Amgen’s Nplate Approved With Restricted Access Program
This article was originally published in The Pink Sheet Daily
Executive Summary
FDA approval of the first platelet producer includes a REMS with a patient and physician tracking program.
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After a false start in July and two missed PDUFA dates, Amgen's Nplate (romiplostim) officially has cleared FDA as the first platelet producer for patients with the rare blood disorder chronic immune thrombocytopenic purpura. Nplate is approved for patients with chronic ITP who do not respond sufficiently to current therapies. Nplate will be available by the end of August at a wholesale acquisition cost of $4,600 per month, Amgen said. The approval positions Nplate for a first-to-market advantage over GlaxoSmithKline/Ligand's Promacta (eltrombopag), a thrombopoietin receptor agonist, which has a Sept. 19 action date. Approval of Nplate was expected after the biologic received a positive advisory committee review in March, and although FDA pushed back the original April 23 PDUFA date to review a risk plan, then missed the revised July 23 action date, it was widely assumed the agency was ironing out the details of a Risk Evaluation and Mitigation Strategy. Approval expectations were only heightened when Amgen jumped the gun by issuing a press release July 14 announcing the drug's approval, then retracting it the same day (1 (Also see "Nplate Risk Plan Includes Patient Support Initiatives" - Pink Sheet, 14 Jul, 2008.)). The approval does include a comprehensive REMS to address the risks of Nplate, which include fibrous deposits in the bone marrow and the possibility that once treatment is stopped, platelet counts could drop below levels prior to treatment. Other risks include blood clots due to excessive platelet increases, and, if given to patients with the blood condition myelodysplasia, a risk of blood cancer. The REMS includes a restricted access program called Nplate NEXUS (Network of Experts Understanding and Supporting Nplate and Patients). Patients and prescribers need to register with the program to prescribe, administer or receive Nplate. The REMS also includes a Medication Guide for patient education. In its unanimous vote of support for Nplate in March, FDA's Oncologic Drugs Advisory Committee agreed that a controlled distribution plan is necessary for the drug due to limited data on long-term safety (2 (Also see "FDA Panel Backs Amgen’s Nplate For Chronic Immune Thrombocytopenic Purpura" - Pink Sheet, 12 Mar, 2008.)). FDA has required long-term safety studies as part of the postmarketing requirements. NEXUS also will include a program to help eligible patients who are uninsured or underinsured to receive reimbursement support. The approval was based on the results of two randomized Phase III trials of about 125 patients who had received at least one prior ITP therapy. ITP is an orphan indication, with the disease affecting only about 60,000 people in the U.S., according to Amgen. Patients with the autoimmune disorder are prone to bruising and at risk for life-threatening bleeding. Current options for treatment include corticosteroids, immunoglobulin or surgery to remove the spleen. Nplate, a peptibody protein, works by stimulating the bone marrow to produce needed platelets. In clinical trials, patients treated with Nplate were able to reduce or discontinue use of concomitant ITP medications and emergency medications. The cost of treating chronic ITP with Nplate will be less than or comparable to the cost of care with standard treatment regimens, Amgen maintained. -Jessica Merrill ([email protected]) |