Not only has CTI BioPharma’s drug Vonjo (pacritinib) already broken new ground by being the first treatment approved for intermediate and high-risk myelofibrosis patients with low platelet counts (below 50×10⁹ per liter of blood), key opinion leaders interviewed by GlobalData note that the drug has promise as a treatment for patients with moderate platelet counts (between 50×10⁹ and 100×10⁹ per liter). The leading data and analytics company highlights that Vonjo’s ability to target such critically underserved and distinct patient populations mean its annual sales are set to reach $235 million by 2027, despite upcoming market competition.

Myelofibrosis is a rare blood cancer that causes scarring in the bone marrow, which makes it more difficult to produce red blood cells. A typical blood platelet count for a healthy adult is between 150×10⁹ to 400×10⁹ per liter. Unchecked low levels, also known as thrombocytopenia, can cause life-threatening internal bleeding, and patients with thrombocytopenia often require blood transfusions, which is a major pain point for healthcare providers and patients alike.

Sam Warburton, Oncology and Hematology Analyst at GlobalData, comments: “Vonjo is uniquely positioned to establish a potential new standard of care for what is a critically underserved patient population in myelofibrosis, which previously had no approved treatment options. Further, the potential for expansion into other populations has influenced our sales estimations.”

While Vonjo is noted to have great off-label potential in patients with higher platelet counts, the drug will begin to face competition from the likes of Jakafi (ruxolitinib).

Warburton continues: “While Vonjo’s Phase III PERSIST-2 trial showed that the drug demonstrated superior splenic volume reduction and total symptom reduction in patients with platelet count less than or equal to 100 × 10⁹ per liter*, Jakafi is well established as a first-line treatment for myelofibrosis and benefits from first-to-market advantage. Vonjo will need to gain clinician familiarity and confidence as well as demonstrating superiority to Jakafi in this patient population.”

Several other pipeline agents are in pivotal Phase III trials, however, Vonjo’s market share is secured by targeting a patient population distinct to those of other pipeline agents.

Warburton continues: “Looking more widely, the myelofibrosis treatment paradigm is set to experience significant developments over the next five years, with several additional FDA approvals soon anticipated—Sierra Oncology’s momelotinib later this year, for example.”

GlobalData’s Pharma Drug Sales & Forecast database integrates proprietary company and prescription drug sales data with analyst consensus forecasts. The database provides annual and quarterly historical drug sales, analyst consensus forecasts provided by drug region and by indication for innovator, generic and biosimilar drugs. It also contains GlobalData’s patient-based forecasts based on a proprietary methodology. GlobalData’s Pharma Drug Sales & Forecast database can also be fully integrated with our NPV models for forecasting revenues.