EMA prioritises AAV gene therapy for Krabbe disease

EMA

The European Medicines Agency (EMA) has granted priority medicines (PRIME) designation to FBX-101, Forge Biologic’s adeno-associated virus (AAV) drug candidate for treating patients with Krabbe disease. 

PRIME is a regulatory designation by the EMA that provides early and proactive support to developers of promising medicines, to advance and speed up their development and usher them to reach patients faster.  

The goal is to help patients benefit as early as possible from innovative new therapies that have demonstrated the potential to significantly address and treat patients that have an unmet medical need. 

“We are grateful to the EMA for recognising FBX-101 as a potentially transformative medicine for patients living with this life-threatening and devastating disease,” said Christopher Shilling, Senior Vice President of Regulatory Affairs and Quality at Forge. “Through the enhanced interactions with the EMA granted by the PRIME designation, we will advance and expedite the development of FBX-101 as the leading worldwide gene therapy for patients with Krabbe disease.” 

A safer environment for gene replacement

The designation follows positive safety and efficacy data from the ongoing Phase I/II RESKUE trial. The results demonstrate that systemically delivered FBX-101 administered after hematopoetic stem cell transplant (HSCT) is safe and well-tolerated in Krabbe patients.  

Patients in the low dose cohort demonstrated restoration of GALC enzyme activity, reduced psychosine, showed encouraging signs of normal myelination of brain white matter, and improved motor development as compared to untreated Krabbe patients or patients treated with HSCT alone. 

Clinical data support preclinical observations that this gene therapy approach after HSCT infusion may lessen many of the immune challenges previously observed with systemic AAV gene delivery and may create a safer environment for gene replacement. Findings also support this novel approach for extending the delivery of gene replacement strategies to target metabolic diseases amenable to HSCT. 

Related Articles

Join FREE today and become a member
of Drug Discovery World

Membership includes:

  • Full access to the website including free and gated premium content in news, articles, business, regulatory, cancer research, intelligence and more.
  • Unlimited App access: current and archived digital issues of DDW magazine with search functionality, special in App only content and links to the latest industry news and information.
  • Weekly e-newsletter, a round-up of the most interesting and pertinent industry news and developments.
  • Whitepapers, eBooks and information from trusted third parties.
Join For Free