International – ATMPs: Global regulators eye harmonization for ultra-orphan products

Top regulators from the US, EU, UK and Japan view harmonization as a critical factor in enabling the development of advanced therapy medicinal products (ATMPs) for ultra-rare diseases.

Particularly for cell and gene therapies targeting patient populations numbering in the hundreds worldwide, commercial viability and the feasibility of clinical trials can be complicated by jurisdictional differences in regulatory requirements. Such challenges were the topic of conversation during a panel discussion with regulators at the Alliance for Regenerative Medicines’ Meeting on the Med on Wednesday…