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Living with DMD, Ukraine
Results Show Long-Lasting and Holistic Improvements in Children with AADC Deficiency Treated with PTC-AADC Gene Therapy
"The transformations we are seeing in the children are remarkable, as they could not even lift their heads before treatment," said
The new, five-year results show that motor function improvements were sustained, demonstrating that the treatment effect of PTC-AADC is durable, which is highly important for a single administration therapy for a chronic, fatal disease.1-2 Across three clinical trials, improvements in motor development were recorded in all children from as early as 3 months.1 Cognitive and language skills were also reported to improve significantly from baseline, as measured by Bayley-III scores, with children able to understand their caregivers and express themselves.2
In addition, the rate of respiratory infection declined from an average of 2.4 episodes per year at 12 months to 0.6 episodes per year at 2 years and 0.3 episodes per year at 5 years.3 Almost all treated children went from a baseline weight below the third percentile to making age-appropriate weight gains by 12 months following gene therapy treatment.3
There are currently no approved disease-modifying therapies for AADC deficiency, a fatal disorder caused by a defective gene that stops the body from producing the dopamine needed to develop and function normally. Delivering PTC-AADC gene therapy directly to the putamen, a site in the brain where dopamine is utilized, corrects this defect.4
"The impact that this one-time gene therapy has had on children with AADC deficiency in these trials is truly life-changing," said investigator
PTC-AADC is currently under review by the
About aromatic L-amino acid decarboxylase (AADC) deficiency
AADC deficiency is a fatal, rare genetic disorder that 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 is exacerbated by episodes of distressing seizure-like oculogyric crises, which can happen daily and last for hours, causing the eyes to roll up in the head, frequent vomiting, behavioral problems, difficulty sleeping, and life-threatening complications such as respiratory infections and gastrointestinal problems.
Current management options yield limited improvement for the majority of patients with AADC deficiency. Managing patients with AADC deficiency requires a multidisciplinary team of specialists and complex coordination of care to address significant health issues, including developmental delays, infections, orthopaedic and cardiac complications, and other comorbidities
While several diagnostic tests for AADC deficiency are available, the condition remains largely undiagnosed.
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As with any pharmaceutical under development, there are significant risks in the development, regulatory approval, and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including PTC-AADC.
The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.
1 Hwu PW-L, et al.
2 Hwu PW-L, et al. Gene Therapy With Eladocagene Exuparvovec Improves Cognition and Language in Patients With Aromatic L-Amino Acid Decarboxylase Deficiency. Poster presented at the CNS Annual Meeting;
3 Hwu PW-L, et al. Eladocagene Exuparvovec Improves Body Weight and Reduces Respiratory Infections in Patients With Aromatic L-Amino Acid Decarboxylase Deficiency. Poster presented at the CNS Annual Meeting;
4 Chien YH, et al. Efficacy and safety of AAV2 gene therapy in children with aromatic L-amino acid decarboxylase deficiency: an open-label, phase 1/2 trial.
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