UCL spinout Bloomsbury Genetic Therapies Limited has received Orphan Drug Designations (ODD) from the US Food and Drug Administration (FDA) and the European Commission (EC) for BGT-NPC, an investigational gene therapy for the treatment of Niemann-Pick Disease Type C (NPC). The clinical-stage biotechnology company is developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases, based on clinically proven gene therapy technologies.

Bloomsbury Genetic Therapies also received a detailed Type B Pre-Investigational New Drug Application (IND) Written Response from the FDA, providing constructive feedback on its plans for chemistry, manufacturing, and controls (CMC), non-clinical and clinical development for BGT-NPC.

Following the FDA feedback and the UK Medicines and Healthcare Products Regulatory Agency (MHRA) feedback disclosed in June 2023, Bloomsbury plans to initiate a toxicology and biodistribution study in rodents ahead of a single, multi-centric Phase 1/2/3 clinical trial. It is currently completing preclinical studies for BGT-NPC in collaboration with partner UCL.

“These Orphan Drug Designations recognise the significant unmet need in patients in the United States and the European Union living with Niemann-Pick Disease Type C, a devastating disease with poor prognosis,” said Adrien Lemoine, Co-Founder & Chief Executive Officer of Bloomsbury. “We are very encouraged by the outcome of our pre-IND interaction with the FDA, and we look forward to an ongoing, constructive dialogue with the FDA as we progress our development plans for BGT-NPC in the US.”

In the US, 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.

Similarly, in the EU, ODD is granted by the EC 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.  This designation provides special incentives for sponsors, including eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees. In addition, if approved for marketing, this designation will provide ten years of marketing exclusivity.

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