FDA Grants Accelerated Approval of Scemblix for Newly-Diagnosed CML

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Novartis announced yesterday the accelerated FDA approval of Scemblix (asciminib) to treat adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP).

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. Patients with private insurance can pay as little as $0 using a Co-pay Plus offer with a $15,000 annual limit. Financial assistance for patients is available by phone or online portal.

The expanded indication in Ph+ CML-CP increases the eligible patient population for asciminib by approximately four times, including both newly diagnosed and previously treated adults.

Most adults are diagnosed with CML in the first phase, also called the chronic phase. The disease is caused when pieces of chromosomes 9 and 22 break off and swap places, creating an abnormal the fusion gene BCR-ABL1, which begins to produce an abnormal protein called BCR-ABL. This protein then sends too many signals to the bone marrow and create too many immature white blood cells.

Asciminib is the first CML treatment that specifically targets the ABL myristoyl pocket, distinct from the ATP-binding site targeted by current TKIs. It is approved in the United States and over 75 countries globally for certain CML patient populations.

While TKIs have transformed CML into a chronic condition, many patients still struggle to meet treatment goals or tolerate side effects. Nearly half of CML patients fail to achieve major molecular response, and almost 25% discontinue or switch therapies within the first year.

The approval is based on data from the phase 3 ASC4FIRST trial, which showed asciminib demonstrated superior major molecular response (MMR) rates at 48 weeks compared with investigator-selected standard of care TKIs and imatinib alone. 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.

Asciminib also exhibited a favorable safety and tolerability profile. Patients had 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.

“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,” Lee Greenberger, Ph.D., CSO at The Leukemia & Lymphoma Society said in the news release. “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.”