Hereditary Tyrosinemia, Type I
6
Pipeline Programs
1
Companies
4
Clinical Trials
4
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
0
0
0
1
4
Early DiscoveryClinical DevelopmentMarket
On Market (4)
Approved therapies currently available
U
HARLIKUApproved
nitisinone
Unknown Company4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor [EPC]oral2017
U
NITISINONEApproved
nitisinone
Unknown Company4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor [EPC]oral2023
U
NITYRApproved
nitisinone
Unknown Company4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor [EPC]oral2017
ORFADINApproved
nitisinone
Swedish Orphan Biovitrum4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor [EPC]oral2002
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Swedish Orphan BiovitrumMA - Waltham
4 programs1
1
1
NitisinonePhase 31 trial
NitisinonePhase 11 trial
NitisinoneN/A1 trial
NitisinoneN/A1 trial
Active Trials
+1 more trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
Swedish Orphan BiovitrumNitisinone
Swedish Orphan BiovitrumNitisinone
Swedish Orphan BiovitrumNitisinone
Swedish Orphan BiovitrumNitisinone
Clinical Trials (4)
Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1
Start: Dec 2014Est. completion: Sep 2015
Phase 3Completed
Taste and Palatability of Orfadin Suspension
Start: Oct 2012Est. completion: Mar 2013
Phase 1Completed
A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients
Start: Sep 2025Est. completion: Jun 2027
N/AWithdrawn
Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care
Start: Sep 2013Est. completion: Sep 2019
N/ACompleted
Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
1 late-stage (Phase 3) programs — potential near-term approvals
1 companies competing in this space