Expanded indication for Iclusig as a first-line treatment

of Ph+ ALL

After receiving expedited FDA approval in May last year, it obtained expanded indication from the Ministry of Food and Drug Safety in KOR

A new option for three or more lines of treatment for Ph+ALL in combination with chemotherapy

Product photo of Iclusig

On the 16th, Korea Otsuka Pharmaceutical (CEO Sung-ho Moon) announced that Iclusig (ingredient name: ponatinib) can now be used in combination with chemotherapy for treating adult patients with Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ ALL).

 

This expanded indication has expanded Iclusig's use of scope from a third-line or higher treatment for Ph+ ALL to Ph+ ALL first-line treatment.

 

Previously, Iclusig was approved for the treatment of adult patients with chronic, accelerated, or blast phase chronic myeloid leukemia (CML) or Ph+ ALL who were refractory to other tyrosine kinase inhibitors (TKIs), as well as for the treatment of adult patients with T315I-positive chronic, accelerated, or blast phase CML or T315I-positive Ph+ ALL.

 

This expansion of indications follows the expedited approval by the U.S.

 

FDA in March 2024 for combining therapy comprised of Iclusig and chemotherapy to treat newly diagnosed Ph+ ALL patients.

 

It has also led to an expansion of its indications in South Korea.

 

Through this, Iclusig can now be used in Korea as a first-line treatment for adult Ph+ ALL patients in combination with chemotherapy for up to 20 cycles.

 

The PhALLCON trial, which served as the basis for this approval, was a Phase 3 trial (randomized, open-label) conducted at 77 institutions across 17 countries.

 

The study evaluated the efficacy and safety of the combination of ponatinib (brand name: Iclusig) with low-intensity chemotherapy versus the combination of the first-generation TKI imatinib (brand name Glivec) with low-intensity chemotherapy in 245 newly diagnosed adult Ph+ ALL patients.

 

Study results showed that the primary evaluation endpoint, the MRD-negative complete remission (MRD-neg CR) rate at the end of induction therapy (12 weeks), was 34.4% in the Iclusig group, approximately twice as high as the 16.7% observed in the imatinib group.

 

Furthermore, the median progression-free survival (PFS) was significantly longer in the Iclusig group at 20.0 months compared to 7.9 months in the imatinib group.

 

In terms of safety, Iclusig demonstrated manageable tolerability.

 

The treatment discontinuation rate due to adverse events was reported to be 12% (n=20/164) patients in the Iclusig group and 12% (n=10/81) in the imatinib group.

 

The incidence of treatment-related serious adverse events was 20.9% (n=34/163) in the Iclusig group and 19.8% (n=16/81) in the imatinib group.

 

Korea Otsuka Pharmaceutical representative stated, "Iclusig demonstrated superior MRD-neg CR and stable drug tolerability compared to the conventional therapy, such as the first-generation TKI, when used as a first-line treatment for patients with Ph+ ALL," and added, "Based on this expansion of indication, Iclusig, which was used for three or more line of treatments, is available as a first-line treatment option."