FDA OKs Vonjo for Certain Myelofibrosis Patients


Vonjo is the first treatment first approved therapy that specifically addresses the needs of patients with cytopenic myelofibrosis.

FDA cleared CTI BioPharma’s Vonjo (pacritinib), the first approved therapy that specifically addresses the needs of patients with cytopenic myelofibrosis, the pharma maker said in a news release.

Vonio is indicated for adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L.

The medication will be available to patients by mid-March, according to Adam R. Craig, M.D., Ph.D., president and CEO of CTI Biopharma.

FDA’s approval of Vonjo “establishes a new standard of care for myelofibrosis patients suffering from cytopenic myelofibrosis," said John Mascarenhas, M.D., associate professor at the Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York.

Myelofibrosis with severe thrombocytopenia, defined as blood platelet counts below 50 × 109/L, results in poor survival outcomes coupled with debilitating symptoms, Mascarenhas noted. “Limited treatment options have rendered this disease as an area of urgent unmet medical need. I am pleased to see that a new, efficacious and safe treatment option is now available for these patients,” he said.

There are around 21,000 patients with myelofibrosis, two-thirds of whom have cytopenias (thrombocytopenia or anemia), commonly resulting from the toxicity of other approved therapies, according to Mascarenhas.

Vonjo is a novel oral kinase inhibitor with specificity for JAK2 and IRAK1, without inhibiting JAK1. The recommended dosage is 200 mg orally twice daily.

The FDA’s accelerated approval is based on efficacy results from the pivotal Phase 3 PERSIST-2 study of Vonjo in patients with myelofibrosis (platelet counts less than or equal to 100 × 109/L).

In the cohort of patients with baseline platelet counts below 50 × 109/L who were treated with pacritinib 200 mg BD, 29% of patients had a reduction in spleen volume of at least 35% compared to 3% of patients receiving best available therapy, which included ruxolitinib (brand names Jakafi and Jakavi).

To fulfill its post-approval requirement, CTI plans to complete the PACIFICA trial, and expects results in mid-2025.

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