FDA’s orphan drug designations increase for myelofibrosis drugs
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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.
Clinical Trials Show Targeted Drug Diminishes Suffering of Myelofibrosis Patients
The drug, pacritinib, can reduce the suffering caused by myelofibrosis. But scientists are unsure if pacritinib has an impact on survival.
The drug, pacritinib, can reduce the suffering caused by myelofibrosis. But scientists are unsure if pacritinib has an impact on survival.
‘The surge in FDA ODD awards reflects a concerted effort to develop novel myelofibrosis drugs without anemia-inducing mechanisms. #orphandrug #cancertreatment #fda’
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"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.
Acute Lymphoblastic Leukemia
Acute lymphoblastic leukemia (ALL) is the cancer of white blood cells characterized by excess lymphoblasts. It is the most common of all childhood cancers and can be fatal in weeks if left untreated.
Acute lymphoblastic leukemia (ALL) is the cancer of white blood cells characterized by excess lymphoblasts. It is the most common of all childhood cancers and can be fatal in weeks if left untreated.
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)
Basics of Rare Diseases
Rare or orphan diseases are least understood of all chronic diseases. They are more common than projected and kill more people than cancer and HIV together.
Rare or orphan diseases are least understood of all chronic diseases. They are more common than projected and kill more people than cancer and HIV together.
Orphan Drugs Deserve Insurance Coverage
There should be an ethical framework to guide coverage and reimbursement decisions for expensive orphan drugs, which has dramatically increased since the passage of the Orphan Drug Act of 1983.
There should be an ethical framework to guide coverage and reimbursement decisions for expensive orphan drugs, which has dramatically increased since the passage of the Orphan Drug Act of 1983.
