FDA approves bone marrow product to treat immune-related low platelet counts.
The condition, which usually develops in adults, is known as chronic immune thrombocytopenic purpura (ITP), a disease that results in a low number of platelets, the blood components that help with clotting. In patients with chronic ITP, the immune system is believed to destroy platelets and the patient's bone marrow is often unable to compensate for the loss.
The estimated 140,000 people with chronic ITP are prone to bruising and at risk for life-threatening bleeding. Current medical treatment includes corticosteroids and immunoglobulin. Surgery to remove the spleen, a procedure known as a splenectomy, may help some patients. Nplate is approved only for patients with chronic ITP who do not respond sufficiently to current treatments.
The FDA said it based its approval on two randomized clinical trials of about 125 patients who received at least one prior ITP treatment. One study enrolled patients who still had their spleen, the other enrolled patients who did not.
During six months of treatment, patients who received Nplate had significantly higher platelet counts and maintained those higher counts compared to those who did not receive the drug. The response to Nplate was higher in those patients who still had their spleen than in those patients who had undergone a splenectomy. In those patients who did not receive Nplate, only one experienced a sustained increase in platelet counts.
Safety concerns with Nplate include fibrous deposits in the bone marrow and the possibility that once Nplate is stopped, platelet counts could drop below they were before beginning the treatment.
The FDA said a Risk Evaluation and Mitigation Strategy (REMS) has developed to address the risks of Nplate therapy. Under the Food and Drug Administration Amendments Act of 2007, the agency has determined that a REMS is necessary for the benefits of Nplate to outweigh the risks of the product. The REMS will include a Medication Guide for patients and requires that all prescribers and patients enroll in a special program to track the long term safety of Nplate therapy.
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|Date:||Nov 1, 2008|
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