Pharma Focus Europe

European Medicines Agency Approves Orphan Disease Application for Cellspan Esophageal Implant

Tuesday, August 22, 2023

Harvard Apparatus Regenerative Technology, Inc. (OTCQB: HRGN), a clinical-stage biotechnology firm specializing in organ regeneration to address severe diseases, has disclosed the European Medicines Agency's (EMA) endorsement of an Orphan Disease Application for its flagship product, the Cellspan Esophageal Implant.

The EMA, responsible for reviewing and authorizing new medicines across the European Union, Iceland, Norway, and Liechtenstein, has granted the Orphan Disease Designation (ODD) for the treatment of Esophageal Atresia. This rare congenital condition impacts infants born with an incomplete or underdeveloped esophagus, occurring at an estimated rate of 1 in 2500 to 1 in 4000 live births. The ODD bestows exclusive marketing rights for the Cellspan Esophageal Implant, along with benefits pertaining to clinical protocol development and Scientific Advice.

Significantly, the Centralized Authorization procedure streamlines the application process by allowing companies to submit a single application to the European Medicines Agency, leading to a unified opinion and decision by the European Commission, which holds validity across all EU Member States. Sponsors holding orphan designation may also access conditional approval through this centralized procedure.

Upon approval, orphan medicines are granted a ten-year safeguard against market competition from similar medicines targeting similar indications. This protection can be extended by two years for medicines that commit to a pediatric investigation plan established during the orphan medicine designation review.

While the Agency itself doesn't provide research grants for orphan medicine sponsors, funding opportunities are available through the European Commission, Horizon Europe, and the E-Rare ERA-NET program for rare diseases.

Jerry He, the CEO, expressed, "The ODD will accelerate our efforts to introduce the Cellspan Esophageal Implant to the European market."

Dr. William Fodor, CSO, added, "The approval of the ODD application further validates our role as a prominent developer of tissue-engineered organ replacement products, spanning congenital organ deficiencies and the restoration of tubular organs damaged by disease or injury."

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