Forge Biologics Receives Priority Medicines (PRIME) Designation from the European Medicines Agency (EMA) for Novel Gene Therapy FBX-101 for the Treatment of Patients with Krabbe Disease - Forge Biologics

Forge Biologics Receives Priority Medicines (PRIME) Designation from the European Medicines Agency (EMA) for Novel Gene Therapy FBX-101 for the Treatment of Patients with Krabbe Disease

  • FBX-101 is granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) after review of Phase 1/2 RESKUE clinical trial safety and efficacy data
  • PRIME designation is granted to drug candidates that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options

COLUMBUS, Ohio – January 17, 2023 Forge Biologics, a gene therapy-focused contract development and manufacturing organization, today announced that the European Medicines Agency (EMA) has granted priority medicines (PRIME) designation to FBX-101, Forge’s lead adeno-associated virus (AAV) drug candidate and novel gene therapy 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 recognizing 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.”

The designation follows positive safety and efficacy data from the ongoing Phase 1/2 RESKUE trial. This positive clinical data was presented by Maria Escolar, M.D., Forge’s Chief Medical Officer at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium in August 2022, and updated in October at the 29th Congress of European Society of Gene & Cell Therapy (ESGCT). The results demonstrated 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.

About Krabbe Disease
Krabbe disease is a rare neurodegenerative disease affecting about 1-2.5 in 100,000 people in the U.S. Krabbe disease is caused by autosomal recessive mutations in the galactocerebrosidase (GALC) gene, an enzyme responsible for the breakdown of certain types of sphingolipids, such as psychosine, associated with myelination of the nervous system. Without functional GALC, psychosine accumulates to toxic levels in cells, specifically in cells insulating the nerves in the brain and peripheral nervous system, causing rapid demyelination. Krabbe disease initially manifests as irritability, developmental delay, and progressive muscle weakness; symptoms rapidly advance to difficulty swallowing, breathing, worsening developmental delay, and vision and hearing loss. Infantile Krabbe disease (0 –12 months of age at onset) usually leads to death in untreated patients by 2 years of age. Late Infantile patients (12-36 months of age at onset) usually die by the age of six. The current standard of care, hematopoietic stem cell transplantation (HSCT), has been shown to stabilize cognitive decline and significantly improve long-term neurological outcomes when performed prior to symptom onset. However, HSCT does not correct the peripheral neuropathy that is progressive as the patient grows, leading to loss of gross motor skills and eventually death. Early diagnosis is key for treating Krabbe patients before significant neurological damage has occurred. Currently, 10 states in the USA are conducting newborn screening for Krabbe disease. Infants who screen positive, meaning insufficient GALC activity is detected, undergo psychosine and mutation analysis to confirm the diagnosis and predict disease onset.

About FBX-101
FBX-101 was developed to treat children with Krabbe disease. FBX-101 is an adeno-associated viral serotype rh10 (AAVrh10) gene therapy that is delivered intravenously after HSCT infusion. The vector delivers a functional copy of the GALC gene to cells in both the central and peripheral nervous system. FBX-101 has been shown to functionally correct the central and peripheral neuropathy associated with Krabbe, improve gross motor outcomes, and significantly prolong lifespan in animal models. This approach has the potential to overcome some of the immunological safety challenges observed in traditional AAV gene therapies and extend the duration of gene transfer.

About the RESKUE Trial
RESKUE a Phase 1/2 clinical trial to investigate the safety and efficacy of FBX-101 in patients with Infantile Krabbe disease. It is a nonblinded, non-randomized dose escalation study of intravenous AAVrh10 after HSCT infusion, in which subjects receive standard of care hematopoietic cell transplantation for Krabbe Disease, followed by a single infusion of an adeno-associated virus gene therapy product. Extensive natural history subjects will be used to compare as control group. More information on the RESKUE trial can be found online at

About Forge Biologics
Forge Biologics is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company. Forge’s mission is to enable access to life changing gene therapies and help bring them from idea to reality. Forge’s 200,000 square foot facility utilizes 20 cGMP suites in Columbus, Ohio, the Hearth, to serve as its headquarters. The Hearth is a custom-designed cGMP facility dedicated to AAV manufacturing and hosts scalable, end-to-end manufacturing services. Offerings include process and analytical development, plasmid DNA manufacturing, viral vector manufacturing, final fill, as well as regulatory consulting support to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing. By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most. To learn more, visit

Media Inquiries
Marina Corleto – Forge Biologics
Associate Director, Marketing and Communications   

Families and Clinician Inquiries
Maria Escolar, M.D. – Forge Biologics
Chief Medical Officer

Business Development  
Magdalena Tyrpien – Forge Biologics
Senior Vice President, Head of Business Development   


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