Quick answers
- What is Nitisinone used for?
- The FDA-approved label (Orfadin, Swedish Orphan Biovitrum AB (publ), December 18, 2025) says: “ORFADIN ® is indicated for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. ORFADIN is a hydroxy-phenylpyruvate dioxygenase inhibitor indicated for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.” This page is not medical advice. Talk to your doctor or pharmacist before you start, stop or change any medicine.
- Does Nitisinone have a boxed warning?
- No. None of the 9 FDA labels we found for this medicine has a boxed warning.
- Is there a shortage of Nitisinone?
- No current shortage is listed by the FDA for the products of this medicine (FDA data from October 30, 2026).
- Has Nitisinone been recalled?
- No recall linked to the products of this medicine appears in the FDA enforcement reports we use.
- Who makes Nitisinone?
- 6 companies list prescription products of Nitisinone in the FDA NDC Directory, including Swedish Orphan Biovitrum AB (publ), Eton Pharmaceuticals, Inc. and Cycle Pharmaceuticals Ltd.
What Nitisinone is used for
Quoted from the “Indications and Usage” section of the FDA-approved label.
ORFADIN ® is indicated for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. ORFADIN is a hydroxy-phenylpyruvate dioxygenase inhibitor indicated for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.
— FDA label: Orfadin, Swedish Orphan Biovitrum AB (publ), effective December 18, 2025. Read the full label on DailyMed →
The FDA has 9 labels for products of this medicine (different makers and forms); uses can differ between them. Check the label that comes with your own medicine.
This page is not medical advice. Talk to your doctor or pharmacist before you start, stop or change any medicine.
Boxed warning
A boxed warning (“black box”) is the FDA’s strongest warning on a drug label, for serious or life-threatening risks.
The label quoted above has no boxed warning.
0 of 9 FDA labels for this medicine have a boxed warning.
Forms and how it is given
Companies that list this medicine
Labelers in the FDA NDC Directory: the company named on the package (maker or distributor), with the number of prescription products.
- Swedish Orphan Biovitrum AB (publ)5 products
- Eton Pharmaceuticals, Inc.4 products
- Cycle Pharmaceuticals Ltd4 products
- Analog Pharma4 products
- Bryant Ranch Prepack3 products
- Par Health USA, LLC3 products
FDA approval history
- NDA: New Drug Application: how a new (usually brand-name) medicine is approved.
- ANDA: Abbreviated New Drug Application: how a generic version is approved.
| Application | Sponsor | Approved | Later changes approved |
|---|---|---|---|
| NDA021232 ORFADIN | Swedish Orphan | January 18, 2002 | 12, latest May 17, 2019 2019: Labeling · 2017: Efficacy · 2017: Manufacturing (CMC) |
| NDA206356 ORFADIN | Swedish Orphan | April 22, 2016 | 4, latest May 17, 2019 2019: Labeling · 2017: Efficacy · 2017: Labeling |
| NDA209449 HARLIKU, NITYR | Cycle | July 26, 2017 | 4, latest June 10, 2025 2025: Efficacy · 2024: Labeling · 2020: Efficacy |
Generic approvals (3)
- ANDA211041Novitium Pharma · August 26, 2019
- ANDA212390Medunik · May 26, 2022
- ANDA216201Eton · May 25, 2023
Shortage status
The FDA drug shortage list does not show these products.
Recalls
No recall linked to these products (by their NDC code) appears in the FDA enforcement reports.
Official identifiers
Sources
- FDA-approved label on DailyMed (National Library of Medicine) — Orfadin, Swedish Orphan Biovitrum AB (publ), effective December 18, 2025; via openFDA, FDA data exported October 30, 2026, retrieved October 31, 2026
- NDC Directory (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
- Drugs@FDA (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
- Drug recall enforcement reports (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 27, 2026, retrieved October 31, 2026
- Drug shortages (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
openFDA data is provided by the FDA as is; it may be incomplete. Always rely on the label that comes with your medicine. FDA Enforcement Report search →