NICE Recommends Upstaza for Rare Genetic Disorder

The UK’s National Institute for Health and Care Excellence (NICE) has recommended PTC Therapeutics’ Upstaza (eladocagene exuparvovec) for National Health Service (NHS) use in treating patients with severe cases of aromatic L-amino acid decarboxylase deficiency.

The rare genetic disorder affects motor development and is associated with high risk of death in childhood.

Infused directly into the brain as a single dose, Upstaza is a gene therapy designed to correct the underlying genetic defect.

NICE noted that the supporting clinical trial data showed the treatment improved motor development but only provided limited information on non-motor outcomes.

The list price of Upstaza is more than $3.6 million for a 0.5 ml solution for infusion. The negotiated price for NHS use was not disclosed.