Advances in orphan drug development for alpha-1 antitrypsin deficiency: a 2025 update from the FDA and EMA
The development of new therapies may offer expanded treatment options for patients with AATD in the future. In addition to pulmonary manifestations, extrapulmonary manifestations could also be treated in the future.
Authors
- Katharina Buschulte (ORCID: https://orcid.org/0000-0002-7598-5276)
- Felix Herth (ORCID: https://orcid.org/0000-0002-7638-2506)
- Konstantina Kontogianni (ORCID: https://orcid.org/0000-0001-9798-9879)
- Arturo Olivares Rivera
- Sebastian Fähndrich (ORCID: https://orcid.org/0000-0002-2277-2672)
- Franziska Christina Trudzinski (ORCID: https://orcid.org/0000-0002-0980-7586)
- Markus Ries (ORCID: https://orcid.org/0000-0002-5054-5741)
- Hilal Ersöz
- Philipp Höger (ORCID: https://orcid.org/0009-0006-6637-9166)
Institutions
- Heidelberg University (DE)
- University Medical Center Freiburg (DE)
- University Hospital Heidelberg (DE)
- German Center for Lung Research (DE)
- Heidelberg University (US)
Publication Details
- Journal
- Therapeutic Advances in Respiratory Disease
- Published
- 2026-01-01
- DOI
- https://doi.org/10.1177/17534666251411227
- Primary Topic
- Protease and Inhibitor Mechanisms
- Type
- article
- Field-Weighted Citation Impact
- 0.00