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FDA grants orphan drug designation to Opna Bio's zavabresib for the treatment of myelofibrosis, highlighting its potential as a transformative therapy. This designation provides significant development incentives and is supported by promising early clinical data from its combination trial with ruxolitinib.

The announcement of the FDA’s ... therapy for MF. This regulatory milestone not only provides significant development incentives but also highlights the promising early clinical data from the zavabresib and ruxolitinib combination trial. For Opna Bio, this designation reinforces a commitment to addressing critical unmet needs in rare oncology through novel, targeted treatments... The announcement of the FDA’s Orphan Drug Designation for zavabresib underscores its potential as a transformative therapy for MF. This regulatory milestone not only provides significant development incentives but also highlights the promising early clinical data from the zavabresib and ruxolitinib combination trial. For Opna Bio, this designation reinforces a commitment to addressing critical unmet needs in rare oncology through novel, targeted treatments. The FDA granted Orphan Drug Designation to Opna Bio's zavabresib, a bromodomain and extra-terminal inhibitor, for treating myelofibrosis. “We believe that selective BET inhibition alongside JAK inhibition offers a promising new therapeutic approach for patients with myelofibrosis. We are further encouraged by recent positive meetings with the FDA to continue to test zavabresib in additional clinical studies,” Diaz concluded. This article originally appeared on Rare Disease Advisor ... Opna Bio announces orphan drug designation granted to OPN-2853 (zavabresib) for the treatment of myelofibrosis. This designation marks a significant regulatory milestone and underscores this promising treatment approach, emphasizing the unmet need for better therapies. MF is a rare, serious blood cancer involving bone marrow scarring that disrupts normal blood cell production, leading to anemia, fatigue, and splenomegaly. It affects around 25,000 people in the US. Current standard treatment often includes JAK inhibitors like ruxolitinib, but many patients eventually become resistant. Early evidence from an ongoing investigator-sponsored phase 1 trial (PROMise) combining zavabresib with ruxolitinib showed spleen length reduction of 50% or more in many patients previously unresponsive to ruxolitinib alone.

Treatment & Clinical News9 months agoRead at source →

Original title: “FDA Grants Orphan Drug Status to Zavabresib for Myelofibrosis Therapy - Hematology Advisor”

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