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Orfadin

(Nitisinone)

PreclinicalSmall molecule

BBicycle Health

Orfadin is one of 40 small molecule programs targeting Toxicity. It is currently in Preclinical.

2

Indications

16

Total Trials

0

Active Trials

39

Competitors

3

FDA Applications

NDA/BLA/ANDA regulatory filings

2

Patents

Pipeline

Status by Indication[3][1]

Indication

ToxicityActive, not recruiting

Oculocutaneous AlbinismActive, not recruiting

ToxicityPreclinical

Active, not recruiting

Oculocutaneous AlbinismPhase 1/Phase 2

Active, not recruiting

Competition

Competitive Landscape

223 competitors in Toxicity

#DrugCompanyPhaseTrials

Erbitux

Coly-Mycin M

Intelence

Curcumin

Trizivir

Cysteine Hydrochloride

Tenofovir/emtricitabine/rilpivirine

MMahidol University

Colistin Sulfomethate

MMahidol University

Cellcept

Afinitor

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Clinical

Trials by Indication[1]

ALK3 trials

Dose Response Study of Nitisinone in Alkaptonuria

Completed·UUniversity of Liverpool·Phase 2·NCT01828463·40 enrolled

Suitability of Nitisinone in Alkaptonuria 2

Unknown status·UUniversity of Liverpool·Phase 3·NCT01916382·140 enrolled

Long-Term Study of Nitisinone to Treat Alkaptonuria

Completed·NNational Human Genome Research Institute (NHGRI)·Phase 2·NCT00107783·40 enrolled

MPA3 trials

Bioequivalence Study of Two Nitisinone Formulations Compared to Orfadin

Completed·CCycle Pharmaceuticals Ltd.·Phase 1·NCT02750709·24 enrolled

Bioequivalence of Orfadin 20 mg Compared to Orfadin 10 mg Capsules.

Completed·SSwedish Orphan Biovitrum·Phase 1·NCT01857362·12 enrolled

Bioequivalence of Orfadin Suspension Compared to Orfadin Capsules, and the Effect of Food on the Bioavailability of the Suspension

Completed·SSwedish Orphan Biovitrum·Phase 1·NCT01682538·12 enrolled

Treatment2 trials

A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients

Withdrawn·SSwedish Orphan Biovitrum··NCT06227429

Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care

Completed·SSwedish Orphan Biovitrum··NCT02320084·315 enrolled

Oral2 trials

Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1)

Completed·CCycle Pharmaceuticals Ltd.·Phase 1·NCT02750332·20 enrolled

Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1)

Completed·CCycle Pharmaceuticals Ltd.·Phase 1·NCT02750345·24 enrolled

Efficacy and Safety1 trial

Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1

Completed·SSwedish Orphan Biovitrum·Phase 3·NCT02323529·18 enrolled

4 more indications

Oculocutaneous Albinism1 trial

Nitisinone for Type 1B Oculocutaneous Albinism

Completed·NNational Eye Institute (NEI)·Phase 1/2·NCT01838655·5 enrolled

Age1 trial

Nitisinone (NTBC) In Different Age Groups Of Patients With Alkaptonuria

Completed·UUniversity of California, San Diego·Phase 2/3·NCT01390077·8 enrolled

Toxicity1 trial

Prevention of Dichloroacetate Toxicity

Completed·UUniversity of Florida·N/A·NCT00031161·30 enrolled

Unspecified2 trials

Taste and Palatability of Orfadin Suspension

Completed·SSwedish Orphan Biovitrum·Phase 1·NCT01734889·18 enrolled

Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine

Unknown status·SSutphin Drugs·N/A·NCT04113772·4 enrolled

Regulatory

FDA Approval Timeline[4]

Jan 18, 2002

ORFADIN

NDA NDA021232

Apr 22, 2016

ORFADIN

NDA NDA206356

Jun 10, 2025

HARLIKU

NDA NDA209449

Sources

References

[1]
ClinicalTrials.gov — 16 trial records for Nitisinone. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Nitisinone compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Nitisinone. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA209449). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Orfadin. U.S. Food & Drug Administration.
[6]
SEC EDGAR — Bicycle Health company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Preclinical[1]
Company: BBicycle Health

Patent Landscape[5]

Patent Cliff

Feb 28, 2033

Earliest patent expiration — generic entry possible after this date

Patent #Expires

9301932Feb 28, 2033

10328029Jan 5, 2035

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