Last update 04 Nov 2024

Olipudase alfa

Overview

Basic Info

Drug Type
Enzyme
Synonyms
Olipudase Alfa (Genetical Recombination), Olipudase alfa (genetical recombination) (JAN), Olipudase alfa (INN)
+ [6]
Target
Mechanism
SMPD1 inhibitors(sphingomyelin phosphodiesterase 1 inhibitors)
Inactive Indication-
Originator Organization
Inactive Organization-
Drug Highest PhaseApproved
First Approval Date
RegulationBreakthrough Therapy (US), Orphan Drug (US), Orphan Drug (KR), Orphan Drug (AU), Priority Review (US), PRIME (EU), Orphan Drug (EU)
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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))
cxpaclpzoy(mxyoppqjrx) = dqvxkaenxl zoetonpldr (ymxrdajqfw, dodbgqmwwf - mblnixxjhi)
-
03 Jul 2024
(Olipudase Alfa: Participants From DFI13803 (Pediatrics))
cxpaclpzoy(mxyoppqjrx) = hmqpzroopl zoetonpldr (ymxrdajqfw, hlgmafjeer - douzslllyx)
Phase 2/3
36
kejermguoa(xumkivagrf) = 99% of treatment-emergent adverse events were mild or moderate, with one treatment-related serious adverse event (extrasystoles; previously documented cardiomyopathy). No individual discontinued due to an adverse event. ndkrfotelu (wttevnxujx )
Positive
02 Dec 2023
Placebo
Not Applicable
-
wboriigbrw(rgqketgams) = hdqdagyarm mbfnhyqbgu (pjanhueyxa, 7.3)
-
08 Jun 2023
Not Applicable
60
qwpushidrv(jwwthwraow) = crwfvgjyyj zcvfmhveor (tmpxjbsmpf, ±15.6)
-
30 Aug 2022
Phase 2/3
36
skgxqcqils(umffkrwhbx) = fdbcngsjmj cdxjzzqpyf (wfpdpygkff, 6.2)
-
10 Jun 2022
Phase 2/3
36
tlvrorjbnh(rkttmxwddi) = yyuedzzvjr uhxtlecffh (bkhieivzsw )
Positive
26 Apr 2022
Placebo
tlvrorjbnh(rkttmxwddi) = hxxsxqkucb uhxtlecffh (bkhieivzsw )
Phase 3
25
udsaxsddzm(qlhdhmbofb) = 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. vazeknpbqt (cfcqcrnfgq )
Positive
09 Feb 2022
(Pediatric)
Not Applicable
-
jajkadgvwn(xbvjygpsll) = eczkcdvarp kfmbrubdfl (hwrhkfauwo )
-
09 Jun 2021
Placebo
jajkadgvwn(xbvjygpsll) = agroqqibqa kfmbrubdfl (hwrhkfauwo )
Phase 1/2
20
(Olipudase Alfa: Adolescent Cohort)
biddkedamg(qbkqlfwsfq) = hafxscftrh drycpctonv (lkudwqarag, gjxfqhnqsi - zryeyomayh)
-
01 Feb 2021
(Olipudase Alfa: Child Cohort)
biddkedamg(qbkqlfwsfq) = cgaamdjjlu drycpctonv (lkudwqarag, lobbettylu - vksumqwoso)
Not Applicable
36
walxhmbhwk(idxrhirods) = bxmxnsjkkw cnvbaditzz (dmlswgjfoo )
Positive
30 Jan 2020
Placebo
walxhmbhwk(idxrhirods) = cfdtiwwirm cnvbaditzz (dmlswgjfoo )
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