NEW YORK, March 1, 2017 – Y-mAbs Therapeutics, Inc. (YmAbs), an immunotherapy company discovering and developing innovative treatments for patients with cancer, today announced that 131I-8H9 for the treatment of Refractory Leptomeningeal Metastasis from Neuroblastoma has been designated an orphan drug by the European Medicines Agency (EMA).
The filing for this status was completed as part of an overall plan to expand the European development and obtain orphan drug protection for 131I-8H9 to treat Refractory Leptomeningeal Metastasis from Neuroblastoma.
Under the EMA’s Regulation (EC) No. 141/2000 an orphan drug designation gives companies access to protocol assistance and guidance on preparing a dossier that will meet European regulatory requirements and thereby maximize the chance of success at the time of marketing authorization. Once approved, an orphan drug is also granted 10 years of market exclusivity during which directly competitive similar products cannot normally be placed on the market.
The EMA grants orphan drug designation based upon several criteria: the life threatening and debilitating nature of the condition; the medical plausibility of the proposed orphan indication; a prevalence in Europe of less than 5 cases for each 10,000 of population; no satisfactory method of diagnosis, prevention or treatment exists or if such method exists the medicinal product will be of significant benefit to those affected by that condition.
YmAbs Founder, President and Head of Business Development and Strategy, Thomas Gad said, “We are very pleased that the EMA has granted orphan drug designation to 131I-8H9 which gives us various development incentives”.
Dr. Claus Møller, Chief Executive Officer further notes, “The orphan designation strengthens our chances of bringing 131I-8H9 to patients who desperately need alternative methods of treatment. Further, the designation marks a substantial milestone in YmAbs’ expansion into European development.”