The European Commission (EC) has approved orphan drug Scemblix (asciminib), providing a new treatment option for patients who are intolerant or resistant to tyrosine kinase inhibitors (TKIs).
It is approved to treat adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia in chronic phase who have been previously treated with two or more TKI therapies.
Scemblix is the first CML treatment in Europe that works by specifically targeting the ABL myristoyl pocket (also known as a STAMP inhibitor in scientific literature).
Hope for TKI intolerant patients
“Until now, patients with CML in Europe had oral TKI therapies with the same mechanism of action to turn to, and those experiencing significant side effects or resistance to these treatment options would often cycle between these very similar therapies, with little success in controlling their disease or improving their quality of life,” said Dr Andreas Hochhaus, Head of the Department of Hematology and Medical Oncology at Jena University Hospital in Germany.
“The approval of Scemblix in Europe is a timely milestone that will help many patients find hope for the management of their CML.”
The EC approval for Scemblix, which is manufactured by Novartis, follows a positive opinion by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) in June, and the previous designation of Scemblix as an orphan drug.
Positive data from ASCEMBL trial
EC approval is based on results from the pivotal Phase III ASCEMBL trial, which showed a near doubling in MMR rate for patients treated with Scemblix vs. Bosulif (bosutinib) (25.5% vs. 13.2%, [P=.029]), with a more than three times lower discontinuation rate due to adverse reactions (5.8% vs. 21.1%), at the 24-week primary endpoint.
These results were confirmed in the 96-week longer-term follow-up where the MMR rate was more than double with Scemblix compared with Bosulif and the discontinuation rate due to adverse reactions was 7.7% for Scemblix and 26.3% for Bosulif.