ATMPs in Germany - Skysona®

👉 Skysona was never launched in Germany as the HTD closed operations in Europe, partly because failing to convince European payers to adequately reimburse Zynteglo.

Skysona (elivaldogene autotemcel) was authorized in July 2021 and was indicated for the treatment of cerebral adrenoleukodystrophy (CALD). The gene therapy Skysona contains hematopoietic stem cells transduced with a lentiviral vector encoding the gene for the human adrenoleukodystrophy protein. The safety and efficacy of Skysona were assessed in a single-arm study in patients with CALD (ALD-102, N = 32) and compared to the efficacy and safety of allogenic stem cell transplantation in a contemporaneous comparator study (ALD-103, N = 59).

The authorization of Skysona was withdrawn in November 2021, just four months after authorization was granted. Consequently, Skysona was never available in Germany.

🏆German HTA rating and 💵 price: As the ATMP Skysona was never launched in Germany neither a price was listed nor an HTA was started.

💡 Adequate agreements on reimbursement and reimbursement prices impact availability of future therapeutic innovations.