Orfadin

Treats hereditary tyrosinemia type 1 (HT-1). NDA 21232, held by Swedish Orphan. Nitisinone.

As of Nov 14, 2026, NDA 21232 (Orfadin), held by Swedish Orphan, was first approved by FDA on Jan 18, 2002, with 12 approved supplements and 0 published complete response letters.

First approved: Jan 18, 2002
Review: Priority
Supplements approved: 12
Complete response letters: 0

What It's For

From the FDA-approved label (Indications and Usage)

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.

Full prescribing information on DailyMed ↗

Approvals and New Uses

The original application and every efficacy supplement, newest first; all 13 submissions below


Sep 1, 2017	Supplement 20Efficacy (new indication or use) · Standard review	Approved	Letter Label
Jan 18, 2002	Original applicationNew molecular entity · Priority review	Approved	Label Review Letter

Products

4 strengths and forms


Orfadin2MG · capsule;oral	Nitisinone	RxReference drug
Orfadin5MG · capsule;oral	Nitisinone	RxReference drug
Orfadin10MG · capsule;oral	Nitisinone	RxReference drug
Orfadin20MG · capsule;oral	Nitisinone	RxReference drug

All 13 submissions, including labeling and manufacturing supplements


May 17, 2019	Supplement 23Labeling · Standard review	Approved	Letter Label
Sep 1, 2017	Supplement 20Efficacy (new indication or use) · Standard review	Approved	Letter Label
Feb 28, 2017	Supplement 19Manufacturing	Approved	Label
Jun 13, 2016	Supplement 17Manufacturing · Priority review	Approved	Label
Jun 5, 2015	Supplement 16Manufacturing · Priority review	Approved	None posted
Oct 30, 2014	Supplement 14Manufacturing · Priority review	Approved	None posted
Oct 27, 2014	Supplement 15Manufacturing · Priority review	Approved	None posted
May 21, 2014	Supplement 13Labeling · Standard review	Approved	Label Letter
Jun 21, 2013	Supplement 12Manufacturing · Priority review	Approved	None posted
Apr 2, 2013	Supplement 11Manufacturing · Priority review	Approved	None posted
Feb 13, 2013	Supplement 10Labeling · Standard review	Approved	Letter Label
Jan 25, 2010	Supplement 8Labeling · Standard review	Approved	Letter Label
Jan 18, 2002	Original applicationNew molecular entity · Priority review	Approved	Label Review Letter

Identifiers

Application: NDA 21232
Drugs@FDA: Overview at FDA
Company: Swedish Orphan

Approvals, supplements, products and documents come from FDA's Drugs@FDA data files, refreshed daily; Drugs@FDA lists approved applications only. Complete response letters are FDA's published letters. Drugs@FDA and FDA complete response letters