FDA Relaxes REMS Requirements on 2 Low Platelet Drugs

Laird Harrison

Disclosures

December 06, 2011

December 6, 2011 — The US Food and Drug Administration (FDA) has dropped requirements that anyone using, prescribing, or providing romiplostim (Nplate, Amgen, Inc) or eltrombopag (Promacta, GlaxoSmithKline) enroll in a monitoring network, the agency announced today.

"An FDA-initiated review of the current information has determined that while safety risks for both Nplate and Promacta still exist, certain restrictive requirements of the [risk evaluation and management strategy (REMS)] programs are no longer necessary to ensure that the benefits of the drugs outweigh their risks," the agency said in a press release.

Romiplostim and eltrombopag were both approved in 2008 to treat adult patients with chronic immune thrombocytopenia (ITP) who have not responded adequately to corticosteroids, immunoglobulins, or splenectomy.

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

The restricted distribution requirements called for healthcare professionals, pharmacies, hospitals, specialty care facilities, and patients to enroll in a special access program to prescribe, dispense, or receive the drugs.

The new ruling makes the following changes:

  • Healthcare 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. Healthcare professionals also will no longer be required to complete periodic safety forms for patients receiving romiplostim or eltrombopag.

  • Pharmacies/pharmacists 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 that will inform healthcare professionals about the changes to the REMS and the safety risks associated with each product.

Since the approval of romiplostim and eltrombopag, the FDA has continued to monitor specific safety risks related to both products, the agency said.

These include bone marrow changes of collagen deposition (reticulin), higher risk for blood clots, increased risk of developing hematological malignancies resulting from the stimulation of bone marrow cells, worsening low blood platelet count, and the risk for bleeding shortly after discontinuing the drugs. Eltrombopag may also cause liver injury.

Other adverse effects seen in patients treated with romiplostim include headaches; dizziness; trouble falling or staying asleep (insomnia); pain in the stomach (abdominal), shoulder, joints (arthralgia), and muscle (myalgia); upset stomach (dyspepsia); and tingling or numbness in the skin (paresthesia).

Eltrombopag has been associated with the following adverse effects: nausea, diarrhea, upper respiratory tract infection, vomiting, rash, influenza, pharyngitis, back pain, and paresthesia.

The FDA gave these additional guidelines for health professionals:

  • The warnings and precautions sections of the product labels have been updated to reflect the current understanding of the safety of the drugs.

  • Healthcare 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.

ITP is a rare blood disorder that results in a low number of platelets, the blood components that help with clotting. Low numbers of platelets can lead to serious bleeding. The treatments work by stimulating the bone marrow to produce needed platelets.

In a separate press release, Amgen said that its investigators, working with the FDA, "have determined that the safety information collected through the REMS, which is based on individual case safety reports, is inherently confounded by underlying medical conditions in the treated patient population and thus cannot be used to determine the precise role of Nplate in the development of the adverse events."

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