January 27, 2026
FDA
Myeloproliferative Neoplasms Primary Myelofibrosis News

FDA grants zavabresib orphan drug designation in myelofibrosis

The US Food and Drug Administration has granted orphan drug designation to zavabresib (OPN-2853) for the treatment of myelofibrosis. The generic name of zavabresib for OPN-2853 has also been approved by the International Nonproprietary Names for Pharmaceutical Substances.

The therapy is currently being evaluated in the Phase 1 PROMise study as an add-on to ruxolitinib in patients with myelofibrosis who are no longer responding to ruxolitinib alone.

Data presented at the American Society of Hematology Annual Meeting & Exposition in December 2025 showed that zavabresib was well tolerated and demonstrated a 50% or greater reduction in spleen length in 16 of 26 evaluable patients, according to a press release from Opna Bio, the manufacturer of the therapy.

Watch Adam Mead, MD, on PROMise trial updates in myelofibrosis