Myelofibrosis drug 'Omjjara' attempts at reimb listing

Has been directly compared with Jakavi, and improved validation

Blocks all three key signaling pathways

'Omjjara,' a targeted treatment for myelofibrosis, attempts to be added to the National Health Insurance reimbursement list.

 

According to industry sources, GSK Korea submitted a reimbursement application for its new myelofibrosis drug, Omjjara (momelotinib), at the end of last year, and is awaiting to be considered for the Health Insurance Review and Assessment Service (HIRA)'s Cancer Disease Review Committee (CDRC).

 

The company has applied for the indication to treat 'adult patients with intermediate or high-risk myelofibrosis who have anemia.' The drug has approved indications to treat primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis.

 

Unlike single-target drugs, Omjjara is a multi-target drug that blocks three key signaling pathways.

 

It is expected to have a significant treatment effect.

 

This drug has an inhibitory ability along three key signaling pathways, including JAK1 and JAK2 proteins that are targets of previous drugs, along with activin A receptor type 1 (ACVR1).

 

The recommended dosage is once daily oral administration of 200 mg, and it can be taken regardless of meal intake.

 

Myelofibrosis is a rare blood cancer in which the buildup of fibrosis in the bone marrow causes symptoms such as anemia, thrombocytopenia, and enlarged spleen and liver.

 

It occurs in 1 out of 100,000 worldwide.

 

In South Korea, it has been reported that as of 2023, about 2292 patients have received inpatient and outpatient treatments.

 

Patients who have anemic symptoms have poor prognosis after treatment, and the problem is most patients experience anemia.

 

According to the research, 87% of myelofibrosis patients were reported to be anemic at the time of diagnosis.

 

In another study, 46% of patients required blood transfusion after one year of diagnosis.

 

Typically, anemia in myelofibrosis patients increases the death risk by twofold compared to other prognostic factors, such as age, increased white blood cells, and symptoms throughout the body.

 

The clinical efficacy and safety profile of Omjjara were confirmed through the Phase 3 'SIMPLIFY-1 study' and 'MOMENTUM study.' The studies confirmed that the drug improved key symptoms, such as enlarged spleen, in adult myelofibrosis patients and reduced dependence on blood transfusions in anemic patients.

 

Firstly, the SIMPLIFY-1 study directly compared Omjjara to 'Jakavi (ruxolitinib)' in 432 adult myelofibrosis patients without prior JAK inhibitor therapy.

 

Subgroup analysis was performed in patients with anemia.

 

The results showed that Omjjara was confirmed to be non-inferior to ruxolitinib in terms of the primary efficacy endpoint, which was a spleen volume at week 24 (≥35% reduction).

 

Non-inferiority was not identified in the total symptom improvement score.

 

The rate of transfusion independence in each patient group was confirmed.

 

The rate of patients with transfusion independence in the Omjjara treatment group was 66.5%, and those in the ruxolitinib treatment group were 49.3%, showing that the Omjjara treatment group had significantly lower transfusion independence.

 

In the MOMENTUM study, which was another basis of approval, the efficacy and safety of Omjjara to 'danazol' were compared in 195 adult patients with myelofibrosis who had previously undergone JAK inhibitor therapy, exhibited symptoms, and had anemia.

 

All study participants had been treated with ruxolitinib before, and 4.6% of the patients received 'fedratinib.' The common primary efficacy endpoint was the percentage of patients with a 50% or greater reduction in the Total Symptom Score (TSS) and transfusion independence at week 24.