Last update 01 Nov 2024

Repinatrabit

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
(R)-3-(1-cyclopropyl-3-(2-fluoro-4-(trifluoromethoxy)benzyl)ureido)piperidine-1-carboxamide
Target
Mechanism
SLC6A19 inhibitors(solute carrier family 6 member 19 inhibitors)
Active Indication
Inactive Indication-
Originator Organization
Active Organization
Inactive Organization-
Drug Highest PhasePhase 3
First Approval Date-
RegulationRare Pediatric Disease (US), Orphan Drug (EU)
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Structure

Molecular FormulaC18H22F4N4O3
InChIKeyFNRHWODWSBDOOY-CYBMUJFWSA-N
CAS Registry2837993-05-0

R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
PhenylketonuriasPhase 3-01 Nov 2024
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 1/2
18
JNT-517 150mg BID
hpykprembt(scswolocua) = The 150mg BID dose of JNT-517 led to a statistically significant (p=<0.0001 vs. placebo) mean blood Phe reduction from baseline of 60% on average across days 14, 21, and 28 and a 58% reduction from baseline to day 28. vrwsihbroh (efhhkphkxu )
Positive
04 Sep 2024
Placebo
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Approval

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Regulation

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