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 BGT-DTDS, 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 or 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.
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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 £5M in Seed funding from UCL Technology Fund and is underpinned by world-leading gene therapy and rare disease expertise from the Company’s academic founders, Professor Paul Gissen, Professor Manju Kurian, Professor Ahad Rahim and Professor Simon Waddington. Bloomsbury is building a pipeline of highly differentiated first- or best-in-class programs. For more information, please visit www.bloomsburygtx.com
About BGT-DTDS
BGT-DTDS is a neuron-targeted AAV2 investigational gene therapy designed to provide a potentially curative solution to DTDS patients following a one-time intra-brain injection to the substantia nigra and ventral tegmental area. BGT-DTDS has completed preclinical efficacy studies and the Company is currently preparing for a first-in-human clinical trial. BGT-DTDS has been granted orphan drug designation for the treatment of DTDS by the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) and has also been granted Rare Paediatric Disease (RPD) Designation for the treatment of DTDS by the FDA. For more information, please visit www.bloomsburygtx.com/pipeline/bgt-dtds/
About Dopamine Transporter Deficiency Syndrome (DTDS)
DTDS is a rare, autosomal recessive childhood neurological disorder with significant disease burden, characterised by a severe parkinsonian movement disorder. DTDS results from biallelic mutations in the SLC6A3 gene which encodes the dopamine transporter (DAT), a key protein regulating dopaminergic signalling. Dopamine re-uptake by DAT is the principal mechanism by which dopamine is removed and recycled from synapses. Mutations in SLC6A3 result in DAT dysfunction and major dysregulation of dopamine neurotransmission, leading to hyperkinetic movement disorders in the early stages of the disease, and then evolution of parkinsonism-like features (slowing of voluntary movements, loss of facial expression, tremor, stiffness) and eye movement disorders such as oculogyric crises. The disease is often fatal in childhood to teenage years due to unexplained death in sleep or respiratory failure. There are currently no disease-modifying treatments or approved therapies for DTDS, and patients derive little clinical benefit from available symptom control medications. The disease is currently poorly diagnosed, due to symptoms similarity with other movement disorders including cerebral palsy, and the number of patients with the condition is unknown.
