Nitisinone

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients	2024/01/26	NCT06227429	N/A	Recruiting	Swedish Orphan Biovitrum
Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine	2019/10/03	NCT04113772	Not Applicable	UNKNOWN	Sutphin Drugs
A Study to Investigate the Potential Influence of Nitisinone on the Metabolism and the Transport of Other Drugs in Healthy Volunteers	2017/04/06	NCT03103568	Phase 1	Completed	Swedish Orphan Biovitrum
Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1)	2016/04/25	NCT02750332	Phase 1	Completed	Cycle Pharmaceuticals Ltd.
Bioequivalence Study of Two Nitisinone Formulations Compared to Orfadin	2016/04/25	NCT02750709	Phase 1	Completed	Cycle Pharmaceuticals Ltd.
Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1)	2016/04/25	NCT02750345	Phase 1	Completed	Cycle Pharmaceuticals Ltd.
Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1	2014/12/23	NCT02323529	Phase 3	Completed	Swedish Orphan Biovitrum
Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care	2014/12/19	NCT02320084	N/A	Completed	Swedish Orphan Biovitrum
Suitability of Nitisinone in Alkaptonuria 2	2013/08/05	NCT01916382	Phase 3	UNKNOWN	University of Liverpool
Bioequivalence of Orfadin 20 mg Compared to Orfadin 10 mg Capsules.	2013/05/20	NCT01857362	Phase 1	Completed	Swedish Orphan Biovitrum

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
Nitisinone	Torrent pharmaceuticals limited	13668-631	ORAL	10 mg in 1 1	1/10/2023
Nitisinone	Eton Pharmaceuticals, Inc.	71863-119	ORAL	2 mg in 1 1	12/27/2023
Nitisinone	Par Pharmaceutical, Inc.	0254-3020	ORAL	2 mg in 1 1	11/12/2019
NITYR	Cycle Pharmaceuticals Ltd.	70709-000	ORAL	10 mg in 1 1	11/14/2018
Nitisinone	Eton Pharmaceuticals, Inc.	71863-121	ORAL	10 mg in 1 1	12/27/2023
Nitisinone	Bryant Ranch Prepack	63629-2234	ORAL	2 mg in 1 1	11/12/2019
ORFADIN	Swedish Orphan Biovitrum AB (publ)	66658-204	ORAL	4 mg in 1 mL	6/20/2022
Nitisinone	Torrent pharmaceuticals limited	13668-629	oral	2 mg in 1 1	1/10/2023
Nitisinone	Eton Pharmaceuticals, Inc.	71863-120	ORAL	5 mg in 1 1	12/27/2023
Nitisinone	Torrent pharmaceuticals limited	13668-630	oral	5 mg in 1 1	1/10/2023

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
Nityr	Cycle Pharmaceuticals (Europe) Ltd,70 Sir John Rogerson's Quay,Dublin 2,Ireland	Authorised	7/26/2018
Orfadin	Swedish Orphan Biovitrum International AB,SE-112 76 Stockholm,Sweden	Authorised	2/21/2005

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
ORFADIN nitisinone 5 mg capsule bottle	164173	A Menarini Australia Pty Ltd	Medicine	A	11/22/2010
NITYR nitisinone 5 mg tablet bottle	288128	Orpharma Pty Ltd	Medicine	A	6/8/2018
NITYR nitisinone 10 mg tablet bottle	288126	Orpharma Pty Ltd	Medicine	A	6/8/2018
ORFADIN nitisinone 10 mg capsule bottle	164174	A Menarini Australia Pty Ltd	Medicine	A	11/22/2010
ORFADIN nitisinone 2 mg capsule bottle	164163	A Menarini Australia Pty Ltd	Medicine	A	11/22/2010
NITYR nitisinone 2 mg tablet bottle	288127	Orpharma Pty Ltd	Medicine	A	6/8/2018
ORFADIN nitisinone 20 mg capsule bottle	297735	A Menarini Australia Pty Ltd	Medicine	A	2/26/2019
ORFADIN nitisinone 4 mg/mL oral suspension bottle	297736	A Menarini Australia Pty Ltd	Medicine	A	2/26/2019

Health Canada Drug Approvals

Approved Product	Company	DIN	Dosage Form	Strength	Market Date
MDK-NITISINONE	Mendelikabs Inc.	02457717	Capsule - Oral	2 MG	10/20/2016
NITISINONE TABLETS	Cycle Pharmaceuticals Ltd.	02458624	Tablet - Oral	5 MG	1/13/2017
MDK-NITISINONE	Mendelikabs Inc.	02470055	Capsule - Oral	20 MG	2/1/2019
MDK-NITISINONE	Mendelikabs Inc.	02457725	Capsule - Oral	5 MG	10/20/2016
ORFADIN	Swedish Orphan Biovitrum AB (Publ)	02459698	Capsule - Oral	2 MG	2/17/2017
ORFADIN	Swedish Orphan Biovitrum AB (Publ)	02469693	Suspension - Oral	4 MG / ML	7/10/2018
MDK-NITISINONE	Mendelikabs Inc.	02457733	Capsule - Oral	10 MG	10/20/2016
ORFADIN	Swedish Orphan Biovitrum AB (Publ)	02459728	Capsule - Oral	10 MG	2/17/2017
NITISINONE TABLETS	Cycle Pharmaceuticals Ltd.	02458632	Tablet - Oral	10 MG	12/19/2016
ORFADIN	Swedish Orphan Biovitrum AB (Publ)	02459736	Capsule - Oral	20 MG	2/17/2017

CIMA AEMPS Drug Approvals

Approved Product	Company	Registration Number	Pharmaceutical Form	Prescription Type	Status
ORFADIN 5 mg CAPSULAS DURAS	Swedish Orphan Biovitrum International Ab	04303002	CÁPSULA DURA	Uso Hospitalario	Commercialized
NITISINONA DIPHARMA 5 MG CAPSULAS DURAS EFG	Dipharma Arzneimittel Gmbh	83468	CÁPSULA DURA	Uso Hospitalario	Commercialized
ORFADIN 2 mg CAPSULAS DURAS	Swedish Orphan Biovitrum International Ab	04303001	CÁPSULA DURA	Uso Hospitalario	Commercialized
NITISINONA DIPHARMA 20 MG CAPSULAS DURAS EFG	Dipharma Arzneimittel Gmbh	85155	CÁPSULA DURA	Uso Hospitalario	Commercialized
ORFADIN 4 mg/ml SUSPENSION ORAL	Swedish Orphan Biovitrum International Ab	104303005	SUSPENSIÓN ORAL	Uso Hospitalario	Commercialized
ORFADIN 20 mg CAPSULAS DURAS	Swedish Orphan Biovitrum International Ab	104303004	CÁPSULA DURA	Uso Hospitalario	Commercialized
NITISINONA DIPHARMA 2 MG CAPSULAS DURAS EFG	Dipharma Arzneimittel Gmbh	85156	CÁPSULA DURA	Uso Hospitalario	Commercialized
NITISINONA DIPHARMA 10 MG CAPSULAS DURAS EFG	Dipharma Arzneimittel Gmbh	83467	CÁPSULA DURA	Uso Hospitalario	Commercialized
NITISINONE MDK 5 MG CAPSULAS DURAS	Mendelikabs Europe Limited	1171217002	CÁPSULA DURA	Uso Hospitalario	Commercialized
NITISINONE MDK 2 MG CAPSULAS DURAS	Mendelikabs Europe Limited	1171217001	CÁPSULA DURA	Uso Hospitalario	Commercialized

Philippines FDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Philippines FDA approvals found for this drug.

Saudi SFDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Saudi SFDA approvals found for this drug.

Malaysia NPRA Drug Approvals

Approved Product	Company	Registration Number	Dosage Form	Strength	Approval Date
No Malaysia NPRA approvals found for this drug.

UK EMC Drug Information

Medicine Name	MA Holder	MA Number	Pharmaceutical Form	Active Ingredient	Authorization Date
No UK EMC drug information found for this drug.

Related News

FDA Approves First Treatment for Alkaptonuria: Nitisinone Shows Clinical Benefits in Rare Metabolic Disorder

2 months ago

The FDA approved nitisinone (Harliku) as the first and only treatment for alkaptonuria, a rare genetic metabolic disorder affecting 1 in 250,000 to 1 million Americans.

Nitisinone Shows Promise as Novel Malaria Control Strategy by Making Human Blood Lethal to Mosquitoes

5 months ago

Researchers have discovered that nitisinone, a drug used to treat rare genetic disorders, makes human blood toxic to mosquitoes that transmit malaria, offering a potential new control strategy.

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7 months ago

The FDA has extended the PDUFA goal date for Eton Pharmaceuticals' ET-400 New Drug Application to May 28, 2025, requiring additional time to review supplemental information.

Innovative Strategies for Patient Recruitment in Rare Disease Trials: Lessons from the Alkaptonuria Experience

12 years ago

The National Alkaptonuria Centre (NAC) and DevelopAKUre clinical trials face significant recruitment challenges, needing to identify patients from a limited global pool of only 1,000 known cases.

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