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US FDA Grants Orphan Drug Designation to Astex Pharmaceuticals' Guadecitabine for Acute Myeloid Leukemia
6 October 2015 - - US-based pharmaceutical company Astex Pharmaceuticals' novel hypomethylating agent, guadecitabine (SGI-110), has been granted an orphan drug designation by the US FDA, the company said on Tuesday. Guadecitabine has been evaluated in multiple Phase 1 and Phase 2 trials to investigate its potential in the treatment of a range of cancers. The company has recently completed a large (over 400 patients) randomized Phase 1/2 study in patients with myelodysplastic syndromes or AML. Astex said that the trial included a Phase I dose escalation stage (93 patients) and a randomised Phase 2 stage (308 patients) that investigated four different patient populations: treatment naïve and relapsed/refractory AML and MDS. The trial demonstrated that guadecitabine was clinically active and well tolerated in all four patient groups. Guadecitabine is now being evaluated in the ASTRAL-1 trial, a large, global, randomized 800-patient study in treatment naïve AML patients who are unfit to receive, or unsuitable for, intensive induction chemotherapy. The trial compares guadecitabine with physician's choice of low-dose cytarabine, decitabine or azacitidine. Orphan Drug designation is granted to drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 people in the US. It qualifies the sponsor for incentives provided for in the Orphan Drug Act, which can include protocol assistance for clinical trials, prescription drug user fee waivers, tax incentives and seven years of market exclusivity.
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