Project Lead Claudia Wild
Author: Ozren Sehic
Publication: June 2022
Language: English (with German summary)
This report is an update of a previous Horizon Scanning report from 2020, which identified 32 ATMPs. Since August 2020, six of these new ATMPs have been approved by the EMA and five more will be decided by 2023. According to market analyses, 535 ATMPs will be investigated in phase 1 to 3 in 2022, with a further 1,451 in preclinical stages. Coagulation factor VIII (haemophilia A) remains the most common target for non-oncology indications and coagulation factor IX (haemophilia B) has risen to become the second most common indication.
To answer the questions, a systematic search of trial registries was conducted to identify gene therapies and ATMPs in development, followed by extraction of data on identified ongoing clinical trials (limited to phase 2/3 and 3), complemented by a search of the EMA database of medicines under assessment to identify those therapies that are close to approval. Published information on the products and the current development/regulatory status was then collected and subsequently converted into short "vignettes".