FDA Recognizes Canavan Disease Gene Therapy With Multiple Designations


Myrtelle’s gene therapy previously demonstrated initial efficacy in the disease in February 2022.

The FDA has granted fast track, rare pediatric disease, and orphan drug designations to Myrtelle’s gene therapy rAAV-Olig001-ASPA for the treatment of Canavan disease.1

The recombinant adeno-associated virus (rAAV) vector-based gene therapy is currently being evaluated in a phase 1/2 first in-human clinical trial (NCT04833907) for the rare disease. The therapy uses the rAAV vector AAV-Olig, which is designed to directly target oligodendrocytes in the brain to restore aspartoacylasefunction, enable N-Acetylaspartate (NAA) metabolism, and support myelination.

“FDA’s decision to grant these designations for our investigational gene therapy utilizing rAAV-Olig001-ASPA aligns with our mission to provide treatments for patients where few if any options exist and highlights the urgency of developing a treatment for patients with Canavan disease, a devastating disease of young children which results in short life expectancy,” Nancy Barone Kribbs, PhD, senior vice president, Regulatory Affairs, Myrtelle , said in a statement.1

The rAAV-Olig001-ASPA therapy is being developed and commercialized in collaboration with Pfizer as of a 2021exclusive worldwide licensing agreement. The trial is being conducted at Dayton Children’s Hospital in Ohio. The trial is currently recruiting, with an expected enrollment of 24 participants with additional patients enrolled in the second quarter of 2022.2 

READ MORE: Myasthenia Gravis Cell Therapy Granted Fast Track Designation

The therapy has been well-tolerated in the first 3 patients dosed in the trial so far. Patients received a single total dose of 3.7 x 1013 vg delivered by intracerebroventricular injection to target oligodendrocytes.

The trial’s primary endpoint is safety as measured by adverse events (AEs) and their severity. Secondary outcomes explore efficacy and include myelination, brain atrophy, NAA concentration in the brain, motor function, neurocognitive function, spasticity, seizures, and cerebrospinal fluid analysis. 

No treatment-related adverse events have been reported so far in the first cohort, aged 3 to 5 years, and initial efficacy seems encouraging. The trial was opened to 2 younger cohorts: 15 to 36 months of age and under 15 months, in January 2022 by Data Monitoring Committee (DMC) recommendation following encouraging safety and efficacy results.3

“We are encouraged by these early observations in the FIH trial supporting the hypothesis that delivery of a functional ASPA gene may lead to restoration of ASPA enzyme activity and improvements in biochemical and functional brain markers. The ongoing clinical trial is expected to continue to generate the critical data needed for further development of this novel gene therapy in the patients with Canavan disease,” Armen Asatryan, MD, MPH, chief medical officer, Myrtelle, said in a statement at the time.2

1. Myrtelle Receives FDA Fast Track, Rare Pediatric Disease, and Orphan Drug Designations for its Proprietary Gene Therapy for the Treatment of Canavan Disease. News release. Myrtelle. March 15, 2022. https://www.businesswire.com/news/home/20220315005230/en/Myrtelle-Receives-FDA-Fast-Track-Rare-Pediatric-Disease-and-Orphan-Drug-Designations-for-its-Proprietary-Gene-Therapy-for-the-Treatment-of-Canavan-Disease
2. Myrtelle announces positive preliminary clinical data for its proprietary gene therapy in canavan disease. News release. Myrtelle. February 15, 2022. https://www.businesswire.com/news/home/20220215005401/en/Myrtelle-Announces-Positive-Preliminary-Clinical-Data-for-Its-Proprietary-Gene-Therapy-in-Canavan-Disease
3. Myrtelle announces successful completion of initial stage of phase 1/2 clinical trial of proprietary gene therapy for Canavan disease and expands treatment to younger patients. News release. Myrtelle. January 11, 2022. https://www.myrtellegtx.com/news/myrtelle-announces-successful-completion-of-initial-stage-of-phase-1%2F2-clinical-trial-of-proprietary-gene-therapy-for-canavan-disease-and-expands-treatment-to-younger-patients
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