Bloomsbury Genetic Therapies Receives Rare Paediatric Disease Designation from the U.S. FDA for BGT-DTDS for the Treatment of Dopamine Transporter Deficiency Syndrome (DTDS)

Bloomsbury Genetic Therapies Receives Rare Paediatric Disease Designation from the U.S. FDA for BGT-DTDS for the Treatment of Dopamine Transporter Deficiency Syndrome (DTDS)

London, UK, 3 May 2023 – Bloomsbury Genetic Therapies Limited, a clinical-stage biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies, announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Paediatric Disease (RPD) Designation for the company’s neuron-targeted AAV2 investigational gene therapy for the treatment of DTDS.

The grant of RPD Designation by the FDA follows the positive feedback that Bloomsbury recently received from the UK Medicines and Healthcare products Regulatory Agency (MHRA) which supported the filing of a clinical trial application for a Phase 1/2/3 clinical trial for BGT-DTDS without the need for further non-clinical studies and the grant of orphan drug designations in the EU and US that was announced in January.

“DTDS is a devastating disease with poor prognosis and paediatric patients living with DTDS face a significant unmet need with no disease-modifying treatments nor approved therapies currently available,” said Adrien Lemoine, Co-Founder & Chief Executive Officer of Bloomsbury. “Obtaining Rare Paediatric Disease Designation is another acknowledgement of the serious and life threatening manifestations of this rare disease, and supports our mission to provide BGT-DTDS as a potential new treatment for patients suffering from DTDS.”

RPD Designation is granted by the FDA for serious or life-threatening diseases which affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. If a Biologics Licensing Application (BLA) for BGT-DTDS for the treatment of DTDS is approved by the FDA, Bloomsbury may be eligible to receive a Priority Review Voucher (PRV) that can be redeemed to receive a priority review for any subsequent marketing application, or may be sold or transferred. This program is intended to encourage the development of new drugs and biologics for the treatment of rare paediatric diseases.