Recently, the FDA granted a breakthrough therapy designation to eltrombopag (Promacta, Novartis) for combination use with standard immunosuppressive therapy as a first-line treatment for patients with severe aplastic anemia (SAA).
Recently, the FDA granted a breakthrough therapy designation to eltrombopag (Promacta, Novartis) for combination use with standard immunosuppressive therapy as a first-line treatment for patients with severe aplastic anemia (SAA).
SAA is a rare blood disorder in which a patient’s bone marrow fails to produce enough red blood cells, white blood cells, and platelets.
Breakthrough designation therapy provides expedited FDA review for drugs that treat serious or life-threatening conditions that demonstrate a substantial improvement over existing therapies. Eltrombopag received this designation based on results from a phase 1/2 study conducted by researchers at the National Heart, Lung, and Blood Institute of the National Institutes of Health.
Novartis, who sponsors the drug, conducted an analysis of the study data and found that 52% of newly treated patients with SAA saw a complete response at 6 months when treated with eltrombopag at the start of and in conjunction with standard immunosuppressive treatment. The overall response rate was 85%.
“Promacta is a promising medicine that, if approved for first-line use in severe aplastic anemia, may redefine the standard of care for patients with this rare and seirous bone marrow condition,” said Samit Hirawat, MD, head of Novartis oncology global drug development in a statement. “We will continue to work closely with the FDA to make Promacta available to patients with SAA who are new to treatment as soon as possible.”
Eltrombopag is the only thrombopoietin receptor agonist indicated for the treatment of patients with SAA. Promacta, marketed as Revolade in most countries outside the United States, is also approved as a second-line therapy in patients with refractory SAA, and indicated for usage in adults and children with refractory chronic immune thrombocytopenia.
NCCN Guidelines Update Adds Momelotinib Below Ruxolitinib for High-, Low-Risk Myelofibrosis
January 23rd 2024Momelotinib was given category 2A and 2B status for patients with high- and low-risk myelofibrosis (MF) and MF with anemia. However, ruxolitinib retains a higher category of recommendation as a treatment for patients with MF.
Read More
Oncology Onward: A Conversation With Dr Shereef Elnahal, Under Secretary for Health
April 20th 2023Shereef Elnahal, MD, MBA, under secretary for health at the Veterans Health Administration (VHA), sat for a conversation with our hosts Emeline Aviki, MD, MBA, Memorial Sloan Kettering Cancer Center, and Stephen Schleicher, MD, MBA, Tennessee Oncology, that covered the cancer footprint of the VHA.
Listen
Interventions Needed to Increase DMT Uptake in Sickle Cell Disease
December 26th 2023A recent study found that uptake of disease-modifying therapies (DMTs) has been low among patients with sickle cell disease, suggesting that more interventions that consider individual patient characteristics are needed to improve adoption.
Read More
Exploring Payer Coverage Decisions Following FDA Novel Drug Approvals
May 3rd 2022On this episode of Managed Care Cast, Ari D. Panzer, BS, lead author and researcher, then at Tufts Medical Center—now at Duke University—discusses the findings from his team’s investigation into coverage decisions by health plan insurers of the 66 drugs approved by the FDA in 2018.
Listen
Exagamglogene Autotemcel Meets End Points in Severe Sickle Cell Disease, β-Thalassemia
December 7th 2023Two posters set to be presented at the 65th American Society of Hematology Annual Meeting & Exposition met their primary and secondary end points regarding exagamglogene autotemcel therapy for sickle cell disease and β-thalassemia.
Read More