Also known as a STAMP inhibitor, Scemblix is the first FDA-approved CML treatment that works by binding to the ABL myristoyl pocket
Bone marrow aspirate showing acute myeloid leukaemia. (Credit: VashiDonsk/Wikipedia.)
Novartis has received the US Food and Drug Administration (FDA) approval for Scemblix (asciminib) to treat of chronic myeloid leukaemia (CML) in two separate indications.
FDA granted accelerated approval for Scemblix to treat Philadelphia chromosome-positive CML in chronic phase (Ph+ CML-CP), in adults previously treated with two or more tyrosine kinase inhibitors (TKIs).
Also, the US regulatory agency has granted full approval for the treatment of adult patients with Ph+ CML-CP with the T315I mutation.
Scemblix, also known as a STAMP inhibitor, is the first FDA-approved CML treatment that works by binding to the ABL myristoyl pocket.
It provides an important treatment option for patients who experience resistance or intolerance to currently available TKI therapies, said the company.
Novartis Oncology president Susanne Schaffert said: “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.
“We would like to thank all those who have been involved in helping to advance this new and important breakthrough.”
The FDA approval of Scemblix is based on results from the Phase 3 ASCEMBL trial and a Phase 1 study that enrolled patients with Ph+ CML-CP with the T315I mutation.
In patients with Ph+ CML-CP who had experienced resistance or intolerance to at least two TKIs, the ASCEMBL trial showed that treatment using Scemblix nearly doubled the MMR rate compared to Bosulif (bosutinib).
The trial also showed that compared to Bosulif, the proportion of patients who discontinued treatment due to adverse reactions was more than three times lower in case of Scemblix.
The most common adverse reactions include upper respiratory tract infections, musculoskeletal pain, decline in platelet, neutrophil and haemoglobin counts and increase in triglycerides, creatine kinase and alanine aminotransferase (ALT).
Scemblix is currently being studied in various treatment lines for CML-CP, including the Phase 3 ASC4FIRST study, and the company has started regulatory filings in various countries across the world.
Recently, Novartis has received the US FDA priority review for Kymriah (tisagenlecleucel) to treat relapsed or refractory (r/r) follicular lymphoma (FL).