FDA Boosts Orphan Drug Designations for Myelofibrosis Treatments
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. ()
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, a leading data and analytics company 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.
‘The surge in FDA ODD awards reflects a concerted effort to develop novel myelofibrosis drugs without anemia-inducing mechanisms. #orphandrug #cancertreatment #fda’
"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.
Since 2020, the FDA has doubled its ODD awards compared to the previous four years. In 2022, four ODDs were awarded, doubling the number seen in the peak years of 2019 and 2020, both with two ODDs.
Morley highlights the shift in FDA regulatory focus away from anemia-inducing JAK inhibition, evident in the diversity of mechanisms among the 12 myelofibrosis drugs awarded ODDs. Only two of these drugs act via JAK inhibition, indicating a shift away from this mechanism.
Merck & Co's Reblozyl (luspatercept), awarded ODD in January 2020, targets growth/differentiation factor (GFD) 11 and 8 inhibition. This drug, currently in Phase III for anemia-associated myelofibrosis, features a different mechanism.
Reference:
- FDA��s orphan drug designations increase for myelofibrosis drugs - (https:www.globaldata.com/media/pharma/fdas-orphan-drug-designations-increase-myelofibrosis-drugs-reveals-globaldata)