Drugs / Nitisinone
Nitisinone
also known as
NTBC · Nitisinona · Nitisinone mdk (previously nitisinone mendelikabs) · Nityr · Orfadin · SC-0735
Small molecule targets CYP2C9, CYP2D6, CYP2E1, HPD via inhibition
Regulatory milestones approvals, filings & regulatory actions · 5 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Label expansions (2) | US (FDA) | HARLIKU | — | 2020-09-02 – 2025-06-10 | fda.gov ↗ |
| Label expansion | US (FDA) | ORFADIN | — | 2017-09-01 | fda.gov ↗ |
| Approved | EU (EMA) | Orfadin | — | 2005-02-21 | europa.eu ↗ |
| Approved | US (FDA) | ORFADIN | — | 2002-01-18 | fda.gov ↗ |
Trials 12
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 13 trials | ||||||
| Phase 1 | NCT03103568 | Mar → Jun 2017 | — | Swedish Orphan Biovitrum | Completed | No outcome recorded |
| Phase 1 | NCT02750332 | Nov → Dec 2015 | tyrosinemia type I | Cycle Pharmaceuticals Ltd. | Completed | No outcome recorded |
| Phase 1 | NCT01734889 | Oct 2012 → Mar 2013 | tyrosinemia type I | Swedish Orphan Biovitrum | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT01838655 | Apr 2013 → Jul 2016 | oculocutaneous albinism, vision disorder | National Eye Institute (NEI) | Completed | No outcome recorded |
| Phase 22 trials · 1 met primary | ||||||
| Phase 2 | NCT01828463 | May → Oct 2013 | alkaptonuria | University of Liverpool | Completed | Met primary |
| Phase 2 | NCT00107783 | Jan 2005 → Apr 2009 | alkaptonuria | National Human Genome Research Institute (NHGRI) | Completed | No outcome recorded |
| Phase 2/31 trial | ||||||
| Phase 2/3 | NCT01390077 | Jan 2011 → Jun 2016 | alkaptonuria | University of California, San Diego | Completed | No outcome recorded |
| Phase 32 trials · 1 met primary | ||||||
| Phase 3 | NCT02323529 | Dec 2014 → Sep 2015 | tyrosinemia type I | Swedish Orphan Biovitrum | Completed | No outcome recorded |
| Phase 3 | NCT01916382 | Apr 2014 → Feb 2016 | alkaptonuria | University of Liverpool | Unknown | Met primary |
| Phase not applicable3 trials | ||||||
| — | NCT04113772 | Nov 2019 → Dec 2020 | tyrosinemia type I | Sutphin Drugs | Unknown | No outcome recorded |
| — | NCT00031161 | Sep 2001 → Sep 2005 | lactic acidosis | University of Florida | Completed | No outcome recorded |
| — | NCT00004443 | Oct 1998 → Sep 2006 | tyrosinemia type I | University of Washington | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 18
| Date | Issuer | Release |
|---|---|---|
| 2025-06-19 | Cycle Pharmaceuticals Ltd. | Regulatory Cycle Pharmaceuticals’ HARLIKU™ (nitisinone) Tablets Receive First FDA-Approval as Treatment for Alkaptonuria (AKU) cyclepharma.com ↗
Cycle Pharmaceuticals announced that the FDA has approved HARLIKU (nitisinone) Tablets for the reduction of urine homogentisic acid (HGA) in adult patients with AKU. |
| 2021-03-15 | Cycle Pharmaceuticals Ltd. | Regulatory NITYR (nitisinone) Tablets for Tyrosinaemia Type 1 receives Marketing Authorization in New Zealand cyclepharma.com ↗
Cycle Pharmaceuticals Ltd. and Orpharma NZ Ltd. are pleased to announce that on 19 th January 2021 they have received the Marketing Authorization for NITYR™ (nitisinone) Tablets by the New Zealand Medicines and Medical Devices Safety Authority (MEDSAFE). |
| 2020-09-18 | Sobi, Inc. | Regulatory Orfadin® (nitisinone) receives positive opinion from CHMP for treatment of AKU sobi.com ↗
Swedish Orphan Biovitrum AB (publ) (Sobi™) today announces that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for Orfadin® (nitisinone) for the treatment of adult patients with alkaptonuria (AKU), the first described human genetic disease. |
| 2020-09-14 | Cycle Pharmaceuticals Ltd. | Regulatory US FDA Grants Once Daily Dosing Option For NITYR (nitisinone) Tablets for HT-1 Patients cyclepharma.com ↗
Cycle Pharmaceuticals is pleased to announce that the U.S. Food and Drug Administration (FDA) has approved the once-daily dosing of NITYR ® . |
| 2020-05-13 | Cycle Pharmaceuticals Ltd. | Regulatory Cycle Pharmaceuticals’ NITYR® (nitisinone) Tablets Receives Marketing Authorization in the Kingdom of Saudi Arabia cyclepharma.com ↗ |
| 2018-11-22 | Cycle Pharmaceuticals Ltd. | Regulatory Cycle Pharmaceuticals’ NITYR (nitisinone) tablets receives marketing authorization in Chile cyclepharma.com ↗
Cycle Pharmaceuticals Ltd. (Cycle) and its licensee Axon Pharma S.P.A. (Axon) are pleased to announce the approval of NITYR™ (nitisinone) tablets by the Chilean Institute of Public Health (Ministry of Health, MINSAL). |
| 2018-11-19 | Cycle Pharmaceuticals Ltd. | Regulatory Longer shelf life for NITYR (nitisinone) tablets for HT-1 in the U.S. granted by the FDA cyclepharma.com ↗
Cycle Pharmaceuticals (Cycle) is pleased to announce that it has obtained the required stability data to extend the shelf life of NITYR™ (nitisinone) tablets from 24 to 36 months at room temperature. |
| 2018-11-12 | Cycle Pharmaceuticals Ltd. | Regulatory Cycle Pharmaceuticals Receives Approval for Updated Infant Administration for NITYR (nitisinone) Tablets for HT-1 in U.S. cyclepharma.com ↗ |
| 2017-11-27 | Sobi, Inc. | Regulatory Sobi receives approval from Health Canada for Orfadin® oral suspension for the treatment of HT-1 sobi.com ↗
Swedish Orphan Biovitrum AB (publ) (Sobi™) has received approval from Health Canada for an oral suspension formulation of Orfadin® (nitisinone). |
| 2017-09-05 | Sobi, Inc. | Regulatory Sobi receives approval from the FDA for once-daily dosing frequency of Orfadin® for the treatment of HT-1 sobi.com ↗
Swedish Orphan Biovitrum AB (publ) (Sobi™) has received approval from the U.S. Food and Drug Administration (FDA) for a reduced dosing frequency for Orfadin® (nitisinone) from twice daily to once daily in patients 5 years of age and older. |
| 2017-08-01 | Cycle Pharmaceuticals Ltd. | Regulatory Cycle Pharmaceuticals Receives FDA Approval of Nityr™ (nitisinone) Tablets for Treatment of HT-1 cyclepharma.com ↗
Cycle Pharmaceuticals is pleased to announce that Cycle’s nitisinone Tablets, NITYR, have been approved by the U.S. Food and Drug Administration for the treatment of Hereditary Tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. |
| 2017-05-17 | Sobi, Inc. | Regulatory Orfadin® capsules approved in the Kingdom of Saudi Arabia for the treatment of hereditary tyrosinemia type 1 sobi.com ↗ |
| 2017-03-07 | Sobi, Inc. | Regulatory FDA approves in-use storage at room temperature for Orfadin® capsules sobi.com ↗
Swedish Orphan Biovitrum AB (publ) (Sobi™) today announces that FDA has approved in-use storage at room temperature (25°C or less) for up to 45 days for all strengths of Orfadin® capsules (2 mg, 5 mg, 10 mg and 20 mg). |
| 2017-02-02 | Sobi, Inc. | Regulatory Sobi obtains approval from the European Commission for new dosing frequency of Orfadin® sobi.com ↗ |
| 2017-01-04 | Sobi, Inc. | Regulatory Health Canada approves Orfadin® capsules for the treatment of hereditary tyrosinaemia type-1 (HT-1) sobi.com ↗
Swedish Orphan Biovitrum AB (publ) (Sobi™) today announces that Health Canada has approved Orfadin® (nitisinone) capsules for the treatment of hereditary tyrosinaemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. |
| 2016-07-14 | Sobi, Inc. | Regulatory Health Canada validates Orfadin® capsule filing sobi.com ↗
Swedish Orphan Biovitrum AB (publ) (Sobi) announced today that Health Canada has initiated review of the application for approval of Orfadin® (nitisinone) capsules for the treatment of hereditary tyrosinaemia type-1 (HT-1). |
| 2016-06-15 | Sobi, Inc. | Regulatory Orfadin® 20 mg capsule approved in the US sobi.com ↗
Swedish Orphan Biovitrum AB (publ) (Sobi) announced today that the Food and Drug Administration (FDA) has approved a higher strength 20 mg capsule of Orfadin® (nitisinone) for the treatment of Hereditary Tyrosinaemia type-1 (HT-1). |
| 2016-04-26 | Sobi, Inc. | Regulatory Orfadin® Oral Suspension approved in the US sobi.com ↗
the US Food and Drug Administration (FDA) has as of 22 April 2016, approved Orfadin® (nitisinone) Oral Suspension |
All press releases naming this drug 22 releases
Evidence & citations 14 cited values
Every value below carries the sentence it was read from. 13 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Nitisinone | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03103568 ↗ |
| Known as | Nitisinona | ChEMBL registry synonym — accepted as the source's own label CHEMBL1337 ↗ |
| Known as | Nitisinone mdk (previously nitisinone mendelikabs) | ChEMBL registry synonym — accepted as the source's own label CHEMBL1337 ↗ |
| Known as | Nityr | ChEMBL registry synonym — accepted as the source's own label CHEMBL1337 ↗ |
| Known as | NTBC | “a compound named 2-(2-nitro-4-trifluoromethylbenzoyl) - 1, 3-cyclohexanedione (nitisinone, NTBC, Orfadin) inhibits 4-hydroxyphenylpyruvate dioxygenase” NCT00107783 ↗ |
| Known as | Orfadin | “Nitisinone 10 mg Tablets (Test Product are bioequivalent to the reference product Orfadin 10 mg” NCT04113772 ↗ |
| Known as | SC-0735 | ChEMBL registry synonym — accepted as the source's own label CHEMBL1337 ↗ |
| Action | Inhibit | “4-hydroxyphenylpyruvate dioxygenase inhibitor” CHEMBL1337 ↗1“inhibits 4-hydroxyphenylpyruvate dioxygenase” NCT00107783 ↗ |
| Modality | Small molecule | — CHEMBL1337 ↗ |
| Route | Oral | “Patients receive oral NTBC twice a day.” NCT00004443 ↗ |
| Target | CYP2C9 | “potential inhibition of nitisinone on CYP2C9, CYP2D6, and CYP2E1” NCT03103568 ↗ |
| Target | CYP2D6 | “potential inhibition of nitisinone on CYP2C9, CYP2D6, and CYP2E1” NCT03103568 ↗ |
| Target | CYP2E1 | “potential inhibition of nitisinone on CYP2C9, CYP2D6, and CYP2E1” NCT03103568 ↗ |
| Target | HPD | “4-hydroxyphenylpyruvate dioxygenase inhibitor” CHEMBL1337 ↗ |