👉 First considerable additional benefit for an ATMP – the additional benefit for Luxturna is based on a pivotal RCT and supportive results from long-term single-arm extension studies.

Luxturna (active substance: voretigene neparvovec) was approved in September 2018 for treatment of patients with vision loss due to inherited retinal dystrophy. Luxturna is an adeno-associated virus-based gene therapy. The pivotal study AAV2-hRPE65v2-301 was an open-label, randomized phase 3 study with a 15-year extension phase (study -302). In this trial 31 patients were either treated with Luxturna or not treated (“watchful-waiting”). Luxturna is available in Germany.

🏆German HTA rating:

To date Luxturna was assed twice. 

1 Initial German HTA rating in October 2019 resulted in a hint for a considerable additional benefit due to statistically significant and clinically relevant benefits in favor of Luxturna. Long-term data showed efficacy for up to 3 years.

The German HTA body commented the submitted evidence in the following way:

✅ The endpoints “light sensitivity via multi-luminance mobility test (MLTM)”, “full-field stimulus threshold testing (FST)”, “visual acuity using ETDRS/HOTV vision chart” and “visual field measured” are patient-relevant and can be used for HTA.

✅ Single-arm study data of the extension study were taken into account to assess sustainability of the effects.

The HTA resolution on Luxturna was limited because the German HTA body wished to reassess further long-term data.

2  Re-assessment rating in September 2022 resulted in a hint for a considerable additional benefit, as well. Again, the extension study confirmed the long-term efficacy of Luxturna for at least 5 years.

💵 Launch price: € 345,000 per solution for injection. Luxturna was approved as a single injection per eye. Reimbursement price: € 280,000 (two price reductions, one for each HTA). Cumulated rebate: 18,8 %.

📘 Special features of reimbursement:

Luxturna is reimbursed. As Luxturna is an inpatient treatment only, it is reimbursed via fees for the so-called new examination and treatment methods (“neue Untersuchungs- und Behandlungsmethoden“, NUB). NUB fees are temporary reimbursements for high-priced product innovations used in hospitals. The ATMPs Kymriah and Yescarta are reimbursed via NUB fees as well.

💡 Endpoints measuring visual acuity are considered patient-relevant and can be used to derive an additional benefit. Interestingly, long-term data from a single-arm study had a substantial impact on the extent of the additional benefit. Submitting such data is advisable, in case the German HTA body might question the sustainability of the efficacy.