ClearPoint Neuro, Inc. (Nasdaq:CLPT) has achieved a significant milestone with the FDA's De Novo marketing authorization of its SmartFlow Neuro Cannula, the first device approved for direct delivery of gene therapy to the brain. This approval specifically targets the intraputaminal administration of PTC Therapeutics' gene therapy KEBILIDI™ (eladocagene exuparvovec-tneq) for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency.
Landmark Approval for Neuro Gene Therapy
This FDA authorization marks a pivotal moment as it is the first time a device has been cleared for delivering gene therapy directly into the brain. The SmartFlow Neuro Cannula allows for precise delivery of therapeutics, bypassing the blood-brain barrier using Convection Enhanced Delivery (CED) under direct image guidance. This approach is particularly crucial for treating neurological disorders where targeted drug delivery is essential.
"Today is one of the most important strategic milestones in the history of our company," commented Joe Burnett, President and CEO at ClearPoint Neuro. "FDA's granting of this De Novo classification is the culmination of years of disciplined co-development, creative problem solving, and tireless efforts."
Addressing AADC Deficiency
AADC deficiency is a rare, genetic disorder that leads to severe disability and suffering from early infancy. Children with AADC deficiency often experience movement disorders, developmental delays, and other neurological issues. KEBILIDI™, delivered via the SmartFlow Neuro Cannula, aims to address the underlying genetic cause of the disease by delivering functional genes directly to the affected brain region.
Dr. Daniel Curry, Director, Functional Neurosurgery and Epilepsy Surgery at Texas Children's Hospital, stated, "I have witnessed first-hand the positive impact these treatments have had on children with AADC Deficiency. Neurosurgeons are leading the way in this promising field to address the underlying genetic cause, and not just the symptoms, of devastating neurological disorders by delivering gene therapy directly to targets in the brain through dedicated platforms."
Comprehensive Solutions for Pharma Partners
ClearPoint Neuro offers a complete medical device solution for pharmaceutical partners, encompassing surgical strategy, benchtop testing, compatibility assessments, pre-clinical study management, clinical trial support, precise navigation, infusion monitoring software, and a routinely audited quality management system and manufacturing processes. This comprehensive approach allows pharma companies to focus on drug development while ClearPoint Neuro manages the delivery aspects.
Jeremy Stigall, Chief Business Officer and Biologics and Drug Delivery Leader at ClearPoint Neuro, commented, "Today's De Novo approval will give confidence to all our current and future biopharma partners that ClearPoint has the experience to help their cell and gene therapies cross the finish line."
Future Implications and Market Opportunity
With over 100 CNS gene therapy programs in development, ClearPoint Neuro is well-positioned to capitalize on the growing market for targeted CNS therapeutics. The SmartFlow Neuro Cannula has already been used in over 8,000 procedures and is utilized by more than 50 pharmaceutical, academic, and biotech partners.
Dr. Gerald A. Grant, Chair of the Dept. of Neurosurgery at Duke University, noted, "This pioneering treatment has the potential to truly transform the standard of care for children with this disorder and this platform holds promise for many other conditions."
