The FDA has granted orphan drug designation to therapies for six hematology/oncology drug indications.
- avatrombopag (Doptelet, Dova Pharmaceuticals), an oral thrombopoietin receptor agonist, for the treatment of chemotherapy-induced thrombocytopenia;
- CLR 131 (Cellectar Biosciences) — a small-molecule, targeted phospho-lipid-drug conjugate designed to deliver cytotoxic radiation to cancer cells and cancer stem cells while minimizing exposure to healthy cells — for the treatment of lymphoplasmacytic lymphoma;
- Durvalumab (Imfinzi, AstraZeneca), a human monoclonal antibody that binds to PD-L1, and tremelimumab (MedImmune/AstraZeneca), an investigational anti-CTLA-4 monoclonal antibody, for the treatment of patients with advanced hepatocellular carcinoma;
- OBI-999 (OBI Pharma) — which targets Globo H, a glycolipid antigen expressed in up to 15 epithelial cancers — for two indications: gastric cancer and pancreatic cancer; and
- zotiraciclib (Adastra Pharmaceuticals), a multikinase inhibitor, for the treatment of glioma.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.