FDA Approves Novartis’ Chronic Myeloid Leukemia Treatment

FDA recently approved Novartis’ Scemblix to treat chronic myeloid leukemia (CML) for adult and pediatric patients with Philadelphia chromosome-positive CML in chronic phase.

Scemblix is the first FDA-approved CML treatment that binds to the ABL myristoyl pocket. The agency based its decision on a Phase 3 ASCEMBL trial and a Phase 1 study that enrolled patients with Philadelphia chromosome-positive CML in chronic phase with a T315l mutation. 

The trial found that Scemblix doubled the major molecular response rate at 24 weeks. And discontinuation rate was over three times lower in the Scemblix group than in the placebo group. 

A Novartis spokesperson explained that continued approval for the first indication is contingent upon verification and description of clinical benefit from confirmatory evidence. 

“Many patients do not respond adequately to at least two available treatments and often experience challenging side effects that add a burden to their daily lives,” Lee Greenberger, chief scientific officer at The Leukemia & Lymphoma Society, said in the announcement.

“The approval of Scemblix may offer hope to patients by addressing gaps in CML care,” Greenberger continued. 

About 15 percent of all leukemia is CML. And about 9,000 individuals in the US a year are diagnosed with the disease. Most of these patients are adults, and 50 percent are older than 64 years of age. 

The standard treatment for chronic phase CML is a TKI, including Gleevac, Tasigna, Sprycel, and Bosulif. 

In a recent analysis, about 55 percent of CML patients treated with two prior TKIs reported an intolerance to previous treatment. And an additional analysis found that 70 percent of patients were unable to achieve major molecular response within two years of follow-up. 

Notably, patients who develop the T315l mutation are resistant to the most available TKI’s. 

FDA’s approval is a vital development for patients who experience resistance or intolerance to currently available therapies. Physicians are currently able to prescribe Scemblix appropriate patients in the US.

“After more than two decades of reimagining CML care, we continue to boldly push the boundaries of innovation to transform the standard-of-care and help even more patients living with this disease,” said Susanne Schaffert, PhD, president of Novartis Oncology.