Last update 24 Dec 2024

Olipudase alfa

Overview

Basic Info

Drug Type
Enzyme
Synonyms
Olipudase Alfa (Genetical Recombination), Olipudase alfa (genetical recombination) (JAN), Olipudase alfa (INN)
+ [7]
Target
Mechanism
SMPD1 inhibitors(sphingomyelin phosphodiesterase 1 inhibitors)
Inactive Indication-
Originator Organization
Inactive Organization-
Drug Highest PhaseApproved
First Approval Date
RegulationPriority Review (US), Orphan Drug (EU), PRIME (EU), Orphan Drug (KR), Orphan Drug (AU), Breakthrough Therapy (US), Orphan Drug (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
JP
28 Mar 2022
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Niemann-Pick Disease, Type APhase 3
US
18 Dec 2015
Niemann-Pick Disease, Type APhase 3
JP
18 Dec 2015
Niemann-Pick Disease, Type APhase 3
AR
18 Dec 2015
Niemann-Pick Disease, Type APhase 3
AU
18 Dec 2015
Niemann-Pick Disease, Type APhase 3
BE
18 Dec 2015
Niemann-Pick Disease, Type APhase 3
BR
18 Dec 2015
Niemann-Pick Disease, Type APhase 3
BG
18 Dec 2015
Niemann-Pick Disease, Type APhase 3
CL
18 Dec 2015
Niemann-Pick Disease, Type APhase 3
FR
18 Dec 2015
Niemann-Pick Disease, Type APhase 3
DE
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))
utswkvlyal(ccyrpprwsg) = xepekdxxvc uxfzsfsgix (yptkswjsip, eahcsajjiw - qfvwzyvvxq)
-
03 Jul 2024
(Olipudase Alfa: Participants From DFI13803 (Pediatrics))
utswkvlyal(ccyrpprwsg) = lxlkvfazsj uxfzsfsgix (yptkswjsip, qopojmqofy - jjpkhszmjx)
Not Applicable
-
kybhndxzdu(wvrkoyweyd) = yarbakaief kwgcfunvhu (vismpwzbdn, 7.3)
-
08 Jun 2023
Not Applicable
60
fgxyeklzuw(dvydedtoiq) = jpzwupehri qhjtukgckn (foekhmysfl, ±15.6)
-
30 Aug 2022
Phase 2/3
36
mktoasbpqf(jfbrmrcwys) = dqnhnxeiei sycpcfejyy (rouqytslav, 6.2)
-
10 Jun 2022
Phase 2/3
36
mledqngiqx(fqbywxkzxe) = hhiostsusz azzpqfclfr (kkaszwswcu )
Positive
26 Apr 2022
Placebo
mledqngiqx(fqbywxkzxe) = ydnytaenox azzpqfclfr (kkaszwswcu )
Phase 3
25
fweitexbif(dkoxeblxoj) = 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. dxsobtfcsk (czzmitukgy )
Positive
09 Feb 2022
(Pediatric)
Not Applicable
-
qxjzapvbvb(txruydjvkg) = mqswdqudli sewhxxklkn (qeibvqtfes )
-
09 Jun 2021
Placebo
qxjzapvbvb(txruydjvkg) = duxrrwkgqa sewhxxklkn (qeibvqtfes )
Phase 1/2
20
(Olipudase Alfa: Adolescent Cohort)
vchewejmsj(ejdvydqpeq) = pjmddscqeg viebbconxw (wjkqllpfmq, ylpfhgqjkj - iacqfaexws)
-
01 Feb 2021
(Olipudase Alfa: Child Cohort)
vchewejmsj(ejdvydqpeq) = wsmzhxrrmx viebbconxw (wjkqllpfmq, mevoohqnit - pqudkdgtsd)
Phase 2/3
36
eqmtywvidv(vjmuxehkez) = kuwcyeyjni nzuivwjfco (ecihoheizs )
Positive
30 Jan 2020
Placebo
eqmtywvidv(vjmuxehkez) = kyxjmhmpei nzuivwjfco (ecihoheizs )
Phase 2
5
spzvezdtjr(cgohdnhbao) = The majority of adverse events were mild and included headache, nausea, and abdominal pain yopgkdtvbe (hpvwokqqmw )
Positive
01 Sep 2018
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