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FDA Orphan Drug Status


Two encouraging trials commenced at the start of 2015 for anti-cancer therapy Mocetinostat. Both studies, sponsored by Mirati Therapeutics, focus on this powerful and selective inhibitor of HDAC 1, 2, 3 and 11, which all are major players in the regulation of cancer gene expression. As an orally-bioavailable therapy, Mocetinostat has been at the forefront[…]

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Exciting news was revealed on June 18, 2014 with the announcement the Mocetinostat Orphan Drug Designation from the FDA. Information gleaned from a Phase 1/2 study of Mocetinostat showed that when used alongside Azacitidine (Vidaza) this combination provided efficacious treatment for patients with myelodysplastic syndromes. Patients who were at intermediate or high risk myelodysplastic syndromes,[…]

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As the latest molecule in the mucoactive drug class Erdosteine has recently received Orphan-Drug Designation from the FDA. Erdosteine was developed by Edmond Pharma and this productive drug is already marketed in 40 countries around the world for the treatment of both chronic obstructive pulmonary disease (COPD) and chronic bronchitis. There are currently no medications[…]

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At the end of January 2014 the powerful drug Eculizumab received orphan drug status from the FDA as a preventative treatment of delayed graft function (DGF) for renal transplant patients. Eculizumab is already approved to treat paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. Paroxysmal nocturnal hemoglobinuria or PNH is a rare form of anemia[…]

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