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FDA Approves Scemblix (asciminib) for Newly Diagnosed Chronic Myeloid Leukemia

• The FDA has granted accelerated approval to Scemblix (asciminib) for adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). • Asciminib is the first CML treatment that specifically targets the ABL myristoyl pocket, offering a novel mechanism of action compared to traditional tyrosine kinase inhibitors (TKIs). • The approval is based on the Phase 3 ASC4FIRST trial, which demonstrated superior major molecular response (MMR) rates at 48 weeks compared to standard-of-care TKIs. • Scemblix also exhibited a favorable safety and tolerability profile, with fewer treatment-related adverse reactions compared to standard TKIs and imatinib.

The FDA has granted accelerated approval to Scemblix (asciminib) for the treatment of adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). This approval marks a significant advancement in CML treatment, offering a new therapeutic option with a distinct mechanism of action.

Novel Mechanism of Action

Asciminib is the first CML treatment to specifically target the ABL myristoyl pocket, a different site than the ATP-binding site targeted by current TKIs. This unique mechanism provides a potential advantage in overcoming resistance and improving treatment outcomes for patients with CML. The expanded indication increases the eligible patient population for asciminib by approximately four times, including both newly diagnosed and previously treated adults.

Clinical Trial Data

The approval was based on data from the Phase 3 ASC4FIRST trial, which compared asciminib to investigator-selected standard-of-care TKIs and imatinib alone. The results showed that asciminib demonstrated superior major molecular response (MMR) rates at 48 weeks. Nearly 20% more patients on asciminib achieved MMR versus standard TKIs (68% vs. 49%), and 30% more achieved MMR versus imatinib alone (69% vs. 40%). The study also includes the phase 2 ASC2ESCALATE trial in previously treated Ph+ CML-CP patients.

Safety and Tolerability

In addition to its efficacy, asciminib also exhibited a favorable safety and tolerability profile. Patients experienced fewer treatment-related grade 3 or higher adverse reactions (25.5% vs. 33% and 42%), dose reductions (6% vs. 14% and 24%), and adverse reactions leading to treatment discontinuation (4.5% vs. 11% and 9.8%) compared to standard TKIs and imatinib.

Impact on CML Treatment

"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," said Lee Greenberger, Ph.D., CSO at The Leukemia & Lymphoma Society. "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."
This approval offers a promising new option for patients newly diagnosed with CML, potentially leading to improved outcomes and a better quality of life. Asciminib is administered orally, with dosing dependent on prior treatment history and specific mutations. Patients previously treated with at least two other tyrosine kinase inhibitors (TKIs) can take 80 mg of asciminib orally daily, according to prescribing information. Patients with the T315I mutation can take 200 mg daily.
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Reference News

[1]
FDA Grants Accelerated Approval of Scemblix for Newly-Diagnosed CML
managedhealthcareexecutive.com · Oct 31, 2024

Novartis announced FDA approval of Scemblix (asciminib) for newly diagnosed Ph+ CML-CP, expanding eligible patient popul...

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