Novartis: Scemblix approved in the EU for adults with chronic myeloid leukemia

Basel: Novartis today announced that the European Commission (EC) has approved 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).

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"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 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; and it is applicable to all 27 European Union member states plus Iceland, Norway and Liechtenstein. The 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 (37.6%, 95% CI: 29.99-45.65) compared with Bosulif (15.8%, 95% CI: 8.43-25.96) and the discontinuation rate due to adverse reactions was 7.7% for Scemblix and 26.3% for Bosulif. These data were shared as oral presentations during the annual meetings of the American Society for Clinical Oncology (ASCO) and the European Hematology Association (EHA) in June 20223,4. Based on all patients exposed to Scemblix in the ASCEMBL study and in the phase I study, the most common (incidence ≥ 20%) adverse reactions in patients receiving asciminib were musculoskeletal pain (37.1%), upper respiratory tract infections (28.1%), thrombocytopenia (27.5%), fatigue (27.2%), headache (24.2%), arthralgia (21.6%), increased pancreatic enzymes (21.3%), abdominal pain (21.3%), diarrhoea (20.5%) and nausea (20.2%).

"Approval of Scemblix from the European Commission is a critical milestone to help bring this novel treatment to patients living with CML in Europe," said Haseeb Ahmad, President, Europe Innovative Medicines, Novartis. "Building on more than twenty years of innovation in CML, we are excited by the potential to once again transform the standard of care for more patients around the world."

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.

Scemblix is the first CML treatment that acts as a STAMP inhibitor, specifically targeting the ABL myristoyl pocket. This novel mechanism of action 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 leukemic cells1,2,14-20.

Scemblix represents an important development for patients who experience resistance and/or intolerance to currently available TKI therapies, and it is being studied across multiple treatment lines for CML-CP, both as a monotherapy and in combination1,14-28. Specifically, the ASC4FIRST Phase III study (NCT04971226) evaluates Scemblix in newly diagnosed adult patients with Ph+ CML-CP vs. an investigator-selected TKI.

Novartis has initiated regulatory filings for Scemblix in multiple countries and regions across the globe. In October 2021, the US FDA granted accelerated approval of Scemblix for adult patients with Ph+ CML-CP, previously treated with two or more TKIs based on MMR rate at 24 weeks, and full approval for adult patients with Ph+ CML-CP with the T315I mutation. In accordance with the Accelerated Approval Program, continued approval for the first indication may be contingent upon verification and description of clinical benefit from confirmatory evidence. The longer-term, 96-week efficacy and safety data have been shared with the FDA and are currently under evaluation through a priority review.

Scemblix has received approval in several countries outside the US, including Japan, Switzerland, and the United Kingdom, for adult patients with Ph+ CML-CP with resistance or intolerance to at least two or more previous therapies.

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