Novartis’ Scemblix (asciminib) has been approved by the European Commission (EC) for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia (CML) in chronic phase, previously treated with two or more tyrosine kinase inhibitors (TKIs), the company announced.
CML is a type of cancer in which the body produces cancerous white blood cells and almost all CML patients have an abnormality known as the Philadelphia chromosome, which causes malignant white blood cells to proliferate.
It is estimated that, every year, more than 6,300 people will be diagnosed with CML in Europe. Although many patients will benefit from available TKI therapies, there are limited options for a significant proportion of patients whose disease has developed resistance to therapy.
Scemblix is the first CML treatment that acts as a STAMP inhibitor, specifically targeting the ABL myristoyl pocket. This treatment approach may help address resistance in patients with CML previously treated with two or more TKIs and overcome mutations at the defective BCR::ABL1 gene, which is associated with the over-production of leukaemic cells.
The approval is based on results from the pivotal phase 3 ASCEMBL trial, which demonstrated a near doubling in maternal mortality ratio (MMR) rate for patients treated with Scemblix compared to Pfizer’s Bosulif (bosutunib).
Moreover, the discontinuation rate due to adverse reactions of those treated with Scemblix was more than three times lower.
The results were confirmed in the 96-week longer-term follow-up where the MMR rate was more than double with Scemblix compared to Bosulif, and the discontinuation rate due to adverse reactions was 7.7% for Scemblix and 26.3% for Bosulif.
Commenting on the approval, Dr Andreas Hochhaus, head of the department of hematology and Medical Oncology at Jena University Hospital in Germany, said: “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.”
The EC’s decision 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.