Amgen and FDA Agree to Modify Nplate (Romiplostim) Risk Evaluation and Mitigation Strategy
Prescribing Physicians, Patients and Institutions no Longer Required to Enroll in Nplate(R) NEXUS Program
THOUSAND OAKS, Calif., Dec. 6, 2011 /PRNewswire/ -- Amgen (NASDAQ: AMGN) today announced the U.S. Food and Drug Administration (FDA) has modified the requirements of the Nplate® (romiplostim) Risk Evaluation and Mitigation Strategy (REMS) Program. Prescribing physicians, patients and institutions are no longer required to enroll in the safety monitoring program, called the Nplate® NEXUS (Network of EXperts Understanding and Supporting Nplate and Patients) Program, in order to prescribe or receive Nplate.
"The goals of the Nplate® NEXUS Program were to educate physicians and patients about treatment risks and benefits, and to define the long-term safety profile of Nplate," said Roger M. Perlmutter, M.D., Ph.D., executive vice president of Research and Development at Amgen. "The FDA has now concluded that enrollment in a restricted distribution program is no longer necessary to prescribe or receive Nplate. Amgen will continue to monitor the safety profile of Nplate through clinical trials and post-marketing pharmacovigilance programs."
The primary modification of the REMS is the removal of the elements to assure safe use; however, a modified REMS communication plan will remain in place. In addition, the Medication Guide will no longer be part of the REMS, but will be part of approved product labeling.
From 2008 to 2011, the Nplate® NEXUS Program has collected long-term safety data from more than 5,200 patients. The restrictive elements of the REMS included enrollment of prescribers, patients and institutions to assist in collecting long-term safety information. Upon further review, the FDA and Amgen 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. Based in part on this determination, and the data submitted from clinical trials, the FDA and Amgen have concluded that the restricted elements of the REMS can be eliminated. For this reason enrollment of prescribers, patients and institutions and mandatory collection of safety data is no longer required.
As of Dec. 7, 2011, prescribing physicians and institutions will be able to order Nplate without enrolling themselves or patients in the Nplate® NEXUS Program. The program will continue to obtain information from already enrolled patients through Jan. 5, 2012, and Nplate® NEXUS Program support assistance will continue for 28 days after approval of the modified REMS.
About Adult ITP
In patients with immune thrombocytopenia (ITP), platelets - blood elements needed to prevent bleeding - are destroyed by the patient's own immune system. Low platelet counts leave adult ITP patients open to sudden serious bleeding events. The risk for serious bleeding events increases when platelet counts drop to less than 30,000 platelets per microliter; normal counts range from 150,000 to 400,000 platelets per microliter. ITP has historically been considered a disease of platelet destruction although recent data suggest that the body's natural platelet production processes in ITP are also unable to compensate for low levels of platelets in the blood. Increasing the rate of platelet production may address low platelet levels associated with ITP. Currently, there are approximately 90,000 adult chronic ITP patients in Europe and the U.S. ITP affects about twice as many adult women as men.
The Nplate FIRST STEP™ Co-Pay Coupon Card Program
Last year, Amgen announced its co-pay coupon umbrella program, the Amgen FIRST STEP™ Program, for commercially insured patients. As part of that program, the Nplate FIRST STEP™ Co-Pay Coupon Card Program, is intended to provide assistance to eligible patients who need help meeting their Nplate deductible, co-insurance and/or co-payment (out-of-pocket) requirements. The Amgen FIRST STEP™ Program is significant among oncology commercial co-pay coupon programs, as it is the first program under the medical benefit with no income eligibility requirement. Under this program, eligible patients will incur no out of pocket costs for their first Nplate treatment associated with a new treatment regimen and will pay a maximum of $25 for subsequent injections. More information, eligibility requirements, restrictions and limitations about the co-pay coupon program are available at www.AmgenFIRSTSTEP.com.
Nplate is approved in the U.S., European Union (EU), Canada, Australia, Russia, Mexico, Switzerland, Lichtenstein, Japan, Argentina, Israel, South Korea, Hong Kong, and Chile. Nplate also has received orphan designation for chronic ITP in the U.S. (2003), the EU (2005), Switzerland (2005), Japan (2006), Mexico (2010) and South Korea (2010).
Nplate is the first FDA-approved treatment specifically for adult chronic ITP. It is also being investigated for potential use in children ages 12 months to 18 years old with persistent severe thrombocytopenia, and chemotherapy-induced thrombocytopenia (CIT).
In the U.S., Nplate is indicated for the treatment of thrombocytopenia in patients with chronic ITP who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy. Nplate is not indicated for the treatment of thrombocytopenia due to myelodysplastic syndrome (MDS) or any cause of thrombocytopenia other than chronic ITP. Nplate should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increase the risk for bleeding. Nplate should not be used in an attempt to normalize platelet counts.
In the EU, Nplate is indicated for the treatment of splenectomized adult chronic ITP patients who are refractory to other treatments (e.g., corticosteroids, immunoglobulins). Nplate may be considered as a second-line treatment for adult non-splenectomized ITP patients for whom surgery is contraindicated.
Nplate was named as a recipient of the U.S. Prix Galien 2009 "Best Biotechnology Product" award and also received the 2009 Scrip Awards for "Best New Drug." Nplate has also been honored with numerous awards throughout the EU, including a 2010 Prix Galien in France in the category of "Drugs for Rare Diseases," and the 2011 Prix Galien in Germany in the category of "Specialist Care." In September 2010, Nplate was awarded the 2010 International Prix Galien Award, an award granted every two years which recognizes the "best of the best" selected from previous national Prix Galien award recipients.
For more information about Nplate, please visit www.Nplate.com.
Important U.S. Nplate Safety Information
The risks associated with Nplate include progression of MDS to acute myelogenous leukemia (AML) in patients with MDS, thrombotic/thromboembolic complications, bone marrow reticulin formation and risk for bone marrow fibrosis, worsened thrombocytopenia after cessation of Nplate, and lack or loss of response to Nplate. In the placebo-controlled studies, headache was the most commonly reported adverse drug reaction.
Important EU Nplate Safety Information
The most common side effects are headache, fatigue, arthralgia and myalgia.
The risks associated with Nplate include reoccurrence of thrombocytopenia, bleeding after cessation of treatment, increased bone marrow reticulin, thrombotic/thromboembolic complications, progression of existing MDS (in patients with MDS), loss of response to Nplate, and effects on red and white blood cells.
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Forward Looking Statements
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Posted: December 2011