Myrtelle Inc. announced that the FDA granted Regenerative Medicine Advanced Therapy (RMAT) Designation to the Company's lead gene therapy product candidate, rAAV-Olig001-ASPA for the treatment of Canavan disease (CD). 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.
"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. The RMAT program provides FDA guidance on drug development and interactions that support accelerated approvals for treatments that serve communities like Canavan who face considerable unmet medical need," said Nancy Barone Kribbs, PhD, 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. In CD, normal brain development is impaired due to a mutation in the ASPA gene that encodes the enzyme aspartoacylase. The lack of normal aspartoacylase activity negatively impacts brain bioenergetics and development, including myelin production. 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 & Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency, as well as Innovative Licensing and Access Pathway (ILAP) from the United Kingdom Medicines & Healthcare products Regulatory Agency.
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