Arog Pharmaceuticals, Inc , a privately held, clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs to treat unmet medical needs in oncology announced that the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) has granted orphan drug designation for its lead product candidate, crenolanib, for the treatment of acute myeloid leukemia (AML) and soft tissue sarcoma.
Crenolanib also recently received Fast Track designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with unresectable or metastatic gastrointestinal stromal tumors (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRα) D842V mutation.
“This orphan designation from the EMA along with our U.S. fast track designation positions crenolanib for an accelerated global regulatory product development pathway to address unmet needs of patients with AML, and soft tissue sarcoma including GIST with a D842V mutation in the PDGFRA gene,” said Vinay Jain, M.D., Chief Executive Officer of Arog. “We will continue to aggressively advance our clinical development of crenolanib and look forward to providing updates on our ongoing Phase 3 trials for these indications.”
Acute myeloid leukemia is life-threatening due to its rapid progression and its 5-year survival of approximately 22% with current treatments and chronically debilitating due to the consequences of bone marrow dysfunction, such as intracranial or gastro-intestinal hemorrhagic episodes, disseminated intravascular coagulation, and the risk of severe infections. The condition is estimated to affect approximately 1 in 10,000 people in the European Union.
Soft tissue sarcoma is chronically debilitating with a high recurrence and metastasis rate, and life-threatening with an overall 5-year survival rate of approximately 60%. The condition is estimated to affect approximately 2.8 in 10,000 people in the European Union.
To receive orphan drug designation from the EMA, a medicinal product must be intended for the treatment of a life-threatening or a chronically debilitating rare disease affecting not more than 5 in 10,000 individuals in the European Union (EU), and have the potential to be of significant benefit to those affected by that condition. Orphan drug designation provides incentives designed to facilitate development, including protocol assistance, reduced fees for regulatory activities and up to ten years of market exclusivity in the EU upon marketing approval for the designated indication.