Application Granted Priority Review Designation
Approval Based on Results That Demonstrated Nplate Successfully Increased and Sustained Platelet Counts in Children Affected by Rare Blood Disorder
THOUSAND OAKS, CA, USA I December 14, 2018 I Amgen (NASDAQ: AMGN) today announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for Nplate® (romiplostim) for the treatment of pediatric patients one year of age and older with immune thrombocytopenia (ITP) for at least six months who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy.
“Today’s approval underscores our long-standing commitment to making a positive impact on the lives of patients with rare and difficult-to-treat hematological disorders,” said David M. Reese, M.D., executive vice president of Research and Development at Amgen. “In the 10 years since the FDA approved Nplate as the first platelet booster for adult patients with chronic ITP, it has made a difference in the lives of thousands of adults, and we’re proud to bring this treatment option to children who need it most.”
The approval was based on two placebo-controlled studies – Phase 3 and Phase 1/2 – evaluating the safety and efficacy of Nplate in pediatric patients. In the Phase 3 study, published in The Lancet, rates of overall platelet response were increased with the Nplate group (71 percent) compared with placebo (20 percent), p<0.05. Additionally, durable platelet response occurred more frequently with Nplate (52 percent) compared with placebo (10 percent), p<0.05. In the two placebo-controlled trials, adverse reactions with an incidence of > 25 percent in the Nplate arm were contusion, upper respiratory tract infection and oropharyngeal pain.
“Children with ITP are at risk for serious bleeding events and spontaneous bruising due to low platelet counts, which can be worrying for these young patients and their parents. Currently, these patients have a limited number of treatment options, especially for those with refractory disease,” said Michael D. Tarantino, M.D., president of the Bleeding and Clotting Disorders Institute and professor of Pediatrics and Medicine, University of Illinois College of Medicine-Peoria, Peoria, Ill. “Today’s approval of Nplate offers new hope to the pediatric ITP community as it provides children with a new treatment option that may help to maintain safe platelet counts.”
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 prevalence of ITP in children is 5.3 per 100,000 children annually.1 The treatment goal for children with ITP is to achieve and maintain a platelet count that reduces the risk of bleeding.2
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).3 In the U.S. and European Union, Nplate is approved for the treatment of chronic ITP in adults and in children age one year and older with ITP for at least six months, 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, Australia and Japan.
For more information about Nplate, please visit 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.
References:
- National Organization for Rare Disorders. Immune Thrombocytopenia. https://rarediseases.org/rare-diseases/immune-thrombocytopenia/. Accessed Nov. 2, 2018.
- Children’s National Health System. Pediatric Idiopathic Thrombocytopenia Purpura (ITP). https://childrensnational.org/choose-childrens/conditions-and-treatments/blood-marrow/idiopathic-thrombocytopenia-purpura-itp. Accessed Nov. 2, 2018
- Nplate® (romiplostim) prescribing information, Amgen.
SOURCE: Amgen
Post Views: 28
Application Granted Priority Review Designation
Approval Based on Results That Demonstrated Nplate Successfully Increased and Sustained Platelet Counts in Children Affected by Rare Blood Disorder
THOUSAND OAKS, CA, USA I December 14, 2018 I Amgen (NASDAQ: AMGN) today announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for Nplate® (romiplostim) for the treatment of pediatric patients one year of age and older with immune thrombocytopenia (ITP) for at least six months who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy.
“Today’s approval underscores our long-standing commitment to making a positive impact on the lives of patients with rare and difficult-to-treat hematological disorders,” said David M. Reese, M.D., executive vice president of Research and Development at Amgen. “In the 10 years since the FDA approved Nplate as the first platelet booster for adult patients with chronic ITP, it has made a difference in the lives of thousands of adults, and we’re proud to bring this treatment option to children who need it most.”
The approval was based on two placebo-controlled studies – Phase 3 and Phase 1/2 – evaluating the safety and efficacy of Nplate in pediatric patients. In the Phase 3 study, published in The Lancet, rates of overall platelet response were increased with the Nplate group (71 percent) compared with placebo (20 percent), p<0.05. Additionally, durable platelet response occurred more frequently with Nplate (52 percent) compared with placebo (10 percent), p<0.05. In the two placebo-controlled trials, adverse reactions with an incidence of > 25 percent in the Nplate arm were contusion, upper respiratory tract infection and oropharyngeal pain.
“Children with ITP are at risk for serious bleeding events and spontaneous bruising due to low platelet counts, which can be worrying for these young patients and their parents. Currently, these patients have a limited number of treatment options, especially for those with refractory disease,” said Michael D. Tarantino, M.D., president of the Bleeding and Clotting Disorders Institute and professor of Pediatrics and Medicine, University of Illinois College of Medicine-Peoria, Peoria, Ill. “Today’s approval of Nplate offers new hope to the pediatric ITP community as it provides children with a new treatment option that may help to maintain safe platelet counts.”
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 prevalence of ITP in children is 5.3 per 100,000 children annually.1 The treatment goal for children with ITP is to achieve and maintain a platelet count that reduces the risk of bleeding.2
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).3 In the U.S. and European Union, Nplate is approved for the treatment of chronic ITP in adults and in children age one year and older with ITP for at least six months, 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, Australia and Japan.
For more information about Nplate, please visit 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.
References:
- National Organization for Rare Disorders. Immune Thrombocytopenia. https://rarediseases.org/rare-diseases/immune-thrombocytopenia/. Accessed Nov. 2, 2018.
- Children’s National Health System. Pediatric Idiopathic Thrombocytopenia Purpura (ITP). https://childrensnational.org/choose-childrens/conditions-and-treatments/blood-marrow/idiopathic-thrombocytopenia-purpura-itp. Accessed Nov. 2, 2018
- Nplate® (romiplostim) prescribing information, Amgen.
SOURCE: Amgen
Post Views: 28
