FDA grants accelerated approval to Novartis’ Scemblix as a first-line leukemia therapy

Novartis’ Scemblix (asciminib) has received accelerated approval by the US FDA as a treatment for adult patients with newly diagnosed Philadelphia chromosome-positive myeloid leukemia in chronic phase (Ph+ CML-CP).

The approval of Scemblix is based on results from the ASC4FIRST phase 3 trial, in which Novartis’ drug was compared to all other standard of care treatments for the condition. After 48 weeks of treatment, Scemblix demonstrated superior rates of major molecular response and fewer reports of adverse reactions compared to imatinib, nilotinib, dasatinib, and bosutinib.

All these standard of care therapies belong to a class of drugs known as tyrosine kinase inhibitors (TKIs). While TKIs have transformed the care of CML, they pose safety challenges and not all patients respond to them. It is estimated that nearly half of patients do not meet the target efficacy goals and almost one in four discontinue or switch treatments within a year.

“Many patients who are newly diagnosed with CML struggle to navigate this chronic condition and may switch or even stop treatment because of side effects that interrupt their daily lives,” said Lee Greenberger, Chief Scientific Officer at The Leukemia & Lymphoma Society. “That’s why approvals of new first-line treatment options are so important. For patients, finding a medicine that’s right for them at the very beginning of treatment may lead to better long-term disease control with fewer side effects.”

Scemblix was first approved by the FDA in 2021 as a treatment for patients with Ph+ CML-CP who had previously been treated with two or more TKIs, as well as patients with the T315I mutation. According to Novartis, the new approval expands the patient population eligible for Scemblix by approximately four times.

“While there are a range of effective TKIs currently available for newly diagnosed patients, clinicians frequently have had to weigh sacrificing either efficacy or tolerability,” said Jorge Cortes, Director of the Georgia Cancer Center. “In the first-of-its-kind ASC4FIRST trial, Scemblix achieved impressive results across all three parameters of efficacy, safety and tolerability versus all standard of care TKIs. This Scemblix data has the potential to be practice-changing.”

The ASC4FIRST trial is still ongoing, with additional data expected after 96 weeks of treatment. The continued approval of Scemblix as a first-line treatment for Ph+ CML-CP will rely on further data reported in the study.