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Novartis' Promacta cleared by FDA for first-line use in severe aplastic anaemia

Published on 19/11/18 at 12:18pm

Novartis has secured expanded approval from the FDA for Promacta (eltrombopag), allowing it to now be used in combination with standard immunosuppressive therapy (IST) for the first-line treatment of severe aplastic anaemia (SAA).

The drug, which is also known as Revolade outside the US, is already approved as a treatment for SAA in patients who have responded inadequately to IST, as well as in chronic immune thrombocytopenia (ITP) in patients who have proven refractory to other therapies, and thrombocytopenia in patients with chronic hepatitis C virus (HCV).

The decision was drawn from research sponsored by the National Heart, Lung and Blood Institute (NHLBI) Division of Intramural Research Program which showed that the combo recorded a complete response rate of 44% within six months – 27% higher than historically seen with IST alone. An overall response rate of 79% was also recorded.  

"Patients with SAA sometimes do not respond to the current treatment standard of IST," commented Dr Phillip Scheinberg, Head, Division of Hematology at Hospital A Beneficência Portuguesa de São Paulo in Brazil. "With this approval, physicians now have an option to add Promacta to the standard IST in a regimen that has demonstrated significant overall and complete response rates upfront in SAA and reduce the numbers of those who are unresponsive to initial therapy."

The rare blood disorder SAA is characterised by insufficient levels of red and white blood cells and platelets in the bone marrow, which can lead to abnormal bleeding or bruising, fatigue, recurrent infections, trouble breathing, and death due to an inability to produce new blood cells.

"Severe aplastic anaemia can be a fatal diagnosis if left untreated, and many patients fail to respond to current initial treatment options," added Novartis Oncology CEO Liz Barrett. "Today's US approval for Promacta is an important step forward for people living with this challenging disease and shows how Novartis continues to reimagine care in areas where few treatment options exist."

Matt Fellows

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