Novartis’s gene therapy Luxturna receives ‘considerable added benefit’ in Germany

 

Luxturna (voretigen neparvovec), a gene therapy developed by Novartis, received ‘considerable added benefit’ from G-BA, the German health technology assessment. Luxturna is a one-off treatment for vision loss in adults and children, caused by retinal dystrophy from mutations in both copies of the RPE65 gene. The orphan disease is ultra-rare, affecting approximately 1 in 200,000 people worldwide. Upon EMA approval in Europe in 2018, Luxturna was launched in Germany in April 2019 at the manufacturer price of €345,000 per eye per patient.

G-BA motivated their decision by Luxturna’s favourable clinical efficacy: improvement of functional vision/orientation, light sensitivity and perimetry. As a next step per the AMNOG law, Novartis will carry out negotiations with GKV-Spitzenverband (GKV-SV), Germany’s payer umbrella group, expecting a new, reimbursed price by April 2020.

In addition, G-BA mandated a reassessment in January 2022 to evaluate Luxturna’s long-time safety and efficacy. The reassessment will be based on data from PASS, the post-approval safety study registry and an ongoing, one-armed observational trial, the 302 study. The reassessment request falls into the remit of GSAV, recently (August 2019) introduced law on better safety in drug supply. Per the GSAV, G-BA can request post-launch data, triggering a reassessment and new price negotiations for drugs with “limited scientific evidence”, such as orphan drugs.

 

By Jack Ziomek – Project Manager, Web Platforms

Link to G-BA’s decision: https://www.g-ba.de/downloads/40-268-6053/2019-10-17_AM-RL-XII_Voretigen-Neparvovec_D-436_TrG.pdf