Bloomsbury Genetic Therapies gets Rare Pediatric Disease designation from the US FDA for the Treatment of Ornithine Transcarbamylase Deficiency (OTCD)

UCL spinout Bloomsbury Genetic Therapies Limited has been granted Rare Pediatric Disease Designation (RPDD) for BGT-OTCD, the company’s investigational liver-targeted gene therapy for the treatment of OTCD, by the US Food and Drug Administration (FDA).

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.

The FDA grants RPDD 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.

“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”.

“OTCD is usually diagnosed during childhood, but we intend to investigate BGT-OTCD in both paediatric and adult populations, since its superior efficacy profile bears the promise of a potentially curative solution for all patients” said Adrien Lemoine, Co-Founder & Chief Executive Officer of Bloomsbury. “We look forward to leveraging the benefits that this new designation brings to the program.”

Sara Garcia Gomez PhD, Senior Business Manager at UCLB, added: “We’re delighted to hear that FDA has granted a Rare Pediatric Disease Designation (RPDD) to the OTCD gene therapy treatment, while Bloomsbury Genetic Therapies is progressing this programme into the clinic. This designation jointly with the previously granted Orphan Drug Designation (ODD) by EMA brings potential benefits down the line as market exclusivity.”

If a Biologics Licensing Application (BLA) for BGT-OTCD for the treatment of OTCD 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.

Read the full story here.

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