Rare Disease Report

FDA Expands Eltrombopag Indication to Include Severe Aplastic Anemia

NOVEMBER 16, 2018
Krista Rossi
The US Food and Drug Administration (FDA) has expanded Novartis’ eltrombopag (Promacta) to include first-line treatment for adults and pediatric patients aged 2 years and older with severe aplastic anemia (SAA) in combination with standard immunosuppressive therapy (IST).

"Severe aplastic anemia can be a fatal diagnosis if left untreated, and many patients fail to respond to current initial treatment options," said Liz Barrett, CEO, Novartis Oncology, in a recent statement. "Today's US approval for eltrombopag 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."

Data from a study sponsored by the National Heart, Lung and Blood Institute (NHLBI) Division of Intramural Research Program and conducted under a Cooperative Research and Development Agreement (CRADA) served as the basis for the approval.

Among the definitive IST-naive SAA patients treated with eltrombopag simultaneously with standard IST, a complete response at 6 months was demonstrated in 44% (95% CI 33, 55) of patients. Compared to the complete response rate historically observed with the standard IST, a complete response rate with the eltrombopag treatment was 27% higher.

At 6 months, the overall response rate was 79% (95% CI 69, 87).

Further building on the IST-refractory indication granted to eltrombopag in 2015 for patients with SAA, this data shows a subset of patients maintained stable counts and exhibited bone marrow function restoration following cessation of eltrombopag treatment.  

From IST-naïve SAA patients administered 6 months of eltrombopag in combination with horse anti-thymocyte globulin (h-ATG) and cyclosporine (CsA) followed by maintenance CsA4, the new data reflected a median duration of response of 24.3 months.  

"Patients with SAA sometimes do not respond to the current treatment standard of IST," said Phillip Scheinberg, MD, head, Division of Hematology, Hospital A Beneficência Portuguesa de São Paulo in Brazil, and previously with the Hematology Branch of the NHLBI, in a recent statement. "With this approval, physicians now have an option to add eltrombopag 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."

Previously, eltrombopag was approved for SAA patients who have had an insufficient response to IST in the refractory setting as well as for adults and children with chronic immune thrombocytopenia (ITP) for patients who are refractory to other treatments and for the treatment of thrombocytopenia in patients with chronic hepatitis C virus (HCV) infection.

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