Here are the G-BA decisions from the G-BA meeting held on 16 May 2024 on the early benefit assessments of Darzalex, Kimmtrak, Voxzogo, Kaftrio+Kalydeco, Enhertu, Ryeqo, Epidyolex, and Onpattro.
A complete database of all previous G-BA resolutions is available upon request.
- Darzalex (daratumumab); reassessment after expiry of previous resolution: multiple myeloma, first-line, ineligible for stem cell transplantation, in combination with bortezomib, melphalan and prednisone – Indication of considerable additional benefit
- Kimmtrak (tebentafusp); reassessment of an orphan drug after exceeding 30 million annual sales threshold: Uveal melanoma, HLA-A*02:01 positive – Hint of considerable additional benefit
- Voxzogo (vosoritide); new indication: achondroplasia, ≥ 4 months to < 2 years – reassessment of an orphan drug after exceeding 30 million annual sales threshold – Hint of not-quantifiable additional benefit
- Kaftrio + Kalydeco (ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor); new indication: cystic fibrosis, from 2 to ≤ 5 years
- homozygous for F508del mutation – Hint of not-quantifiable additional benefit
- heterozygous for F508del and MF mutation – Hint of not-quantifiable additional benefit
- heterozygous for F508del and gating mutation – Additional benefit not proven
- heterozygous for F508del and RF mutation – Additional benefit not proven
- heterozygous for F508del and other or unknown mutation – Additional benefit not proven
- Enhertu (trastuzumab deruxtecan); new indication: non-small cell lung cancer, HER2 (ERBB2) mutation, previously treated – Additional benefit not proven in both subgroups
- Ryeqo (relugolix/estradiol/norethisterone acetate); new indication: endometriosis, after medical or surgical treatment – Additional benefit not proven
- Epidyolex (cannabidiol); patients aged ≥ 2 years; reassessment of an orphan drug after exceeding 30 million annual sales threshold:
- Dravet syndrome; in combination with clobazam – Additional benefit not proven
- Lennox-Gastaut syndrome; in combination with clobazam – Additional benefit not proven
- seizures associated with tuberous sclerosis – Additional benefit not proven
- Onpattro (patisiran); reassessment of an orphan drug after exceeding 30 million annual sales threshold: hereditary transthyretin amyloidosis with polyneuropathy (stage 1 or 2) – Indication of a lesser (inferior) benefit
- Tecfidera (dimethyl fumarate); new indication: relapsing-remitting multiple sclerosis, ≥ 13 years – Removal of time restriction applied to previous resolution
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