Every medicine the European Commission has designated as an orphan medicine for a rare disease, on the EMA's recommendation, with its current status.
| Substance | Intended use | Status | Designated |
|---|---|---|---|
| (14S)-8-[3-(2-{dispiro[2.0.24.13]heptan-7-yl}ethoxy)-1H-pyrazol-1-yl]-12,12-dimethyl-2lamba6-thia-3,9,11,18,23-penta-azatetracyclo[17.3.1.111,14.05,10]tetracos… EU/3/21/2527 · now authorised as Alyftrek 2 clinical trials → |
Treatment of cystic fibrosis | Active (positive) | 12 Nov 2021 |
Every row is copied from the EMA's orphan designations data, reproduced with acknowledgement of the source. Each substance links to its official EMA page.
A daily job downloads the EMA's orphan designations JSON report and lists every designation, newest first. No AI drafting and no editorial pass. See our editorial standards. The the EMA data on this page was last retrieved on 2 Oct 2026. How every register is built: methodology · fixes we have made: corrections log.