The Only Device Approved to Deliver First Disease-Modifying Treatment for AADC Deficiency in the United States

SOLANA BEACH, CA / ACCESSWIRE / November 13, 2024

ClearPoint Neuro, Inc. (Nasdaq:CLPT), a global therapy-enabling platform company providing navigation and delivery to the brain, announced that the U.S. Food and Drug Administration (FDA) has granted marketing authorization for the SmartFlow Neuro Cannula using the De Novo pathway. This device is intended for intraputaminal administration of PTC Therapeutics’ gene therapy KEBILIDI™ (eladocagene exuparvovec-tneq) for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency.

Impact on Individuals:

For individuals suffering from AADC deficiency, the approval of the SmartFlow Neuro Cannula and KEBILIDI™ gene therapy represents a significant milestone. This is the first disease-modifying treatment approved in the United States for AADC deficiency, offering hope for improved quality of life and disease management.

Impact on the World:

On a broader scale, the approval of this therapy marks a breakthrough in the field of neurology and gene therapy. It sets a precedent for future treatments targeting rare genetic disorders and demonstrates the potential of innovative medical technologies to address unmet medical needs.

Conclusion:

The FDA’s approval of the SmartFlow Neuro Cannula for delivering the KEBILIDI™ gene therapy is a significant advancement in the treatment of AADC deficiency. This milestone not only offers hope for individuals with this rare genetic disorder but also paves the way for future developments in the field of neurology and gene therapy.