GSK’s momelotinib receives orphan drug designation in KOR

GSK’s new myelofibrosis drug

Inhibits 3 signaling pathways for JAK1·JAK2·ACVR1

The new myelofibrosis drug momelotinib has been designated as an orphan drug in Korea.

 

The Ministry of Food and Drug Safety announced so through an orphan drug designation notice issued on the 6th.

 

Momelotinib is a treatment for myelofibrosis that blocks the major signaling pathways for JAK1, JAK2, and activin A receptor type 1 (ACVR1).

 

The drug was a candidate drug for Sierra Oncology, but was acquired by GSK last year for USD 1.9 billion (KRW 2.56 trillion) and recently received final approval from the US Food and Drug Administration.

 

Based on its mechanism of action, momelotinib is known to improve systemic symptoms and splenomegaly (enlarged spleen) by inhibiting JAK1 and JAK2, while directly inhibiting ACVR1 to reduce the level of circulating hepcidin, which contributes to anemia.

 

Results of the Phase III MOMENTUM trial conducted on patients with myelofibrosis that was published in the global medical journal, The Lancet, showed that the drug demonstrated significant results in myelofibrosis patients with anemia, where treatment limitations had previously existed.

 

The MOMENTUM study investigated momelotinib versus danazol in 195 patients with myelofibrosis who were symptomatic and anemic and had been previously treated with an approved JAK inhibitor at 107 sites in 21 countries.

 

The primary endpoint was the total symptom score response (≥50% reduction from baseline) rate assessed through MFSAF(Myelofibrosis Symptom Assessment Form).

 

Study results showed that 25% of patients treated with momelotinib met the primary endpoint as compared with 9% in the danazol arm.

 

Also, at 24 weeks, 40% of patients in the momelotinib arm achieved a spleen volume reduction of at least 25% from baseline at week 24, 35% of which did not require transfusion at week 24.

 

The levels were 6% and 17% respectively for the danazol arm.

 

Meanwhile, myelofibrosis is a disease in which fibrous scar tissue replaces blood-producing cells in the bone marrow, and is characterized by abnormally shaped red blood cells, anemia, and an enlarged spleen.