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Bloomsbury Genetic Therapies Announces Orphan Drug Designations of BGT-DTDS for the Treatment of Dopamine Transporter Deficiency Syndrome

London, UK, 26 January 2023 – Bloomsbury Genetic Therapies Limited (Bloomsbury), 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, today announced that both the United States Food and Drug Administration (FDA) and the European Commission (EC) have granted Orphan Drug Designation (ODD) for BGT-DTDS, the company’s neuron-targeted AAV2 investigational gene therapy for the treatment of Dopamine Transporter Deficiency Syndrome (DTDS). BGT-DTDS has completed preclinical efficacy studies and preparations for a first-in-human clinical trial are ongoing.

In the US, ODD is granted by the FDA's Office of Orphan Products Development to promote the development of products that may offer therapeutic benefits for diseases with a prevalence of fewer than 200,000 individuals per year.  Orphan drug designation provides opportunities for grant funding towards clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the United States, if granted FDA approval.

Similiarly, in the EU, ODD is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products (COMP). It is intended to encourage the development of drugs that may provide significant benefit to patients suffering from rare, life-threatening diseases.  If approved for marketing, this designation will provide ten years of marketing exclusivity and also provide special incentives for sponsors, including eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees.

“These Orphan Drug Designations recognise the significant unmet need in patients in the United States and the European Union living with DTDS; a devastating disease with poor prognosis,” said Adrien Lemoine, Co-Founder & Chief Executive Officer of Bloomsbury. “We look forward to continuing to investigate BGT-DTDS and to leveraging the benefits that ODD brings, including significant developmental benefits and the provision of post-approval market exclusivity.”

“DTDS is a rare, often poorly-diagnosed, devastating neurological condition caused by a faulty gene that affects brain cells. Infants with DTDS are rarely able to learn to walk or speak and there are currently no effective treatments, with many children sadly dying before reaching adulthood,” said Professor Manju Kurian, Professor of Neurogenetics at University College London and National Institute of Health and Care Research (NIHR) Research Professor.  “The promise of gene therapy for DTDS is one we are very excited about, particularly given the excellent preclinical data we have amassed. We are hopeful that it could offer patients the real potential of being a transformative, or even curative, therapy.”

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Last Updated: 26-Jan-2023