Starting from more than 200 medicines in the pipeline (status January 2024) mentioned in The High Impact Reports of the International Horizon Scan Initiative, a selection of impactful medicines was made. Criteria such as new chemical entity and potential game changer in clinical prescription were applied. The members of the Beneluxa Initiative established the following list of medicines, in alphabetic order, for which the Beneluxa Initiative would be interested to start a joint and full assessment:
| Active principle | Condition | Pharmaceutical company |
1. | blarcamesine | Rett syndrome | Anavex Life Sciences |
2. | delandistrogene moxeparvovec | Duchenne muscular dystrophy | Hoffmann-La Roche |
3. | encaleret | hypoparathyroidism | Bridge Bio Pharma |
4. | ganaxolone | CDKL5 deficiency disorder | Marinus Pharmaceuticals |
5. | givinostat | Duchenne muscular dystrophy | Italfarmaco |
6. | hypericin | Cutaneous T-cell lymphoma | Minoryx Therapeutics |
7. | leniolisib | Activated PI3 kinase delta syndrome | Pharming Group |
8. | leriglitazone | Adrenoleukodystrophy | Minoryx Therapeutics |
9. | lonafarnib | Hepatitis D | Eiger Bio Pharmaceutica |
10. | marnetegragene autotemcel | Leukocyte adhesion deficiency type I | Rocket Pharmaceuticals |
11. | N-acetyl-L-leucine | Niemann-Pick disease | IntraBio |
12. | sparsentan | IgA nephropathy | CSL Vifor |
13. | trofinetide | Rett syndrome | Acadia Pharmaceuticals |
If pharmaceutical companies are interested in the possibility of a joint submission for a joint HTA procedure across jurisdictions, the Beneluxa Initiative is willing to organize an exploratory meeting.
It is to be stressed that, beyond this list, Beneluxa remains open for other medicines for which companies seek to submit jointly in more than one member state.
Last update: 20 January 2025