Krystal Biotech’s KB103 Receives Orphan Medicinal Product Designation in Europe for Dystrophic Epidermolysis Bullosa

Krystal Biotech (NASDAQ:KRYS), a gene therapy company dedicated to developing and commercializing novel treatments for patients suffering from dermatological diseases, today announces that KB103, a gene therapy candidate for dystrophic epidermolysis bullosa (“DEB”), was granted Orphan Medicinal Product Designation (OMPD) from the European Medicines Agency (EMA).  KB103 represents the first investigational HSV-1 based gene therapy in DEB to receive this designation.

As quoted in the press release:

In Europe, OMPD is available to companies developing products intended to treat a life-threatening or chronically debilitating condition that have a patient prevalence in the European Union (“EU”) of no more than five in 10,000.  This designation is only granted when there is no approved satisfactory treatment for the specific condition. OMPD offers a company a) product market exclusivity for ten years in the EU following regulatory approval, b) protocol assistance from the EMA at reduced fees during product development phase, c) access to centralized marketing authorization and d) tax and financial incentives for companies developing medicines for such orphan indications.

“Being granted Orphan Medicinal Product Designation in Europe represents another important global milestone for our KB103 program,” said Suma  Krishnan, founder and chief operating officer of Krystal. “Last month we filed an Investigational New Drug application for KB103 in the U.S. and intend to advance the clinical development of KB103 in U.S. in 2018 and in EU in 2019.”

Click here to read the full press release.