“Myrtelle” or the “Company”, a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA), the healthcare regulatory body of the United Kingdom (UK), granted Innovative Licensing and Access Pathway (ILAP) designation to the Company’s lead gene therapy product candidate, rAAV-Olig001-ASPA for the treatment of Canavan disease. ILAP provides expedited access to a range of UK development services and tools for life-threatening or seriously debilitating conditions for which there is a significant patient need. These services include the potential for frequent MHRA interactions, accelerated Marketing Authorization Application (MAA) assessment, innovative and flexible licensing routes, engagement on market access activities, and a continuous benefit-risk assessment integrating real world evidence.
“The ILAP designation by MHRA for rAAV-Olig001-ASPA provides access to tools that aim to accelerate the development of this potential treatment for children with Canavan disease who currently have significant unmet medical needs and are without approved treatment options”
“The ILAP designation by MHRA for rAAV-Olig001-ASPA provides access to tools that aim to accelerate the development of this potential treatment for children with Canavan disease who currently have significant unmet medical needs and are without approved treatment options,” said Nancy Barone Kribbs, PhD, Senior Vice President of Global Regulatory Affairs at Myrtelle.
rAAV-Olig001 is a novel vector from a class of recombinant AAVs (rAAVs) that selectively target oligodendrocytes – the cells in the brain responsible for producing myelin, the insulating material that enables proper function of neurons and makes up the brain’s white matter. The Company’s lead program is in Phase 1/2 clinical development for Canavan disease (CD) a fatal childhood genetic disorder characterized by the degeneration of the white matter in the brain. The production of myelin is affected in CD due to a mutation in the Aspartoacylase gene (ASPA) leading to deficiency in Aspartoacylase enzyme (ASPA). The oligodendrocyte-targeted gene therapy using the rAAV-Olig001 vector is intended to restore ASPA function, thus enabling metabolism of N-Acetylaspartic Acid (NAA), a neurochemical abundant in the brain, and supporting myelination. Myrtelle entered into an exclusive worldwide licensing agreement with Pfizer Inc. in 2021 to develop and commercialize this novel gene therapy for the treatment of CD.
In addition to ILAP designation, rAAV-Olig001-ASPA has been granted Orphan Drug Designation & Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency, as well as Orphan Drug, Rare Pediatric Disease, and Fast Track designations from the US FDA.
Source: https://www.businesswire.com/
