RPDD granted in the USA for Bloomsbury Genetic's for BGT-OTCD

UK biotech start-up Bloomsbury Genetic Therapies says that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation (RPDD) for BGT-OTCD, the company’s investigational liver-targeted gene therapy for the treatment of Ornithine transcarbamylase deficiency (OTCD).

In May, the UK Medicines and Healthcare Products Regulatory Agency (MHRA) approved the clinical trial application submitted by Bloomsbury’s collaborators at University College London (UCL), to initiate a Phase I/II clinical trial of BGT-OTCD, halting Ornithine transcarbamylase deficiency with Recombinant AAV in ChildrEn (HORACE), in pediatric patients diagnosed with OTCD, which is expected to begin enrolment in the UK in third-quarter 2023.

“Current medical management of OTCD patients and dietary protein restriction do not prevent recurrent metabolic crises, with hyperammonaemia leading to neurological damage,” said Professor Paul Gissen, professor of metabolic medicine and head of gene and stem cell therapy at UCL Great Ormond Street Institute of Child Health. With the highest unmet medical need seen in the paediatric population, we are pleased to see this recognition from the FDA as we complete our preparations for the HORACE trial,” he added.