The US Food and Drug Administration (FDA) has granted breakthrough therapy designation for GlaxoSmithKline’s (GSK) Promacta/Revolade (eltrombopag) for the treatment of cytopenias in patients with severe aplastic anaemia (SAA) who have had insufficient response to immunosuppressive therapy.
The FDA breakthrough therapy designation was based on the results from an open-label, Phase II National Institute of Health (NIH) study (09-H-0154) of eltrombopag conducted in 43 heavily pre-treated SAA patients with an insufficient response to IST.
The breakthrough therapy designation includes all the features of the fast track designation, as well as more intensive guidance from the FDA on a drug’s clinical development programme.
FDA breakthrough therapy status is aimed at accelerating the development and review times of drugs for serious or life-threatening conditions.
The company said that eltrombopag is not approved or licensed anywhere in the world for use in this rare disorder SAA.
Eltrombopag olamine is a small-molecule, nonpeptide thrombopoietin receptor agonist with megakaryopoiesis-stimulating activity.
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It is currently under development for the treatment of thrombocytopenia with chronic hepatitis C and compensated liver cirrhosis, haeme-oncology-related thrombocytopaenia, acute myelocytic leukaemia, myelodysplastic syndrome and aplastic anaemia.
In SAA, the bone marrow fails to make enough new blood cells, and currently there are no approved therapies available for SAA patients unresponsive to initial immunosuppressive therapy (IST).
Out of those patients unresponsive to initial IST, about 40% die from infection or bleeding within five years of their diagnosis.
Image: Eltrombopag is marketed under the brand name Revolade in Europe. Photo: courtesy of freedigitalphotos.net.