Orphan designation: allogeneic human induced pluripotent stem cell-derived photoreceptor precursor cells Treatment of syndromic inherited retinal dystrophies of the rod-dominant phenotype, 20/05/2026 Positive

Orphan designation: (S)-N-(2-(2-Cyclopropylpyridin-4-yl)-4,5,6,7-tetrahydrobenzo[d]oxazol-4-yl)-1-methyl-3-(trifluoromethyl)-1H-pyrazole-5-carboxamide Treatment of KCNT1-related epilepsy, 20/05/2026 Positive

Orphan designation: antisense oligonucleotide against USH2A pre-mRNA Treatment of inherited retinal disease due to dysfunction in the USH2A gene, 20/05/2026 Positive

Orphan designation: quemliclustat Treatment of pancreatic cancer, 20/05/2026 Positive

Orphan designation: igermetostat maleate Treatment of peripheral T-cell lymphoma, 20/05/2026 Positive

Orphan designation: serabelisib Treatment of PIK3CA related overgrowth spectrum, 20/05/2026 Positive

EMA/PE/0000235869

EMA/PE/0000239491

EMA/PE/0000222352

EMA/PE/0000240548