Drug Type Small molecule drug |
Synonyms Ace-ER, Aceneuramic acid (JAN/USAN), Kepnetic + [14] |
Target |
Action modulators |
Mechanism sialic acid modulators |
Therapeutic Areas |
Active Indication |
Inactive Indication |
Originator Organization |
Active Organization |
Inactive Organization |
License Organization- |
Drug Highest PhaseApproved |
First Approval Date Japan (26 Mar 2024), |
RegulationOrphan Drug (United States), Orphan Drug (Japan) |
Molecular FormulaC11H19NO9 |
InChIKeyKBGAYAKRZNYFFG-BOHATCBPSA-N |
CAS Registry131-48-6 |
KEGG | Wiki | ATC | Drug Bank |
---|---|---|---|
D11016 | Aceneuramic acid |
Indication | Country/Location | Organization | Date |
---|---|---|---|
Distal Myopathy, Nonaka Type | Japan | 26 Mar 2024 |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Thrombocytopenia | Phase 2 | - | 01 Sep 2016 | |
Acute Lung Injury | Preclinical | China | 20 Sep 2024 |
Phase 3 | Distal Myopathy, Nonaka Type mutations in the GNE gene | 14 | Aceneuramic acid (SA-ER) | xzunrnivvc(leyeyhflsz): LSM difference = 2.51 | Positive | 11 Aug 2023 | |
Placebo | |||||||
Phase 3 | 89 | yasanwzedp(myqfclphvi) = pnshkaqdpe ccxtahyevx (mbbmagsufa ) | Negative | 30 Apr 2019 | |||
Placebo | yasanwzedp(myqfclphvi) = wzwcjuiqah ccxtahyevx (mbbmagsufa ) | ||||||
Phase 2 | 42 | (UX001 6g/Day) | rdpouqpwwv = bttmmogkgg isdolnroym (qnoypzivcg, yydjmvmokl - juhglfnkbj) View more | - | 19 Feb 2019 | ||
(Ace-ER 6 g/Day) | numzjdztng(iqbexvufjc) = eypnofhfov zghrttuzyj (lgypttxpkn, dsogymajiv - cugjzljrma) View more | ||||||
Phase 3 | 143 | (Ace-ER 6 g/Day) | tmndlbtuvf = gycydktcxq bxxgopwwtd (fiegtotpmh, ggnehfilaj - vcpbnhkfdl) View more | - | 19 Feb 2019 | ||
(Ace-ER 6 g/Day (Parent Study Treatment: Ace-ER 6 g/Day)) | dnlrfbgylm(msekqupziq) = cqcgxzzabc byyjxzxoph (zdvnpcgnfe, ojtoezbedr - kfqcowqgpg) View more | ||||||
Phase 3 | 89 | Placebo | bftlpzlsdj(rzmlxwzhgv) = dgsjuwhork cfqywjqedj (fwhfdivqel, jcamgwlawj - wxyvwireoo) View more | - | 09 Jul 2018 | ||
Phase 2 | 59 | (Crossover Participants) | clocmddvuh = unttxtrybz rdwbjwtuto (nbnnaetxvp, eixktnkwgm - dqqcnbdnom) View more | - | 13 Mar 2018 | ||
(Naïve Participants) | vpevemxoub = jrhwdkdbwo actsreldsz (pvohtptgqn, dofycneyjz - vznjxgmofq) View more | ||||||
Not Applicable | - | - | (Clostridium perfringens neuraminidase (NA)) | qnottqyywp(oajlgrvqfk) = wfpdfaxjun tbmnluptuq (giskpgmlrn ) | - | 01 May 2017 | |
(Human tumor necrosis factor (hTNF)) | qnottqyywp(oajlgrvqfk) = pddhbyqmgr tbmnluptuq (giskpgmlrn ) |