RARE Daily

FDA Grants RMAT Designation to Myrtelle’s Gene Therapy Candidate for Canavan Disease

April 3, 2024

Rare Daily Staff

The U.S. Food and Drug Administration granted Regenerative Medicine Advanced Therapy designation to Myrtelle’s lead gene therapy candidate for the treatment of Canavan disease.

The RMAT designation program was created to expedite drug development and the review process of promising pipeline drugs, including gene therapies. Designation is granted based on preliminary clinical evidence indicating that a drug or therapy has the potential to address unmet medical needs and is intended to treat, modify, reverse, or cure serious or life-threatening conditions.

Canavan disease (CD) is a fatal childhood genetic brain disease caused by mutations in the ASPA gene, which prevent the normal expression of aspartoacylase, a critical enzyme produced in oligodendrocytes. The lack of normal aspartoacylase expression negatively impacts brain bioenergetics and development, including myelin production. Patients with CD 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.

“The RMAT designation by FDA for rAAV-Olig001-ASPA is significant in that it recognizes the potential of this treatment for children with Canavan disease who are without approved treatment options,” said Nancy Kribbs, senior vice president of Global Regulatory Affairs at Myrtelle.

Myrtelle’s FIH trial utilizes the company’s proprietary rAAV vector to directly target oligodendrocytes, the brain cells affected in CD that are responsible for producing myelin – the insulating material that enables proper neuronal function. The oligodendrocyte-targeting rAAV vector-based gene therapy is intended to restore ASPA function and brain development in patients with CD.

In addition to RMAT designation, rAAV-Olig001-ASPA has been granted Orphan Drug, Rare Pediatric Disease, and Fast Track designations from the FDA, Orphan Drug Designation and Advanced Therapy Medicinal Product classification from the European Medicines Agency, as well as Innovative Licensing and Access Pathway from the United Kingdom Medicines & Healthcare products Regulatory Agency.

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

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