Scemblix now OK’d in Canada for treating newly diagnosed CML
Health Canada expands therapy's approval to more adults with leukemia
More people in Canada with the rare blood cancer Ph+ CML — fully, Philadelphia chromosome-positive chronic myeloid leukemia — in the chronic phase are now eligible for treatment with Novartis’ Scemblix (asciminib tablets).
Health Canada has expanded its approval of Scemblix, a therapy for adults with this type of leukemia, which previously had been indicated only for patients with Ph+ CML who had received at least two tyrosine kinase inhibitors (TKIs), a class of anticancer treatment.
With the new indication, Scemblix is now also authorized for patients who are newly diagnosed or who have received only one prior TKI.
“We are proud that Health Canada has expanded its approval of Scemblix, making it a new option for all Canadians with chronic myeloid leukemia, whether they are newly diagnosed or have been previously treated,” Mark Vineis, country president of Novartis Canada, said in a company press release, which noted that nearly 25% of CML patients now change treatments within the first year of use.
“This approval means patients and their physicians now have more choices when deciding on the best course of treatment, offering renewed hope for individuals living with CML, their families, and the healthcare teams dedicated to their care,” Vineis said.
Novartis noted that the clinical effectiveness, safety, and cost-effectiveness of Scemblix are currently being reviewed by Canada’s Drug Agency (CDA) and Institut National d’Excellence en Santé et Services Sociaux (INESSS) — the agencies that help determine coverage by Canada’s national insurance programs. Novartis said it looks forward to sharing their recommendations, once available, with the community.
Scemblix expanded approval welcomed by community
Dennis Kim, MD, PhD, a professor at Princess Margaret Cancer Centre Cancer in Ontario, said the expanded approval of Scemblix in Canada “represents a significant step forward, expanding treatment options for CML patients.”
“Having a diverse range of therapies available allows care teams to keep the unique needs of the patient at the centre of treatment plans, optimizing outcomes,” Kim said. “The ability to prescribe [Scemblix] to newly diagnosed and previously treated patients offers a promising new pathway in our efforts to manage this complex disease effectively and safely.”
The expanded approval also was welcomed by Lisa Machado, founder of the Canadian CML Network.
As a person living with CML and an advocate, I am hopeful that expanded access to this innovative treatment option will offer patients not only continued positive outcomes, but also provide the opportunity to maintain a quality of life that meets their expectations as they and their families navigate the complexities of CML management.
“The approval of a new treatment option for newly diagnosed and previously treated Canadians living with CML is an important milestone,” Machado said. “As a person living with CML and an advocate, I am hopeful that expanded access to this innovative treatment option will offer patients not only continued positive outcomes, but also provide the opportunity to maintain a quality of life that meets their expectations as they and their families navigate the complexities of CML management.”
CML is a type of blood cancer caused by a specific genetic mutation known as the Philadelphia chromosome. This mutation creates an abnormal fusion gene called BCR-ABL1, which in turn produces an abnormal enzyme (a tyrosine kinase) that signals cells to grow and divide uncontrollably, driving the development of CML. Scemblix is a targeted therapy designed to block the activity of this abnormal enzyme, helping to slow or stop the growth of leukemia cells.
Blood cancer occurs in 3 phases
This blood cancer occurs in three phases: chronic, accelerated, and blast. Most patients are diagnosed in the chronic phase, which is the earliest phase. With appropriate treatment, it is often possible to avoid advancing to further stages. However, according to Novartis, approximately half of people with CML do not meet efficacy milestones with current standard of care treatment.
The new Health Canada approval was based mainly on data from a Phase 3 clinical trial called ASC4FIRST (NCT04971226). The study enrolled more than 400 people with newly diagnosed Ph+ CML in the chronic phase. The participants were randomly assigned to receive Scemblix or their doctor’s choice of a TKI.
The results showed that, after about a year, the number of patients with a major molecular response — meaning very low levels of the BCR-ABL1 in the blood or bone marrow — was significantly higher for patients given Scemblix (67.7% vs. 49%). Data also suggested that, with Scemblix, serious side effects and side effects leading to treatment discontinuation were less common.
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