ATMPs in Germany - Zolgensma®

👉 HTA for Zolgensma gives insights on the legal option of the German HTA body to oblige routine practice data collection and reassessment.

Zolgensma (active substance: onasemnogene abeparvovec) was approved in March 2020 for treatment of patients with spinal muscular atrophy (SMA). Zolgensma is an adeno-associated virus-based gene therapy. The pivotal study CL-303 is a single-arm study that enrolled 22 patients. Zolgensma is available in Germany.

🏆German HTA rating:

To date Zolgensma was assed only once. Initial German HTA was suspended in December 2020 as Zolgensma exceeded the sales threshold for orphan drugs (📍see previous post on orphan drug reassessment). The German HTA without orphan drug privilege started in May 2021 and resulted in November 2021 in no additional benefit, because no RCT comparing Zolgensma vs. the appropriate comparator Spinraza exists.

💵 Launch price: € 1,945,000. Zolgensma is a one-time treatment. Reimbursement price: € 1,385,000 (two price reductions, only one price reduction seems to be associated with the result of an HTA). Cumulative Rebate: 29 %.

📘 Special features of reimbursement:

Zolgensma is subject to routine practice data collection (a specific kind of RWE collection). This obligation restricts the prescription of Zolgensma to physicians, who participate in the required data collection (📍learn more in the forthcoming post).

Zolgensma is also subject to the ATMP Quality Assurance Guideline (like Kymriah and Yescarta).

💡 If an orphan drug exceeds the sales threshold during the HTA, the assessment will be suspended and a new HTA is initiated. Even though no additional benefit was granted for Zolgensma, Zolgensma itself is now part of the appropriate comparative therapies in SMA (compare HTA resolution for Evrysdi from March 2024).

💡The obligation for routine practice data collection restricts the use of Zolgensma to physicians, who participate in the required data collection. This might hinder fast market entry.