoring products for continued safety after they are in use.

  • Updated: July 3, 2013