Novartis Promacta® Approved as First-Line Treatment for Severe Aplastic Anemia(SAA)

Novartis Promacta® is approved as the first-line treatment for adults and pediatric patients two years and older with Severe Aplastic Anemia (SAA) in combination with standard immunosuppressive therapy (IST).

Promacta is an oral thrombopoietin receptor agonist (TPO-RA) that is already approved for SAA for patients who have had an insufficient response to IST.

It can also be used for adults and children with chronic immune thrombocytopenia (ITP) who are refractory to other treatments, and for the treatment of thrombocytopenia in patients with chronic hepatitis C virus (HCV) infection.

The results were based on Novartis' analysis of research 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).

The study showed that 44% (95% CI 33, 55) of definitive IST-naive SAA patients achieved complete response at 6 months when treated with Promacta concurrently with standard IST.

It was 27 percentage points higher than the complete response rate historically observed with the standard IST alone. The overall response rate was 79% (95% CI 69, 87) in 6 months.

The response rates showed that there has been a substantial clinical impact for patients with SAA who were not treated earlier with standard IST.

These results further build on the IST-refractory indication for patients with SAA which Promacta was granted in 2015, in which a subset of patients maintained stable counts and demonstrated restoration of bone marrow function following Promacta discontinuation.

The new data in IST-naïve SAA includes sustained response with a median duration of response of 24.3 months for patients receiving 6 months of Promacta in combination with horse anti-thymocyte globulin (h-ATG) and cyclosporine (CsA) followed by maintenance CsA4.

The most common adverse reactions reported (incidence ?5%) were abnormal liver function tests, rash, and skin discoloration including hyper pigmentation.

Physicians can add Promacta to the standard IST in a regimen that has demonstrated significant overall and complete response rates upfront in SAA.

FDA has approved Promacta® for first-line SAA and grants breakthrough therapy designation for additional new indication.

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