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FDA grants breakthrough therapy designation to Promacta for severe aplastic anemia
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The FDA granted breakthrough therapy designation to eltrombopag in combination with standard immunosuppressive therapy for the initial treat of severe aplastic anemia, according to the agent’s manufacturer.
Eltrombopag (Promacta, Novartis), a TPO receptor agonist, is approved for second-line therapy of patients with refractory severe aplastic anemia. It also is approved for adults and children with low blood platelet counts due to chronic immune thrombocytopenia who are refractory to other treatments or for whom splenectomy did not work well enough.
Severe aplastic anemia is a rare blood disorder in which a person’s bone marrow does not produce sufficient red blood cells, white blood cells or platelets. This can lead to potentially debilitating symptoms or complications, including difficulty breathing, fatigue, abnormal bleeding or bruising, or recurring infections.
As many as one-third of patients either do not respond to or relapse after treatment with available therapies, according to a Novartis-issued press release.
The FDA based the breakthrough therapy designation for eltrombopag on Novartis’ analysis of research conducted by the NHLBI. Results showed 52% of patients with treatment-naive severe aplastic anemia who received the agent in combination with standard immunosuppressive therapy achieved complete response at 6 months. Researchers reported an 85% overall response rate.
The most common side effects reported among patients who received eltrombopag for severe aplastic anemia include nausea, fatigue, cough, diarrhea, headache, shortness of breath, fever, dizziness, abdominal pain, muscle spasms, abnormal liver function tests, runny nose, or pain in the legs, arms, feet or hands.
Novartis expects to complete regulatory filings for the first-line indication in the United States and Europe later this year.
“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 serious bone marrow condition,” Samit Hirawat, MD, head of Novartis Oncology global drug development, said in the press release. “We will continue to work closely with the FDA to make Promacta available to patients with severe aplastic anemia who are new to treatment as soon as possible.”