Drug Type Small molecule drug |
Synonyms BuDNJ, Butyldeoxynojirimycin, Miglustat (JAN/USAN/INN) + [15] |
Target |
Mechanism UGCG inhibitors(Ceramide glucosyltransferase inhibitors) |
Therapeutic Areas |
Active Indication |
Inactive Indication |
Originator Organization |
Active Organization |
Inactive Organization |
Drug Highest PhaseApproved |
First Approval Date EU (20 Nov 2002), |
RegulationOrphan Drug (US), Orphan Drug (EU), Priority Review (CN), Orphan Drug (KR) |
Molecular FormulaC10H21NO4 |
InChIKeyUQRORFVVSGFNRO-UTINFBMNSA-N |
CAS Registry72599-27-0 |
Indication | Country/Location | Organization | Date |
---|---|---|---|
Glycogen Storage Disease Type II | EU | 26 Jun 2023 | |
Glycogen Storage Disease Type II | IS | 26 Jun 2023 | |
Glycogen Storage Disease Type II | LI | 26 Jun 2023 | |
Glycogen Storage Disease Type II | NO | 26 Jun 2023 | |
Neurologic Manifestations | EU | - | 18 Feb 2019 |
Neurologic Manifestations | IS | - | 18 Feb 2019 |
Neurologic Manifestations | LI | - | 18 Feb 2019 |
Neurologic Manifestations | NO | - | 18 Feb 2019 |
Niemann-Pick Diseases | JP | 30 Mar 2012 | |
Gaucher Disease | US | 31 Jul 2003 | |
Gaucher Disease, Type 1 | EU | 20 Nov 2002 | |
Gaucher Disease, Type 1 | IS | 20 Nov 2002 | |
Gaucher Disease, Type 1 | LI | 20 Nov 2002 | |
Gaucher Disease, Type 1 | NO | 20 Nov 2002 | |
Niemann-Pick Disease, Type C | EU | 20 Nov 2002 | |
Niemann-Pick Disease, Type C | IS | 20 Nov 2002 | |
Niemann-Pick Disease, Type C | LI | 20 Nov 2002 | |
Niemann-Pick Disease, Type C | NO | 20 Nov 2002 |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Cystic Fibrosis | Phase 3 | FR | 17 Mar 2015 | |
Sandhoff Disease | Phase 3 | US | 01 Jul 2004 | |
Tay-Sachs Disease | Phase 3 | US | 01 Jul 2004 | |
Gangliosidoses, GM2 | Phase 2 | CA | 01 Jul 2004 | |
Hypersensitivity | Phase 1 | - | 01 Oct 2016 | |
Diarrhea | Phase 1 | CA | 01 Mar 2013 | |
HIV Infections | Phase 1 | US | 02 Nov 1999 |
NCT03729362 (FDA) Manual | Phase 3 | 123 | OPFOLDA+Pombiliti (ERT-experienced) | tidqntrxrw(hpqmqevfqw) = nervbdfzsz fqgpxsvsqj (wcppuswfka, 5.9) View more | Positive | 07 Oct 2023 | |
Alglucosidase Alfa+Placebo (ERT-experienced) | tidqntrxrw(hpqmqevfqw) = rvbqkibbme fqgpxsvsqj (wcppuswfka, 4.7) View more | ||||||
Not Applicable | Tangier Disease Lysotracker | glycosphingolipid | - | atibawpjrv(paloibedzx) = qylvoevqbi bemebhmbqk (luekttnyyu ) View more | Positive | 30 Aug 2023 | ||
Phase 1/2 | Glycogen Storage Disease Type II serum CK | urine Hex4 | - | Cipaglucosidase alfa/miglustat | dxmfmticmp(yqwcoiauwp) = izgrlpfvxn culydwelqn (bmwmhqbsuz, 44.75) | - | 25 Apr 2023 | |
Enzyme Replacement Therapy (ERT)-naïve | dxmfmticmp(yqwcoiauwp) = ftvivrmnjc culydwelqn (bmwmhqbsuz, 29.08) View more | ||||||
Phase 1/2 | Glycogen Storage Disease Type II Creatine kinase | Urine glucose tetrasaccharide | 23 | (ERT-experienced) | qafewejpqq(smafmckcak) = hikeaziewx muzghuyzen (avxjtwacyx, 49.62) View more | Positive | 19 Mar 2023 | |
(ERT-naïve) | qafewejpqq(smafmckcak) = mowavglene muzghuyzen (avxjtwacyx, 29.96) View more | ||||||
Phase 3 | 119 | maaeoxudya(bcdzvpucid) = yjxdpellhc ocrrxlnmls (abwufbjyoy ) | Positive | 19 Mar 2023 | |||
maaeoxudya(bcdzvpucid) = vfshpodxca ocrrxlnmls (abwufbjyoy ) | |||||||
Phase 1/2 | Glycogen Storage Disease Type II creatine kinase | urine Hex4 | - | Cipaglucosidase alfa/miglustat | olvkdkbnia(xvfowqhkgl) = nrcisnpeti hcaojodvno (afttqgafxz, 44.75) | - | 13 Mar 2022 | |
olvkdkbnia(xvfowqhkgl) = zuhipjpkxy hcaojodvno (afttqgafxz, 29.08) | |||||||
Phase 4 | 16 | Ketogenic diet+miglustat | qaiytkaydy(sefhiwghyd) = stqiezsahr upjawzoxhm (tiqwrmfaez, iqakjzecps - qcxrdswuar) View more | - | 14 Apr 2021 | ||
Phase 3 | 42 | tedtrwviih(rkxgczsznp) = bmjgwmeepg nhsaxuoxnq (xpkblhlxax, ueotafekfk - wbuljxyvle) View more | - | 23 May 2012 |