Hereditary Tyrosinemia Type
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CLINICAL_TRIALS withdrawal · 2025-12-23Clinical Trial Update: A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients
A non-interventional post-marketing study assessing outcomes of nitisinone treatment in patients with hereditary tyrosinemia type I, which h…
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FDA approval · 2025-07-21FDA Approved: NITISINONE — Supplemental Application (Endo USA, Inc.)
The FDA approved a supplemental application for Nitisinone from Endo USA, Inc., updating regulatory approval for this drug.
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EMA approval · 2025-07-04EMA Authorised: Orfadin (nitisinone) — Swedish Orphan Biovitrum International AB
The European Medicines Agency authorised Orfadin (nitisinone) by Swedish Orphan Biovitrum International AB for hereditary tyrosinemia type I…
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FDA approval · 2025-06-10FDA Approved: NITYR — Supplemental Application (Cycle Pharmaceuticals Ltd.)
The FDA approved a supplemental application for NITYR (nitisinone) by Cycle Pharmaceuticals Ltd. This action updates the approved product an…
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CLINICAL_TRIALS trial_result · 2024-09-19Clinical Trial Update: Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care
A long-term safety study of Orfadin (nitisinone) in patients with hereditary tyrosinemia type I in standard clinical care has completed.
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FDA approval · 2024-01-18FDA Approved: NITYR — Supplemental Application (Cycle Pharmaceuticals Ltd.)
FDA approved a supplemental application for NITYR (nitisinone) filed by Cycle Pharmaceuticals Ltd.
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FDA approval · 2022-05-26FDA Approved: NITISINONE — Original Application (Analog Pharma)
The FDA approved Analog Pharma's Original Application for nitisinone, authorizing a generic version of the drug.
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FDA approval · 2020-09-02FDA Approved: NITYR — Supplemental Application (Cycle Pharmaceuticals Ltd.)
FDA approved a supplemental application for NITYR (nitisinone) from Cycle Pharmaceuticals Ltd. The action confirms approval of changes submi…
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FDA approval · 2019-08-26FDA Approved: NITISINONE — Original Application (Endo USA, Inc.)
FDA approved an original application for nitisinone (Endo USA, Inc.), a generic version of nitisinone for hereditary tyrosinemia type I.
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FDA approval · 2019-05-17FDA Approved: ORFADIN — Supplemental Application (Swedish Orphan Biovitrum AB (publ))
The FDA approved a supplemental application for Orfadin (nitisinone) by Swedish Orphan Biovitrum AB (publ). This action updates the drug's r…
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FDA approval · 2018-11-05FDA Approved: NITYR — Supplemental Application (Cycle Pharmaceuticals Ltd.)
FDA approved a supplemental application for NITYR (nitisinone) from Cycle Pharmaceuticals Ltd. The approval updates the product labeling.
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FDA approval · 2017-09-01FDA Approved: ORFADIN — Supplemental Application (Swedish Orphan Biovitrum AB (publ))
The FDA approved a supplemental application for Orfadin (nitisinone) submitted by Swedish Orphan Biovitrum AB (publ), updating the product l…
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FDA approval · 2017-07-26FDA Approved: NITYR — Original Application (Cycle Pharmaceuticals Ltd.)
The FDA approved NITYR (Nitisinone) as an original NDA from Cycle Pharmaceuticals Ltd. for the treatment of hereditary tyrosinemia type I.
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FDA approval · 2017-06-16FDA Approved: ORFADIN — Supplemental Application (Swedish Orphan Biovitrum AB (publ))
FDA approved a supplemental application for Orfadin (nitisinone) submitted by Swedish Orphan Biovitrum AB (publ).
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CLINICAL_TRIALS trial_result · 2016-12-13Clinical Trial Update: Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1) [Phase 1]
Phase 1 bioavailability/food-effect study of an oral nitisinone formulation for Hereditary Tyrosinemia Type I completed with results posted.
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CLINICAL_TRIALS trial_result · 2016-12-13Clinical Trial Update: Bioequivalence Study of Two Nitisinone Formulations Compared to Orfadin [Phase 1]
A Phase 1 bioequivalence trial compared two nitisinone formulations to Orfadin for hereditary tyrosinemia type I; the study is completed and…
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CLINICAL_TRIALS trial_result · 2016-10-17Clinical Trial Update: Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1) [Phase 1]
A Phase 1 study comparing two oral formulations of nitisinone for hereditary tyrosinemia type I completed, with bioequivalence results poste…
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FDA approval · 2016-06-13FDA Approved: ORFADIN — Supplemental Application (SWEDISH ORPHAN BIOVITRUM AB (PUBL))
The FDA approved a supplemental application for ORFADIN (nitisinone) submitted by Swedish Orphan Biovitrum AB (publ).
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CLINICAL_TRIALS trial_result · 2015-11-11Clinical Trial Update: Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1 [Phase 3]
A Phase 3 trial comparing once-daily versus twice-daily dosing of nitisinone in Hereditary Tyrosinemia Type I has completed.
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FDA approval · 2015-06-05FDA Approved: ORFADIN — Supplemental Application (SWEDISH ORPHAN BIOVITRUM AB (PUBL))
FDA approved a supplemental application for Orfadin (nitisinone) submitted by Swedish Orphan Biovitrum AB (publ).
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FDA approval · 2014-10-30FDA Approved: ORFADIN — Supplemental Application (SWEDISH ORPHAN BIOVITRUM AB (PUBL))
The FDA approved a supplemental application for Orfadin (nitisinone) submitted by Swedish Orphan Biovitrum AB (publ).
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CLINICAL_TRIALS trial_result · 2014-03-04Clinical Trial Update: Taste and Palatability of Orfadin Suspension [Phase 1]
This Phase 1 study evaluated the taste and palatability of Orfadin suspension in patients with hereditary tyrosinemia type I, with results p…
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CLINICAL_TRIALS trial_result · 2014-02-20Clinical Trial Update: Bioequivalence of Orfadin Suspension Compared to Orfadin Capsules, and the Effect of Food on the Bioavailability of the Suspension [Phase 1]
A Phase 1 study in healthy volunteers evaluated the bioequivalence of Orfadin suspension to Orfadin capsules and the effect of food on suspe…
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FDA approval · 2013-04-02FDA Approved: ORFADIN — Supplemental Application (SWEDISH ORPHAN BIOVITRUM AB (PUBL))
FDA approved a supplemental application for ORFADIN (nitisinone) from Swedish Orphan Biovitrum AB (publ).
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FDA approval · 2013-02-13FDA Approved: ORFADIN — Supplemental Application (SWEDISH ORPHAN BIOVITRUM AB (PUBL))
FDA approves Orfadin's supplemental application for nitisinone filed by Swedish Orphan Biovitrum AB (publ).