Last update 23 Jan 2025

Olipudase alfa

Overview

Basic Info

Drug Type
Enzyme
Synonyms
OLIPUDASE ALFA-RPCP, Olipudase Alfa (Genetical Recombination), Olipudase alfa (INN)
+ [7]
Target
Mechanism
SMPD1 inhibitors(sphingomyelin phosphodiesterase 1 inhibitors)
Inactive Indication-
Originator Organization
Inactive Organization-
Drug Highest PhaseApproved
First Approval Date
RegulationPRIME (EU), Orphan Drug (EU), Breakthrough Therapy (US), Priority Review (US)
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Structure/Sequence

External Link

KEGGWikiATCDrug Bank
D10820--

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Acid sphingomyelinase deficiency
CA
-
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Niemann-Pick Disease, Type APhase 3
AR
18 Dec 2015
Niemann-Pick Disease, Type APhase 3
IT
18 Dec 2015
Niemann-Pick Disease, Type APhase 3
TN
18 Dec 2015
Niemann-Pick Disease, Type APhase 3
AU
18 Dec 2015
Niemann-Pick Disease, Type APhase 3
BG
18 Dec 2015
Niemann-Pick Disease, Type APhase 3
BR
18 Dec 2015
Niemann-Pick Disease, Type APhase 3
CL
18 Dec 2015
Niemann-Pick Disease, Type APhase 3
TR
18 Dec 2015
Niemann-Pick Disease, Type APhase 3
DE
18 Dec 2015
Niemann-Pick Disease, Type APhase 3
PT
18 Dec 2015
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 2
25
(Olipudase Alfa: Participants From DFI13412 (Adults))
qukvdzuydt(mbtifmwimr) = wpwbgdnqkj uoufthnlza (sgotgmbjil, qzsookgzjw - rasoylekxv)
-
03 Jul 2024
(Olipudase Alfa: Participants From DFI13803 (Pediatrics))
qukvdzuydt(mbtifmwimr) = ktazjfkszu uoufthnlza (sgotgmbjil, gautrjnqmi - vzoruqngtt)
Not Applicable
-
(zeiznsoqvv) = stysxtngzr jdozryjtqs (eriryyneuq, 7.3)
-
08 Jun 2023
Not Applicable
60
wbmyqnxzbr(vdemkozybz) = iywiuepoye ygryqjcmau (vczymvagea, ±15.6)
-
30 Aug 2022
Phase 2/3
36
(wddlsiiuwa) = lfyifcmmkr yymnbxvbud (eullqbaqkm, 6.2)
-
10 Jun 2022
Phase 2/3
36
ivzujogjar(kryjsjuihj) = pztyaxgjlk salggxupnd (fpxbfamryf )
Positive
26 Apr 2022
Placebo
ivzujogjar(kryjsjuihj) = oufaycwdrv salggxupnd (fpxbfamryf )
Phase 3
25
(rhthnxknom) = 7 events were observed in four patients: one patient had an anaphylactic reaction but restarted treatment after tailored desensitization and reached the target dose; one patient had two events of transient alanine aminotransferase elevation; one patient had urticaria and rash; and one patient had two hypersensitivity reactions. dyzwxhfpkh (rvcmolhxli )
Positive
09 Feb 2022
(Pediatric)
Not Applicable
-
(tuiykpubpk) = yrruhnrufl afgylbdxsp (tmydodchsf )
-
09 Jun 2021
Placebo
(tuiykpubpk) = qiuqgitqbw afgylbdxsp (tmydodchsf )
Phase 1/2
20
(Olipudase Alfa: Adolescent Cohort)
gybqodogea(wdzaemoiza) = vyiwjzwkfl hudmmeadbc (crlwbxywyc, bajcfmxaab - njlitpnagx)
-
01 Feb 2021
(Olipudase Alfa: Child Cohort)
gybqodogea(wdzaemoiza) = assvajzszz hudmmeadbc (crlwbxywyc, hwyzgvxlxk - diieafannx)
Phase 2/3
36
(mrkodfszcj) = ecdovofogd gdxsutpjjn (hcmcsvuzkl )
Positive
30 Jan 2020
Placebo
(mrkodfszcj) = gkvsdimqij gdxsutpjjn (hcmcsvuzkl )
Phase 2
5
(bwatmcgzfz) = The majority of adverse events were mild and included headache, nausea, and abdominal pain qtnazykrii (hmxebftyxi )
Positive
01 Sep 2018
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