Concerns regarding the suitability of current FDA-approved myelofibrosis treatments due to their association with anemia have prompted significant actions. The FDA has notably increased the allotment of orphan drug designations (ODDs) for myelofibrosis-related medications in recent years. GlobalData, a leading data and analytics company, reports a total of 11 ODDs granted since 2014, with eight awarded after 2020.

All About Myelofibrosis

Myelofibrosis, an aggressive form of blood cancer characterized by scar tissue accumulation in the bone marrow, disrupts the body’s normal blood cell production. This rare condition has an annual reporting rate of approximately 1.5 cases per 100,000 people in the US, according to the National Organization for Rare Disorders (NORD).

Jasper Morley, Drugs Intelligence Analyst at GlobalData, notes the heightened FDA focus on exploring alternative treatment mechanisms to address myelofibrosis’s unmet needs. Presently, the four FDA-approved innovator drugs for myelofibrosis are all Janus kinase (JAK) inhibitors.

“While effective in treating myelofibrosis, JAK inhibitors are known for myelosuppression, worsening anemia symptoms by reducing oxygen circulation. Given that roughly half of patients are anemic at diagnosis, there’s a vital need for myelofibrosis treatments benefiting this patient population,” Morley emphasizes.