According to a story from financialbuzz.com, the biopharmaceutical company Principia Biopharma recently announced that its experimental medical product PRN1008 has received Orphan Drug designation from the US Food and Drug Administration (FDA). PRN1008 received the designation for treating the rare disease immune thrombocytopenic purpura (ITP). Principia is committed to the development of orally active, transformative therapies that address serious unmet medical needs.
About Immune Thrombocytopenia Purpura (ITP)
Immune thrombocytopenia purpura, which is also known as immune thrombocytopenia (ITP), is a condition of low platelet count in the blood without bone marrow abnormalities or other readily apparent cause. ITP is autoimmune in origin, with the immune system mistakenly developing antibodies to platelet cells and destroying them. Symptoms include bruising and petechiae (tiny bruises) forming on the extremities, prolonged bleeding from injuries, heavy menstrual bleeding, bleeding from the gums and mouth, and hematomas. Very low platelet counts increase the risk of potentially fatal complications. There is an increased risk of death for older people with ITP, which usually appears as a chronic condition. Treatment is usually only employed for cases of serious bleeding or very low platelet counts and may include a number of medications, platelet transfusion, and surgery. To learn more about ITP, click here.
About Orphan Drug Designation
Orphan Drug designation is typically reserved for therapies that are designed to treat rare diseases, which the FDA defines as any disease that affects 200,000 people or less. In addition, a drug can only receive the designation if it displays potential advantages in effectiveness or safety in comparison to current treatments. Otherwise is must address an unmet medical need. The designation confers several benefits to the recipient company, such as tax breaks, the waiving of certain fees, and a seven year period of market exclusivity if the drug is approved for public use.
PRN1008 is currently in the midst of a Phase II clinical trial that is testing the drug’s effectiveness as a treatment for ITP. This experimental treatment is classified as a Bruton’s tyrosine kinase (BTK) inhibitor. It is well optimized for use as a long term treatment because of its targeted mechanism of action and quick elimination from the body, which limits its systemic impact.