Business
ClearPoint Neuro Congratulates Partner PTC Therapeutics on Being Granted Marketing Authorization by the European Commission for Upstaza™ - First Disease-Modifying Treatment for AADC Deficiency
First Gene Therapy Approved for Direct Infusion into the Brain will be Administered with ClearPoint’s SmartFlow® Neuro CannulaSOLANA BEACH, Calif., July 21,
About this update from Clearpoint Neuro Inc.
[{"type":"text","content":"First Gene Therapy Approved for Direct Infusion into the Brain will be Administered with ClearPoint’s SmartFlow® Neuro CannulaSOLANA BEACH, Calif., July 21, 2022 (GLOBE NEWSWIRE) -- ClearPoint Neuro, Inc. (Nasdaq: CLPT) (the “Company”), a global therapy-enabling platform company providing navigation and delivery to the brain, congratulates partner PTC Therapeutics for being granted marketing authorization for Upstaza™ (eladocagene exuparvovec) by the European Commission. Upstaza is the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency, a rare and life-threatening disease impacting children, and is the first marketed gene therapy approved for direct infusion into the brain. The use of ClearPoint Neuro’s proprietary CE Marked SmartFlow® Neuro Cannula for minimally invasive infusion of the gene therapy is included in the Upstaza SmPC (Summary of Product Characteristics). The marketing authorization, approved for patients 18 months and older, is applicable to all 27 European Union member states, as well as Iceland, Norway and Liechtenstein. “The approval of Upstaza by the European Commission will have an immense impact on patients, their families, and the global gene therapy research community. Upstaza is the first gene therapy approved anywhere in the world to be dosed by direct infusion into the brain using ClearPoint’s SmartFlow Cannula during a minimally invasive neurosurgical procedure,” commented Joe Burnett, President and CEO of ClearPoint Neuro. “We believe the precedent set by this regulatory approval provides a pathway for our 45 current and future pharmaceutical, academic and biotech partners. We are incredibly proud of our partnership with PTC and look forward to having our team continue in-person support of patients and providers during these procedures at neurosurgery centers around the world.” 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 s...