Crenolanib is an oncolytic in phase II clinical trials at AROG Pharmaceuticals for the treatment of gastrointestinal stromal cancer (GIST) in patients with the D842V mutation in the PDGFRA gene and for the treatment of adult gliomas. St Jude Children's Research Hospital is evaluating the compound in phase I clinical trials for the treatment of children and young adults (18 months to 21 years) with newly diagnosed diffuse intrinsic pontine glioma or recurrent high-grade glioma.The compound had been in early clinical trials at Pfizer for the treatment of non-small cell lung cancer (NSCLC); however, development for this indication was discontinued in 2007. 

The drug candidate is a potent inhibitor of the angiogenic platelet-derived growth factor receptor (PDGFR) beta kinase, which also modulates tumor interstitial fluid pressure and influences cell growth, migration and survival. 

Crenolanib was jointly discovered through a collaboration between OSI Pharmaceuticals and Pfizer that was penned in April 1986, and renewed in April 1991 and April 1996. Since April 2001, when the collaboration was allowed to expire, Pfizer continues to develop the drug candidate. In 2010, crenolanib was licensed to AROG Pharmaceuticals by Pfizer. In 2012, orphan drug designation was assigned in the U.S. for the treatment of acute myeloid leukemia.

Phase Organization ConditionPhase IIAROG PharmaceuticalsGastrointestinal stromal tumor (GIST)Phase IIAROG PharmaceuticalsLeukemia, acute myeloidPhase IAROG PharmaceuticalsGlioma