Novartis Promacta® Approved as First-Line Treatment for Severe Aplastic Anemia(SAA)
Novartis' Promacta® has received approval as the primary treatment option for Severe Aplastic Anemia (SAA) in both adults and pediatric patients aged two years and above. This approval is in combination with standard immunosuppressive therapy (IST).
Promacta, an oral thrombopoietin receptor agonist (TPO-RA), was initially approved for SAA patients who did not respond adequately to IST. However, it can now be utilized for adults and children with chronic immune thrombocytopenia (ITP) who have not responded to other treatments. Additionally, Promacta is used to treat thrombocytopenia in patients with chronic hepatitis C virus (HCV) infection.
The approval was based on Novartis' analysis of research conducted under a Cooperative Research and Development Agreement (CRADA), sponsored by the National Heart, Lung, and Blood Institute (NHLBI) Division of Intramural Research Program.
The study revealed that when Promacta was administered alongside standard IST, 44% (95% CI 33, 55) of SAA patients who had not undergone IST treatment achieved a complete response within six months. This rate was 27 percentage points higher compared to the historical complete response rate achieved with standard IST alone. The overall response rate within six months was 79% (95% CI 69, 87).
These results demonstrate significant clinical benefits for SAA patients who did not receive standard IST treatment earlier. The findings also reinforce Promacta's indication for patients with SAA who are unresponsive to IST, which was granted in 2015. In that indication, a subset of patients maintained stable blood counts and experienced the restoration of bone marrow function after discontinuing Promacta.
In the new data for IST-naïve SAA patients, sustained response was observed with a median duration of response of 24.3 months among patients who received six 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 hyperpigmentation.
Physicians now have the option to incorporate Promacta into the standard IST regimen, which has demonstrated significant overall and complete response rates as an upfront treatment for SAA.
Furthermore, the FDA has granted Promacta breakthrough therapy designation for an additional new indication, further highlighting its potential in the field.
