CPAC ‘Scemblix’s orphan drug designation is valid’

Treatment for chronic myeloid leukemia

Iclusig is the only treatment available in Korea

The CPAC deems drug Scemblix’s designation valid based on its improved safety and efficacy compared with its alternative

Pic of Scemblix

The next-generation chronic myeloid leukemia drug ‘Scemblix (asciminib)’ is expected to be available for adult patients with chronic myeloid leukemia (CML) with the T315I mutation or Philadelphia chromosome-positive acute lymphocytic leukemia (Ph+ ALL) with the T315I mutation.

 

According to the minutes of the Central Pharmaceutical Affairs Council that were disclosed by the Ministry of Food and Drug Safety on the 11th, the CPAC had discussed Scemblix’s orphan drug designation agenda at its last meeting.

 

In Korea, Scemblix is currently only approved as a treatment for adult patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in the chronic phase previously treated with two or more tyrosine kinase inhibitors (TKIs).

 

Iclusig(ponatinib) is currently the only treatment option available for the "patients with T315I-positive CML," for which the CPAC has been discussing Scemblix’s orphan drug designation.

 

Iclusig was designated as an orphan drug for “patients with T315I-positive CML” and “adult patients with chronic, accelerated, or acute phase chronic myeloid leukemia (CML) or Philadelphia chromosome-positive acute lymphocytic leukemia (Ph+ ALL) that is resistant or intolerant to other tyrosine kinase inhibitors (TKIs).

 

Regarding the designation decision, a CPAC member stated, "Scemblixworks in patients who are not sufficiently responding to treatment with other alternatives and in patients for whom alternative medicines are not available.

 

Therefore, it is the only available alternative that offers improvement for difficult-to-treat patients.” According to data submitted by Novartis, Scemblix is a valid and viable alternative treatment option for CML patients with the T315I mutation, regardless of their Iclusig use.

 

Also, the reported adverse events were manageable with dose adjustment, discontinuation, combination medications or supportive care, and a favorable safety profile compared to alternatives.

 

The CPAC member said, "When considering the number of patients, prevalence, and safety and efficacy assessments, the results justify the drug’s orphan drug designation.” Another CPAC member said, “Given that the indication is limited to chronic myeloid leukemia and the submitted study is a Phase I study, its efficacy cannot be directly compared with Iclusig.

 

However, in the submitted Phase I study, 38.5% of patients who have been previously treated with Iclusig achieved major molecular response (MMR).

 

Given that Iclusig is the only treatment option for patients with T315I-mutant Ph+ CML, we believe that an orphan drug designation can provide patients and their physicians with more options.” Some noted that the definition of “significant” in the orphan drug designation criteria typically implies a statistically significant difference and that it would be unreasonable to conclude that a drug has significantly improved safety or efficacy based on the results of a Phase I trial.

 

One member noted, "Given the limited number of CML patients with the T315I mutation, it will be some time before a direct comparison study is published.” However, the majority of committee members agreed that Scemblix is a viable treatment option for patients with leukemia that has become resistant to other therapies and that its orphan drug designation is valid.