RARE Daily

Myrtelle’s Gene Therapy Candidate for Canavan Disease Receives ATMP Classification from EMA

September 8, 2022

The European Medicines Agency has classified Myrtelle’s lead gene therapy product candidate, rAAV-Olig001-ASPA for the treatment of Canavan disease, as an Advanced Therapy Medicinal Product (ATMP), specifically a Gene Therapy Medicinal Product (GTMP).

Photo: Nancy Barone Kribbs, senior vice president of Global Regulatory Affairs at Myrtelle

ATMP classification, which is determined by the Committee for Advanced Therapies, was established to regulate cell and gene therapy and tissue engineered medicinal products, support development of these products, and provide a benchmark for the level of quality compliance for pharmaceutical practices. As a designated GTMP product, rAAV-Olig001-ASPA will follow the Centralized Procedure through the EMA and benefit from a single evaluation and authorization process. Additional benefits established through the ATMP regulation include pathways for Scientific Advice and significant fee reductions for such advice.

Canavan disease (CD) is a fatal childhood genetic brain disease in which mutations in the aspartoacylase gene (ASPA) prevent the normal expression of aspartoacylase (ASPA), a critical enzyme produced in oligodendrocytes that breaks down the neurochemical N-acetylaspartate (NAA). When not properly metabolized by oligodendrocytes, NAA accumulates in the brain and negatively affects bioenergetics, myelin production, and brain health. CD patients are impacted at birth but may appear normal until several months old when symptoms begin to develop. Poor head control, abnormally large head size, difficulty in eye tracking, excessive irritability, severely diminished muscle tone, and delays in reaching motor milestones, such as rolling, sitting, and walking, are the typical initial manifestations of CD. As the disease progresses, seizures, spasticity, difficulties in swallowing, and overall muscle deterioration emerge with most affected children developing life-threatening complications by approximately 10 years of age. Currently, there are no cures for CD and only palliative treatments are available.

Myrtelle’s rAAV-Olig001 is a novel vector from a class of recombinant AAVs that selectively target oligodendrocytes, the cells in the brain responsible for producing myelin, which is in phase 1/2 clinical development for Canavan disease The oligodendrocyte-targeted gene therapy using the rAAV-Olig001 vector is intended to restore ASPA function, thus enabling metabolism of NAA and supporting myelination. Myrtelle entered into an exclusive worldwide licensing agreement with Pfizer in 2021 to develop and commercialize this novel gene therapy for the treatment of CD.

In addition to ATMP classification, rAAV-Olig001-ASPA has been granted U.S. Orphan Drug, Rare Pediatric Disease, and Fast Track designations by the FDA.

“The designation by the EMA of rAAV-Olig001-ASPA as a Gene Therapy Medicinal Product as a potential treatment for patients with Canavan disease provides important benefits in the development of this innovative therapy. The ATMP classification will facilitate discussions with the EMA as part of our strategy to seek product registration in the EU,” said Nancy Barone Kribbs, senior vice president of Global Regulatory Affairs at Myrtelle.

Author: Rare Daily Staff

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