Orphan designation: encaleret Treatment of hypoparathyroidism, 15/10/2021 Positive
Submitted by Anonymous (not verified) on 11 May 2026 - 9:16
Orphan designation: encaleret Treatment of hypoparathyroidism, 15/10/2021 Positive
Orphan designation: eltanexor Treatment of myelodysplastic syndromes, 21/06/2022 Positive
Submitted by Anonymous (not verified) on 11 May 2026 - 9:07
Orphan designation: eltanexor Treatment of myelodysplastic syndromes, 21/06/2022 Positive
Orphan designation: (Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N'-(pyrazin-2-yl)acrylohydrazide Treatment of diffuse large B-cell lymphoma, 22/08/2014 Positive
Submitted by Anonymous (not verified) on 11 May 2026 - 9:05
Orphan designation: (Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N'-(pyrazin-2-yl)acrylohydrazide Treatment of diffuse large B-cell lymphoma, 22/08/2014 Positive
Orphan designation: selinexor Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma, 19/11/2014 Positive
Submitted by Anonymous (not verified) on 11 May 2026 - 9:03
Orphan designation: selinexor Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma, 19/11/2014 Positive
Orphan designation: selinexor Treatment of myelofibrosis, 11/10/2022 Positive
Submitted by Anonymous (not verified) on 11 May 2026 - 9:01
Orphan designation: selinexor Treatment of myelofibrosis, 11/10/2022 Positive
Orphan designation: selinexor Treatment of glioma, 15/10/2021 Positive
Submitted by Anonymous (not verified) on 11 May 2026 - 9:00
Orphan designation: selinexor Treatment of glioma, 15/10/2021 Positive
Orphan designation: (Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N'-(pyrazin-2-yl)acrylohydrazide Treatment of acute myeloid leukaemia, 22/08/2014 Positive
Submitted by Anonymous (not verified) on 11 May 2026 - 8:59
Orphan designation: (Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N'-(pyrazin-2-yl)acrylohydrazide Treatment of acute myeloid leukaemia, 22/08/2014 Positive
Orphan designation: beta-lapachone Treatment of primary sclerosing cholangitis, 20/06/2025 Positive
Submitted by Anonymous (not verified) on 11 May 2026 - 8:55
Orphan designation: beta-lapachone Treatment of primary sclerosing cholangitis, 20/06/2025 Positive
Orphan designation: pridopidine hydrochloride Treatment of amyotrophic lateral sclerosis, 19/07/2021 Positive
Submitted by Anonymous (not verified) on 11 May 2026 - 8:53
Orphan designation: pridopidine hydrochloride Treatment of amyotrophic lateral sclerosis, 19/07/2021 Positive
Orphan designation: 4-[3-(Methylsulfonyl)phenyl]-1-propylpiperidine x HC1 (pridopidine hydrochloride) Treatment of Huntington’s disease, 20/06/2005 Positive
Submitted by Anonymous (not verified) on 11 May 2026 - 8:51
Orphan designation: 4-[3-(Methylsulfonyl)phenyl]-1-propylpiperidine x HC1 (pridopidine hydrochloride) Treatment of Huntington’s disease, 20/06/2005 Positive