ATMPs in Germany - Libmeldy®

👉 Quantifiable additional benefit without evidence from RCTs - HTA on Libmeldy demonstrates that a sibling analysis with dramatic effects can justify a considerable additional benefit.

Libmeldy (active substance: atidarsagene autotemcel) was authorized in October 2020 for treatment of metachromatic leukodystrophy (MLD). The ATMP Libmeldy is an autologous CD34⁺ cell-based gene therapy. The pivotal study 201222 was a non-randomized single center study. In this trial 22 patients with MLD were treated with Libmeldy and compared to a cohort of 31 untreated siblings, who also suffer from MLD (study TIGET NHx). Libmeldy is available in Germany.

🏆German HTA rating:

German HTA rating in November 2021 resulted in a hint for a considerable additional benefit for one subpopulation. The additional benefit of Libmeldy is based on a dramatic effect in prevention of severe motor impairment compared to the natural course of the disease in siblings (dramatic effect = ~ 50 to 90 % lower risk for an unfavorable event). ➡️ Of note: The German HTA body sliced the population into two subgroups on its own discretion as data from siblings were only available for one subpopulation. This strengthened the structural equality and was probably one of the reasons to allow quantification of the additional benefit.

The HTA resolution on Libmeldy was initially limited to July 2024, because final long-term data were still expected. The limitation was afterwards extended to July 2026, as the original timeline for finalization could not be met. Both limitations were in line with conditions imposed by the EMA.

💵 Launch price: € 2,875,000 per infusion. Libmeldy is a one-time treatment. Reimbursement price: € 2,350,000 (two price reductions, only one price reduction seems to be associated with a result of an HTA). Cumulated rebate: 18 %.

📘 Special features of reimbursement:

Libmeldy is reimbursed. As Libmeldy is an inpatient treatment only, it is reimbursed via NUB fees (📍see previous post on Luxturna). Already 4 months after launch in Germany and before price negotiation, the rebate contract coverage was above 95 %, most likely to facilitate market entry.

💡 Sibling analysis are relevant for German HTA. These analyses allowed quantification of an additional benefit even without randomization and with limited patient numbers. Slicing the population of Libmeldy by the German HTA body into different subpopulations presumably led to a higher additional benefit and to a more favorable position in price negotiation.