NICE Recommends Novel Gene Therapy for Rare Genetic Brain Disorder

The UK’s National Institute for Health and Care Excellence (NICE) has issued a draft guidance recommending the approval of Upstaza, PTC Therapeutic’s gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency. The gene therapy, if approved, would cost £3 million and would be the only treatment for the ultra-rare disorder.

According to Zoey Becker, “AADC deficiency is so rare that it affects an estimated 10 children in the U.K., only a few of whom could be eligible for the treatment. The disease causes a wide range of severe symptoms, but around 80% of patients have a severe form that leaves them fully dependent on caregivers and unable to meet normal developmental milestones.”

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(Source: Fierce Pharma, March 23^rd, 2023)