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  • GSK¡¯s momelotinib receives orphan drug designation in KOR
  • by Eo, Yun-Ho | translator Kim, Jung-Ju | Oct 10, 2023 05:26am
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 inhibit
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