Novartis announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion and recommended granting marketing authorization for Scemblix® (asciminib) for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP), previously treated with two or more tyrosine kinase inhibitors (TKIs). If approved, Scemblix will be the first CML treatment in Europe that works by specifically targeting the ABL myristoyl pocket (also known as a STAMP inhibitor in scientific literature), representing an important therapeutic advancement for patients who experience intolerance and/or resistance to currently available TKI therapies.
It is estimated that, every year, more than 6,300 people will be diagnosed with CML in Europe. While many patients will benefit from available TKI therapies, a significant proportion may experience intolerance or resistance to these treatments. In an analysis of patients with CML treated with two prior TKIs, approximately 55% reported intolerance to previous treatment; and a pooled analysis of patients in the second-line setting revealed that up to 70% are unable to achieve major molecular response (MMR) within two years of follow-up.
"Although CML treatments have advanced over the last 20 years, many patients continue to experience side effects and resistance to treatment, affecting their quality of life and putting them at risk of disease progression or even death," says Dr. Andreas Hochhaus, Head of the Department of Hematology and Medical Oncology at Jena University Hospital in Germany. "If approved, the novel mechanism of action of Scemblix brings us another option to combat these challenges faced by patients — offering new hope in the management of their disease."
The positive CHMP opinion for Scemblix is based on results from the pivotal Phase III ASCEMBL trial, which showed a near doubling of MMR rate for patients treated with Scemblix vs. Bosulif®* (bosutinib) (25.5% vs. 13.2%) at 24-weeks, with a more than three times lower discontinuation rate due to adverse reactions (5.8% vs. 21.1%). The most common (incidence ≥ 20%) adverse reactions reported in this analysis were thrombocytopenia (29.5%) and neutropenia (23.1%) in the Scemblix arm; and diarrhea (71.1%), nausea (46.1%), increased ALT (28.9%), vomiting (26.3%), rash (23.7%), increased AST (21.1%) and neutropenia (21.1%) in the Bosulif arm.
These results were confirmed in longer-term follow-up, where the MMR rate at week 96 was more than double with Scemblix (37.6%, 95% CI: 29.99-45.65) compared with Bosulif (15.8%, 95% CI: 8.43-25.96). This data was shared at oral presentations during the American Society for Clinical Oncology (ASCO) and the European Hematology Association (EHA) annual meetings in June 2022.
“We are pleased with the recommendation of Scemblix and hope to offer patients living with CML in Europe timely access to this innovative therapy, if approved,” said Haseeb Ahmad, President, Europe, Novartis. “We’ve worked relentlessly to improve CML care over the past two decades, and must seize this opportunity to help patients in need achieve better outcomes. With the strong clinical results seen to-date, we believe we have the potential to transform the standard of care in CML yet again with Scemblix.”
The CHMP recommended approval of Scemblix in CML will be referred to the European Commission (EC). The EC will review the CHMP recommendations and deliver a final decision in the coming months.
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