Avatrombopag for treating primary chronic immune thrombocytopenia

1.1 Avatrombopag is recommended, within its marketing authorisation, as an option for treating primary chronic immune thrombocytopenia (ITP) refractory to other treatments (for example, corticosteroids, immunoglobulins) in adults. It is only recommended if the company provides it according to the commercial arrangement.

Why the committee made these recommendations

Current treatment for newly diagnosed primary chronic ITP usually includes corticosteroids and immunoglobulins. This is followed by thrombopoietin receptor agonists (TPO‑RAs). Avatrombopag is another TPO‑RA.

Clinical trial evidence shows that avatrombopag is more effective than placebo at increasing the number of platelets in the blood (cells that help the blood to clot) to a level that meaningfully reduces the risk of bleeding. Avatrombopag may be as effective as other TPO‑RAs, but it has only been compared with them indirectly, which is uncertain.

There are also uncertainties with some assumptions in the economic modelling. Despite this, avatrombopag is likely to provide benefit for people who have primary chronic ITP. Also, the cost-effectiveness estimates are below what NICE normally considers an acceptable use of NHS resources. So, avatrombopag is recommended.