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.

 

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About Bloomsbury

Bloomsbury is 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. The Company was spun out of University College London and launched in October 2022 with funding from UCL Technology Fund. Bloomsbury is building a pipeline of highly differentiated first- or best-in-class programs. For more information, please visit www.bloomsburygtx.com

 

About BGT-OTCD

BGT-OTCD is a liver-targeted AAV-LK03 gene therapy designed to provide a potentially curative solution to OTCD patients following a one-time intravenous injection. AAV-LK03 was selected for its high tropism for liver cells and its success in other liver disorders such as haemophilia A. Following approval of a Clinical Trial Application (CTA) by the UK Medicine and Healthcare products Regulatory Agency (MHRA), BGT-OTCD will be evaluated in HORACE (Halting Ornithine transcarbamylase deficiency with Recombinant AAV in ChildrEn), a Phase 1/2 clinical trial which will be initiated in Q3 2023. BGT-OTCD has been granted Orphan Drug Designation (ODD) for the treatment of OTCD by the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) and has received Rare Pediatric Disease Designation (RPDD) from the FDA.

 

About Ornithine transcarbamylase deficiency

Ornithine transcarbamylase deficiency (OTCD) is a rare, X-linked genetic disorder that is characterised by complete or partial lack of the OTC enzyme. OTC is a key component of the urea cycle and patients with OTCD accumulate nitrogen waste in the form of excess ammonia (hyperammonaemia) in the blood, causing hyperammonaemic decompensations with symptoms including vomiting, impaired voluntary movement and progressive lethargy. If left untreated, these may progress to coma and life-threatening complications. While later onset disease can occur in adults with a milder form of the disorder, symptoms present within a few days of birth of males with severe OTCD. Patients are rapidly diagnosed (urine and blood biochemical analyses, gene sequencing) when they present at hospital/are admitted to intensive care with acute hepatic decompensation and hyperammonaemia.

Current standard of care involves protein-restricted diets and ammonia-scavenger medications; however, these approaches can have a significant impact on patients’ quality of life and patients still face lifelong risk of decompensation and neurological damage resulting in intellectual disability, developmental delays, and movement disorder. Liver transplant is the only curative option, but is often unavailable and comes with significant morbidity/mortality risk and lifelong immunosuppression and arginine supplementation. Over 10,000 patients suffering from OTCD have been identified worldwide.