INAD Gene Therapy in the UK Receives EU and US Orphan Drug Designation
December 2023
Bloomsbury Genetic Therapies Limited, based in London, has received orphan drug designations (ODD) from both the European Commission (EC) and the US Food and Drug Administration (FDA) for BGT-INAD, an investigational AAV9 gene therapy to treat Infantile Neuroaxonal Dystrophy (INAD). This therapy offers a potentially curative option for INAD patients through a single cerebrospinal fluid (CSF) injection.
In the US, an ODD designation, which is for diseases with fewer than 200,000 affected US individuals, opens doors for grant opportunities, FDA user-fee benefits, tax advantages, and a seven-year market exclusivity upon regulatory approval. If approved for marketing, the EU provides a similar designation with ten years of market exclusivity.
Bloomsbury also secured a rare pediatric disease designation in the US. They also received a Type B Pre-Investigational New Drug Application (IND) meeting with the FDA, obtaining detailed advice on their plans for BGT-INAD's clinical development.
According to Adrien Lemoine, Co-Founder & CEO of Bloomsbury, the Pre-IND meeting's paramount benefit lies in receiving early insights from the FDA and understanding their expectations for the drug development program. Lemoine expressed satisfaction with the meeting, citing active engagement and constructive guidance from the FDA.
Collaborating with the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) and following June feedback, Bloomsbury is progressing through preclinical studies for BGT-INAD. These studies have already shown compelling preclinical efficacy data in partnership with University College London (UCL) in preparation for a Phase 1/2/3 clinical trial in Europe and the US.
Anticipating completion of efficacy studies early next year, the next steps involve rodent biodistribution/toxicology studies and obtaining a vector adhering to strict regulatory standards and Good Manufacturing Practices before initiating a clinical trial.
Lemoine stated that Bloomsbury anticipates filing an IND to start a clinical trial no earlier than 2025, with the trial slated for 2025/2026, considering the necessary time for these comprehensive activities and contingent upon their funding success.
