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news.The US Food and Drug Administration (FDA) has approved GlaxoSmithKline's (GSK) supplemental new drug application (sNDA) for Promacta (eltrombopag) to treat patients with severe aplastic anaemia (SAA) who have had an insufficient response to immunosuppressive therapy (IST).
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In order to increase production of blood cells, the once-daily use of an oral thrombopoietin (TPO) receptor agonist promacta helps to induce proliferation and differentiation of bone marrow stem cells.
SAA is a rare and serious blood disorder condition wherein the bone marrow fails to make enough red blood cells, white blood cells, and platelets.
The approval is based on results from an investigator-sponsored Phase II trial (09-H-0154) carried out by the National Heart, Lung and Blood Institute (NHLBI) at the National Institutes of Health (NIH).
In this single-arm, single-center, open-label trial, haematologic response has been observed in SAA. Around 43 patients were treated with eltrombopag who had an insufficient response to IST.
GSK president of Oncology Paolo Paoletti said the FDA approval of Promacta addresses a significant treatment need for this very rare but serious blood disorder in those who have failed current treatment options.
"Through collaboration with the National Institutes of Health, whose studies demonstrate the potential for Promacta to achieve a haematologic response in at least one lineage – red blood cells, platelets, or white blood cells – patients now have a treatment option where one didn’t previously exist," Paoletti said.
The UK drug-maker has secured the FDA breakthrough therapy designation for Promacta in January 2014 and Priority Review in April 2014.
During the trial, the drug was given at an initial dose of 50mg once daily for two weeks and increased over two-week periods up to a maximum dose of 150mg once daily.
Image: GlaxoSmithKline headquaters in London, UK. Photo: courtesy of Maxwell Hamilton.