Is Nitisinone registered with the SFDA?

The SFDA human drug list (edition 4-2026, as published by the Council of Health Insurance) lists 8 valid registrations of Nitisinone, including NITYR 10 mg Tablet (10 mg, Tablet, SAR 9067.85); NITYR 2 mg Tablet (2 mg, Tablet, SAR 2413.55); NITYR 5 mg Tablet (5 mg, Tablet, SAR 5012.55); ORFADIN 10 MG CAPSULE (10 mg, Capsule, hard, SAR 11160.45); ORFADIN 2 MG CAPSULE (2 mg, Capsule, hard, SAR 2970.50).

Is Nitisinone approved by the US FDA?

Drugs@FDA lists 8 application product lines for Nitisinone, including ORFADIN (Prescription); ORFADIN (Prescription); NITYR (Prescription); HARLIKU (Prescription); NITISINONE (Prescription).

Is Nitisinone authorised in the European Union?

EMA lists 2 centrally authorised medicines containing Nitisinone: Orfadin, Nityr.

Are there safety signals or safety updates for Nitisinone?

US FDA: 4 entries (latest 2025-06-10).

Active substance

Nitisinone

SFDA 8
US FDA 8
EMA 3
Health Canada 8
Safety 4

Open in the medicines table

SFDA Saudi Arabia — SFDA human drug list

Trade name	Strength	Form · route	Pack	Public price	Type	Company	Status
NITYR 10 mg Tablet	10 mg	Tablet · Oral use	60 Bottle	SAR 9067.85	NCE · Prescription	Cycle Pharmaceuticals LTD	Valid
NITYR 2 mg Tablet	2 mg	Tablet · Oral use	60 Bottle	SAR 2413.55	NCE · Prescription	Cycle Pharmaceuticals LTD	Valid
NITYR 5 mg Tablet	5 mg	Tablet · Oral use	60 Bottle	SAR 5012.55	NCE · Prescription	Cycle Pharmaceuticals LTD	Valid
ORFADIN 10 MG CAPSULE	10 mg	Capsule, hard · Oral use	60 Bottle	SAR 11160.45	NCE · Prescription	SWEDISH ORPHAN BIOVITRUM INTERNATIONAL AB	Valid
ORFADIN 2 MG CAPSULE	2 mg	Capsule, hard · Oral use	60 Bottle	SAR 2970.50	NCE · Prescription	SWEDISH ORPHAN BIOVITRUM INTERNATIONAL AB	Valid
ORFADIN 20 MG CAPSULE	20 mg	Capsule, hard · Oral use	60 Bottle	SAR 23260.70	NCE · Prescription	SWEDISH ORPHAN BIOVITRUM INTERNATIONAL AB	Valid
ORFADIN 5 MG CAPSULE	5 mg	Capsule, hard · Oral use	60 Bottle	SAR 6169.30	NCE · Prescription	SWEDISH ORPHAN BIOVITRUM INTERNATIONAL AB	Valid
Orfadin	4 mg/ml	Oral suspension · Oral use	1 Bottle	SAR 9220.80	NCE · Prescription	SWEDISH ORPHAN BIOVITRUM INTERNATIONAL AB	Valid

Indications — CHI Daman Drug Formulary

HEREDITARY TYROSINEMIA TYPE 1

US FDA United States — FDA approvals (Drugs@FDA)

Product	Strength	Form · route	Applicant	Status	First approved
ORFADIN	2MG; 5MG; 10MG; 20MG	capsule · oral	SWEDISH ORPHAN	Prescription	18 Jan 2002
ORFADIN	4MG/ML	suspension · oral	SWEDISH ORPHAN	Prescription	22 Apr 2016
NITYR	2MG; 5MG; 10MG	tablet · oral	CYCLE	Prescription	26 Jul 2017
HARLIKU	2MG	tablet · oral	CYCLE	Prescription	26 Jul 2017
NITISINONE	2MG; 5MG; 10MG	capsule · oral	NOVITIUM PHARMA	Prescription	26 Aug 2019
NITISINONE	2MG; 5MG; 10MG; 20MG	capsule · oral	MEDUNIK	Prescription	26 May 2022
NITISINONE	2MG; 5MG; 10MG; 20MG	capsule · oral	ETON	Prescription	25 May 2023
NITISINONE	2MG; 5MG; 10MG; 20MG	capsule · oral	TORRENT	Discontinued	9 Jan 2023

EMA European Union — centrally authorised medicines

Orfadin Swedish Orphan Biovitrum International AB · Authorised · authorised 21 Feb 2005 · ATC A16AX04

Therapeutic indication (EMA)

Hereditary tyrosinemia type 1 (HT 1)Orfadin is indicated for the treatment of adult and paediatric (in any age range) patients with confirmed diagnosis of hereditary tyrosinemia type 1 (HT 1) in combination with dietary restriction of tyrosine and phenylalanine. Alkaptonuria (AKU)Orfadin is indicated for the treatment of adult patients with alkaptonuria (AKU).

Nityr Cycle Pharmaceuticals (Europe) Ltd · Authorised · authorised 26 Jul 2018 · ATC A16AX04

Therapeutic indication (EMA)

Treatment of adult and paediatric patients with confirmed diagnosis of hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.

Nitisinone MDK (previously Nitisinone MendeliKABS) MendeliKABS Europe Ltd · Withdrawn · authorised 24 Aug 2017 · ATC A16AX04

Therapeutic indication (EMA)

Treatment of adult and paediatric (in any age range) patients with confirmed diagnosis of hereditary tyrosinemia type 1 (HT 1) in combination with dietary restriction of tyrosine and phenylalanine.

Canada Canada — marketed products (Health Canada)

Brand	DIN	Strength	Form · route	Company
MDK-NITISINONE	02457717	2 MG	CAPSULE · ORAL	MENDELIKABS INC
MDK-NITISINONE	02457725	5 MG	CAPSULE · ORAL	MENDELIKABS INC
MDK-NITISINONE	02457733	10 MG	CAPSULE · ORAL	MENDELIKABS INC
ORFADIN	02459698	2 MG	CAPSULE · ORAL	SWEDISH ORPHAN BIOVITRUM AB (PUBL)
ORFADIN	02459701	5 MG	CAPSULE · ORAL	SWEDISH ORPHAN BIOVITRUM AB (PUBL)
ORFADIN	02459728	10 MG	CAPSULE · ORAL	SWEDISH ORPHAN BIOVITRUM AB (PUBL)
ORFADIN	02459736	20 MG	CAPSULE · ORAL	SWEDISH ORPHAN BIOVITRUM AB (PUBL)
ORFADIN	02469693	4 MG / ML	SUSPENSION · ORAL	SWEDISH ORPHAN BIOVITRUM AB (PUBL)

Safety signals and safety-related labeling changes

US FDA 10 Jun 2025 HARLIKU — safety-related labeling change
Labeling sections changed: Warnings and Precautions; Adverse Reactions; Drug Interactions; Use in Specific Populations; Patient Counseling Information / Patient Information / Medication Guide
US FDA 18 Jan 2024 NITYR — safety-related labeling change
Labeling sections changed: Warnings and Precautions; Adverse Reactions; Drug Interactions; Use in Specific Populations; Patient Counseling Information / Patient Information / Medication Guide
US FDA 17 May 2019 ORFADIN — safety-related labeling change
Labeling sections changed: Warnings and Precautions; Adverse Reactions; Drug Interactions; Use in Specific Populations; Patient Counseling Information / Patient Information / Medication Guide
US FDA 17 May 2019 ORFADIN — safety-related labeling change
Labeling sections changed: Warnings and Precautions; Adverse Reactions; Drug Interactions; Use in Specific Populations

All safety entries for nitisinone →

Values as published by each source: SFDA human drug list (4-2026, as published by the Council of Health Insurance; indications from the CHI Daman Drug Formulary), Drugs@FDA, EMA medicines data, Health Canada Drug Product Database (Open Government Licence – Canada), CMS NADAC, NHS Drug Tariff Part VIIIA (NHSBSA Copyright, Open Government Licence v3.0), WHO Model List of Essential Medicines (CC BY 3.0 IGO; regrouped by active substance, not created by WHO), EMA PRAC, US FDA safety-related labeling changes and MHRA Drug Safety Update (Open Government Licence v3.0). Sources and reuse. Not a substitute for the official registers or the product information.