Primary ITP is a rare autoimmune condition characterised by high platelet destruction

Sobi has announced that the National Institute for Health and Care Excellence (NICE) has issued the Final Appraisal Document in England and Wales recommending the use of Doptelet.

Also known as avatrombopag, the treatment involves adults with primary chronic immune thrombocytopenia (ITP) who are refractory to other treatments such as corticosteroids or immunoglobulins.

The appraisal follows results from a double-blind, randomised phase 3 study with open-label extension and two sequential phase 2 studies. This research investigated the safety and efficacy of avatrombopag as a therapy for adults with primary ITP.

During the research patients received one or more therapies for ITP prior to the study. Among the 49 patients, Doptelet was superior to placebo for the primary endpoint of the cumulative number of weeks of platelet response.

Dr Drew Provan, Consultant Haematologist at Barts and The London School of Medicine and Dentistry, explained: “The introduction of the TPO-RAs, as a class of treatment, has changed the ITP management landscape significantly, allowing us to avoid immune suppression, especially important during the pandemic. Avatrombopag has no food-type restrictions, no routine liver function monitoring and no need to dose reduce in East Asian patients.”

Mervyn Morgan, CEO of the UK and Ireland Patient Support Organisation for Immune Thrombocytopenia, added: “Chronic ITP is a long-term burdensome disease with many suffering fatigue levels that impact their everyday life. Some patients prefer oral treatments with less dietary restrictions, so having different treatment options is incredibly important.

“I am pleased that ITP patients in England and Wales now have access to avatrombopag, like patients in Scotland and other parts of Europe.”

Primary ITP is a rare autoimmune disorder characterised by high platelet destruction and impaired platelet production, with 3,000-4,000 adults in the UK estimated to have ITP at any one time and 80% of those adults experiencing a chronic condition.

Doptelet is already approved in Scotland for the treatment of ITP.