Arog Pharmaceuticals Receives Orphan Drug Designation in the European Union for Crenolanib for the Treatment of Acute Myeloid Leukemia and Soft Tissue Sarcoma

DALLAS, Nov. 21, 2016 (GLOBE NEWSWIRE) -- 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, today 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.

About Arog Pharmaceuticals, Inc.

Arog Pharmaceuticals is a private, clinical-stage biopharmaceutical company that has leveraged its platform of benzimidazole derivatives to develop a robust drug pipeline of orally available, potent, and selective small molecule type I kinase inhibitors.  Arog is poised to enroll patients in pivotal, randomized Phase 3 trials of its lead molecule, crenolanib.  In addition to the six clinical trials it has already completed, Arog is also engaged in five ongoing Phase 2 clinical trials. For more information, please visit the company’s website,

About Crenolanib 

Arog’s lead molecule, crenolanib, is currently being clinically investigated as a treatment for multiple cancers, including acute myeloid leukemia (AML), gastrointestinal stromal tumors (GIST), glioma, and non-small cell lung cancer (NSCLC).  It is an orally bioavailable benzimidazole type I kinase inhibitor that selectively and potently inhibits signaling of wild-type and mutant isoforms of class III receptor tyrosine kinases FLT3 and PDGFRα/β. This molecule has an established record of patient safety and has been used to treat over 300 patients from around the world.

About FLT3 

FLT-3 is a class III receptor tyrosine kinase, and its signaling is considered important for the normal development of hematopoietic stem cells and progenitor cells.  The FLT-3 gene is one of the most frequently mutated genes (~35%) in acute myeloid leukemia (AML).  One such mutation, internal tandem duplications of FLT-3 (FLT3-ITD), is a prognostic indicator associated with adverse disease outcome. Also, FLT-3 (FLT3-D835), is known to be a common cause of resistance to other FLT3 inhibitors.

About PDGFRα/β  

Platelet-derived growth factor receptors (PDGFR) -α and -β are cell surface tyrosine kinase receptors and are important factors regulating cell proliferation and cell development, as well as several diseases, including cancers like brain tumors and sarcomas.  In clinical tests, crenolanib has been shown to inhibit both PDGFR-α and -β phosphorylation, thus preventing downstream signaling.


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