ClearPoint Neuro, Inc. (Nasdaq:CLPT) (the “Company”), a global therapy-enabling platform company providing navigation and delivery to the brain, announced the U.S. Food and Drug Administration (FDA) has granted marketing authorization for the SmartFlow Neuro Cannula using the De Novo pathway. The SmartFlow Neuro Cannula 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. This signifies the first-ever FDA marketing authorization of a device used to deliver gene therapy directly to regions of interest within the brain.
“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. We could not be prouder of the PTC and ClearPoint Teams and how we have worked together. Most importantly, we are thrilled and humbled that children with this terrible disease will now be treated with the first-ever neuro gene therapy available commercially in the United States.”
“I am proud to have served as a PI in the trial supporting today’s landmark approvals,” stated Dr. Daniel Curry, Director, Functional Neurosurgery and Epilepsy Surgery at Texas Children’s Hospital and Professor, Neurosurgery and Surgery at Baylor College of Medicine. “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.”
“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,” commented Jeremy Stigall, Chief Business Officer and Biologics and Drug Delivery Leader at ClearPoint Neuro. “We offer a complete medical device solution so that our pharma partners can concentrate on the development of their drug and trust us to handle all aspects related to delivery. We are effectively a medical device division that works on behalf of pharma partners, providing 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. Furthermore, we can customize devices for new routes of administration that leverage our unique intellectual property and experience with the FDA to co-develop products that are ideally suited to different cell and gene therapy suspensions and different targets in the brain and spine. More than anything, we offer our partners a head start, and now have proof that we can take these projects to commercial clearance.”
“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,” said Dr. Gerald A. Grant, The Allan H. Friedman Distinguished Professor of Neurosurgery, Chair of the Dept. of Neurosurgery at Duke University. “I feel so privileged to have been part of this clinical trial team and am hopeful that this innovation will open doors for treating many other conditions. The power of hope is so impactful for these children and their families.”
About aromatic L-amino acid decarboxylase (AADC) deficiency
AADC deficiency is a fatal, rare genetic disorder that typically causes severe disability and suffering from the first months of life, affecting every aspect of life-physical, mental and behavioral. The suffering of children with AADC deficiency may be exacerbated by episodes of distressing seizure-like oculogyric crises causing the eyes to roll up in the head, frequent vomiting, behavioral problems, and difficulty sleeping.
The lives of affected children are severely impacted and shortened. Ongoing physical, occupational and speech therapy, and interventions, including surgery, also are often required to manage potentially life-threatening complications such as infections, severe feeding and breathing problems.
