AMY-101 is a new drug for treatment of PNH. It inhibits C3, a component of the immune system, and may offer an alternative to Soliris for the treatment of the disease. Research that led to AMY-101 included a study of PNH patients who did not respond to Soliris.
AMY-101 is set to begin human trials in 2015. Its development will benefit from its designation as an orphan drug by both the European Medicines Agency
(EMA) in August and by the U.S. Food and Drug Administration
(FDA) in October. It is hoped that it will be cheaper and more effective for the majority of PNH patients.
AMY-101 was developed at University of Pennsylvania School of Medicine
, then licensed to Amyndas Pharmaceuticals
in Greece for further development. Details about how AMY-101 works can be found in a HematologyTimes article
Orphan drug status provides temporary protection from competition, tax credits, fee reductions, expedited reviews, and other benefits to a company developing a drug for treatment of a rare disease.