A report from the Institute for Clinical and Economic Review (ICER) has provided a negative cost-effectiveness appraisal of Spark Therapeutics’ (Nasdaq: ONCE) orphan gene therapy Luxturna (voretigene neparvovec).
The novel drug was given the nod late last year, becoming the first US Food and Drug Administration-approved directly administered gene therapy that targets a disease caused by mutations in a specific gene.
Spark has since announced the one-time treatment will carry a list price of $850,000, or $425,000 per eye. The therapy treats a form of blindness that affects up to 2,000 people in the USA.
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