FDA Grants Accelerated Approval to Vonjo for Myelofibrosis With Severe Thrombocytopenia

The Food and Drug Administration’s greenlight of Vonjo marks the first approved therapy to address the needs of patients with cytopenic myelofibrosis, a type of myeloproliferative neoplasm.

The Food and Drug Administration (FDA) on Monday evening approved Vonjo (pacritinib) to treat adults with intermediate- or high-risk primary or secondary myelofibrosis, a type of blood cancer, according to a news release from the agent’s manufacturer, CTI BioParma Corp.

“(The) approval of Vonjo establishes a new standard of care for myelofibrosis patients suffering from cytopenic myelofibrosis,” Dr. John Mascarenhas, an associate professor of Medicine and Hematology and Medical Oncology at Tisch Cancer Institute at Mount Sinai in New York, said in the release. “Myelofibrosis with severe thrombocytopenia, defined as blood platelet counts below 50 × 109/L, has been shown to result in poor survival outcomes coupled with debilitating symptoms. 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.”

The agency’s decision to approve Vonjo — a novel oral kinase inhibitor — is based on data from the phase 3 PERSIST-2 trial, which demonstrated that the study drug delivered twice a day at a dose of 200 mg each time resulted in a reduction in spleen volume of at least 35% in 29% of patients. This was compared to 3% of patients who received best available therapy.

The trial demonstrated that the most common side effects (defined as occurring in more than 20% of patients) following twice daily Vonjo were diarrhea, nausea, anemia, peripheral edema (swelling of lower legs or hands) and thrombocytopenia (low platelet counts). Serious side effects included, but were not limited to, pneumonia, cardiac failure and skin cancer.

The FDA requires CTI to continue to further evaluate Vonjo in a confirmatory trial, which the manufacturer noted it plans to via the PACIFICA trial which is anticipated to have results in mid-2025.

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