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Amgen Submits Supplemental Biologics License Application For Nplate® (romiplostim)
Application Seeks to Include Adult Patients who Have had Immune Thrombocytopenia for 12 Months or Less

THOUSAND OAKS, Calif., Dec. 19, 2018 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced the submission of a Supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) for Nplate® (romiplostim) to include the treatment of adult patients with immune thrombocytopenia (ITP) who have had ITP for 12 months or less and an insufficient response to corticosteroids, immunoglobulins or splenectomy.

"Patients living with ITP need more flexibility in their treatment options, and we believe Nplate has the potential to play a role earlier in the paradigm," said David M. Reese, M.D., executive vice president of Research and Development at Amgen. "We are proud that Nplate has helped thousands of patients with chronic ITP and look forward to working with the FDA on this expanded label application."

The submission is supported by nine studies evaluating the safety and efficacy of Nplate in adults with ITP, including two long-term open-label extension studies. Expansion of the indication for Nplate to include adult patients with ITP for 12 months or less is supported by data from 311 patients who were diagnosed with ITP within 12 months prior to study enrollment.

ITP is a rare, serious autoimmune disease characterized by low platelet counts in the blood (a condition known as thrombocytopenia) and impaired platelet production.1 In the U.S., the estimated incidence of ITP is 3.3 per 100,000 adults annually.1

About Nplate® (romiplostim)

Nplate is a thrombopoietin (TPO) receptor agonist that mimics the body's natural TPO and is designed to increase platelet counts in patients with chronic immune thrombocytopenia (ITP).2 In the U.S. and European Union (EU), Nplate is approved for the treatment of chronic ITP in adults who have had an insufficient response to other medicines or had surgery to remove the spleen. In the U.S. and EU, Nplate is also approved for the treatment of ITP in children age one year and older who have had the disease for at least six months and who have had an insufficient response to other medicines or had surgery to remove the spleen.

Nplate is also approved in 67 countries, including Canada and Australia.

For more information about Nplate, please visit www.Nplate.com.

Important U.S. Nplate® Safety Information

Risk of Progression of Myelodysplastic Syndromes to Acute Myelogenous Leukemia

  • In Nplate® clinical trials of adult patients with myelodysplastic syndromes (MDS) and severe thrombocytopenia, progression from MDS to acute myelogenous leukemia (AML) has been observed.
  • Nplate® is not indicated for the treatment of thrombocytopenia due to MDS or any cause of thrombocytopenia other than chronic ITP.

Thrombotic/Thromboembolic Complications

  • Thrombotic/thromboembolic complications may result from increases in platelet counts with Nplate® use. Portal vein thrombosis has been reported in patients with chronic liver disease receiving Nplate®.
  • To minimize the risk for thrombotic/thromboembolic complications, do not use Nplate® in an attempt to normalize platelet counts. Follow the dose adjustment guidelines to achieve and maintain a platelet count of ≥ 50 x 109/L.

Loss of Response to Nplate®

  • Hyporesponsiveness or failure to maintain a platelet response with Nplate® should prompt a search for causative factors, including neutralizing antibodies to Nplate®.
  • To detect antibody formation, submit blood samples to Amgen (1-800-772-6436). Amgen will assay these samples for antibodies to Nplate® and thrombopoietin (TPO).
  • Discontinue Nplate® if the platelet count does not increase to a level sufficient to avoid clinically important bleeding after 4 weeks at the highest weekly dose of 10 mcg/kg.

Adverse Reactions

Adult ITP

  • In the placebo-controlled trials of adult ITP patients, headache was the most commonly reported adverse drug reaction, occurring in 35% of patients receiving Nplate® and 32% of patients receiving placebo. Headaches were usually of mild or moderate severity.
  • Most common adverse reactions in adults (≥ 5% higher patient incidence in Nplate® versus placebo) were Arthralgia (26%, 20%), Dizziness (17%, 0%), Insomnia (16%, 7%), Myalgia (14%, 2%), Pain in Extremity (13%, 5%), Abdominal Pain (11%, 0%), Shoulder Pain (8%, 0%), Dyspepsia (7%, 0%), and Paresthesia (6%, 0%).

Pediatric ITP

  • In pediatric patients of age > 1 year receiving romiplostim for ITP, adverse reactions with an incidence of > 25% in the two randomized trials were: contusion (41%), upper respiratory tract infection (31%), and oropharyngeal pain (25%).
  • Most common adverse reactions (≥ 5% incidence and > 5% more frequent in the romiplostim arm) across the two placebo-controlled trials were contusion (41%), upper respiratory tract infection (31%), oropharyngeal pain (25%), pyrexia (24%), diarrhea (20%), rash (15%), and upper abdominal pain (14%).

Nplate® administration may increase the risk for development or progression of reticulin fiber formation within the bone marrow. This formation may improve upon discontinuation of Nplate®. In a clinical trial, one patient with ITP and hemolytic anemia developed marrow fibrosis with collagen during Nplate® therapy.

Women who become pregnant during Nplate® treatment are encouraged to enroll in Amgen's Pregnancy Surveillance Program. Patients or their physicians should call 1-800‑77‑AMGEN (1-800‑772‑6436) to enroll.

Please see full U.S. Prescribing Information and Medication Guide at www.Nplate.com.

About Amgen

Amgen is committed to unlocking the potential of biology for patients suffering from serious illnesses by discovering, developing, manufacturing and delivering innovative human therapeutics. This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology.

Amgen focuses on areas of high unmet medical need and leverages its expertise to strive for solutions that improve health outcomes and dramatically improve people's lives. A biotechnology pioneer since 1980, Amgen has grown to be one of the world's leading independent biotechnology companies, has reached millions of patients around the world and is developing a pipeline of medicines with breakaway potential.

For more information, visit www.amgen.com and follow us on www.twitter.com/amgen.

Forward-Looking Statements

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CONTACT:
Amgen, Thousand Oaks
Kristen Davis, 805-447-3008 (Media)
Andrea Fassacesia, 805- 905-2575 (Media)
Arvind Sood, 805-447-1060 (Investors)

References:

  1. National Organization for Rare Disorders. Immune Thrombocytopenia. https://rarediseases.org/rare-diseases/immune-thrombocytopenia/. Accessed Nov. 2, 2018.
  2. Nplate® (romiplostim) prescribing information, Amgen.

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