Bloomsbury Genetic Therapies Receives Orphan Drug Designation from the U.S. FDA for BGT-OTCD for the Treatment of Ornithine Transcarbamylase Deficiency (OTCD)

Bloomsbury Genetic Therapies Receives Orphan Drug Designation from the U.S. FDA for BGT-OTCD for the Treatment of Ornithine Transcarbamylase Deficiency (OTCD)

London, UK, 9 August 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 Orphan Drug Designation (ODD) for BGT-OTCD, the Company’s investigational liver-targeted gene therapy for the treatment of 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 1/2 clinical trial of BGT-OTCD, Halting Ornithine transcarbamylase deficiency with Recombinant AAV in ChildrEn (HORACE), in paediatric patients diagnosed with OTCD, which is expected to begin enrolment in the UK in Q3 2023.

“Having already received an ODD from the European Medicine Agency and the Rare Pediatric Disease Designation from the FDA for BGT-OTCD, we now look forward to leveraging the benefits that this new designation brings, including the provision of post-approval market exclusivity in the US” said Adrien Lemoine, Co-Founder & Chief Executive Officer of Bloomsbury.

The FDA grants ODD to a drug or biologic intended to treat a rare disease or condition, which generally includes a disease or condition that affects fewer than 200,000 individuals in the US. ODD provides opportunities for grant funding towards clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the US in the event of regulatory approval.