FDA Announces New REMS for Drugs to Treat Low Platelet Counts

The FDA has approved changes to the Risk Evaluation and Mitigation Strategies (REMS) for both Nplate (romiplostim) and Promacta (eltrombopag).

A review of current information by the agency determined that while safety risks for both Nplate and Promacta still exist, certain restrictive requirements of the REMS programs are no longer necessary to ensure that the benefits of the drugs outweigh the risks. The FDA will continue to monitor these drugs for safety risks.

The agency concluded that the long-term safety of Nplate and Promacta can be evaluated based on ongoing clinical trials, post-approval studies agreed to by both companies, and adverse event reports submitted to FDA.

Major changes to the REMS for both products include:

• Health care professionals, hospitals, specialty care facilities, and patients are no longer required to be enrolled in the Nplate NEXUS (Network of Experts Understanding and Supporting Nplate and Patients) Program or the Promacta CARES Program to prescribe, dispense, or receive these products. Health care professionals also will no longer be required to complete periodic safety forms for patients receiving Nplate or Promacta.
• Pharmacies/pharmacists are no longer are required to enroll in the Promacta CARES Program or verify prescriber and patient enrollment before dispensing Promacta.
• The modified REMS programs will include a communication plan to inform health care professionals about the changes to the REMS and the safety risks associated with each product.

Health care professionals and patients should also be aware that:

• The warnings and precautions sections of the product labels have been updated to reflect the current understanding of the safety of the drugs.
• Health care professionals should continue to refer to the drug prescribing information for the latest recommendations on prescribing Nplate and Promacta and report adverse events for both products to the FDA MedWatch program.
• Patients should read the Medication Guide that they receive with their Nplate or Promacta prescription and contact their health care professional regarding any questions or concerns.

“REMS continue to be vital tools for the agency to employ as we work with companies to address the serious risks associated with drugs and monitor their appropriate and safe use in various health care settings,” said Janet Woodcock, MD, director of FDA’s Center for Drug Evaluation and Research, in a statement. “The agency remains committed to exercising a flexible and responsible regulatory approach that ensures REMS programs are being effectively and efficiently used and not resulting in an unnecessary burden on health care professionals and patients.”

Nplate and Promacta were both approved in 2008 to treat adult patients with chronic immune thrombocytopenia (ITP), who have not responded adequately to corticosteroids, immunoglobulins, or to the removal of their spleen. ITP is a rare blood disorder that results in a low number of platelets. The treatments work by stimulating the bone marrow to produce needed platelets.

At the time of approval, experience with both drugs was limited, and safety concerns led to a decision to require a REMS that included restricted distribution requirements for each drug to ensure that the benefits of the drugs outweighed their risks.

Since the approval of Nplate and Promacta, the FDA has continued to monitor specific safety risks related to both products, including bone marrow changes of collagen deposition, higher risk for blood clots, increased risk of development of other blood-related cancers resulting from the stimulation of bone marrow cells, worsening low blood platelet count and the risk of bleeding shortly after discontinuing the drugs. Promacta may also cause liver injury.

Nplate is manufactured by Amgen Inc, and Promacta is manufactured by GlaxoSmithKline.