Procysbi

(Cysteamine Bitartrate)

PreclinicalSmall molecule

HHorizon Therapeutics Ireland DAC

Procysbi is one of 56 small molecule programs targeting Cystinosis. It is currently in Preclinical.

Indications

Total Trials

Active Trials

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

CystinosisActive, not recruiting

Major Depressive DisorderActive, not recruiting

AbsorptionCompleted

CystinosisPreclinical

Active, not recruiting

Major Depressive DisorderPreclinical

Active, not recruiting

AbsorptionPreclinical

Completed

Competition

Competitive Landscape

115 competitors in Major Depressive Disorder

#DrugCompanyPhaseTrials

Zoloft

VViatris Specialty LLC

Approved

15 2

Effexor

WWyeth-Lederle Vaccines

Approved

8 3

Ulotaront

IIntra-Cellular Therapies, Inc.

Approved

6 4

Valcyte

EEngelhard Arzneimittel GmbH & Co.KG

Approved

—5

Seroquel

EEngelhard Arzneimittel GmbH & Co.KG

Phase 3

12 6

Ketalar

Boehringer Ingelheim

Phase 3

5 7

Lithobid

SSolvay Pharmaceuticals

Phase 3

1 8

Clozaril

LLife Extension Foundation Inc.

Phase 3

1 9

Psilocybin

Ssris

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

Clinical

Trials by Indication[1]

Cystinosis3 trials

Observational Study to Assess the Quality of Life in Nephropathic Cystinosis Patients

Enrolling by invitation·CChiesi SA/NV··NCT04246060·31 enrolled

Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis

Completed· Amgen logo

Amgen·Phase 3·NCT01000961·43 enrolled

A Phase 1/2 Pharmacokinetic and Pharmacodynamic Study of NPI-001 Oral Solution Compared to Cysteamine Bitartrate in Cystinosis Patients

Completed·NNacuity Pharmaceuticals, Inc.·Preclinical·NCT05994534·8 enrolled

Mitochondrial Disease2 trials

Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease

Completed· Amgen logo

Amgen·Phase 2·NCT02023866·36 enrolled

A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease

Terminated· Amgen logo

Amgen·Phase 2·NCT02473445·22 enrolled

Major Depressive Disorder1 trial

Cysteamine Therapy for Major Depressive Disorder

Terminated·IIcahn School of Medicine at Mount Sinai·N/A·NCT00715559·3 enrolled

ART1 trial

Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)

Completed· Amgen logo

Amgen·Phase 3·NCT01197378·60 enrolled

Absorption1 trial

Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™

Completed·UUniversity of California, San Diego·N/A·NCT01432561·8 enrolled

1 more indication

Asthma1 trial

Cysteamine for Asthma

Completed·—·Phase 1·NCT03883984·60 enrolled

Regulatory

FDA Approval Timeline[4]

Aug 15, 1994

CYSTAGON

NDA NDA020392

Apr 30, 2013

PROCYSBI

NDA NDA203389

Feb 14, 2020

PROCYSBI

NDA NDA213491

Sources

References

[1]
ClinicalTrials.gov — 9 trial records for Cysteamine Bitartrate. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Cysteamine Bitartrate compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Cysteamine Bitartrate. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA213491). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Procysbi. U.S. Food & Drug Administration.
[6]
SEC EDGAR — Horizon Therapeutics Ireland DAC company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Preclinical[1]
Company: HHorizon Therapeutics Ireland DAC

Patent Landscape[5]

Patent Cliff

Jan 26, 2027

Earliest patent expiration — generic entry possible after this date

Patent #Expires

9192590Jan 26, 2027

9198882Jan 26, 2027

9925156Jan 26, 2027

9925158Jan 26, 2027

9925157Jan 26, 2027

19 more patents

9198882*PEDJul 26, 2027

9925158*PEDJul 26, 2027

9925157*PEDJul 26, 2027

9925156*PEDJul 26, 2027

9192590*PEDJul 26, 2027

8026284Sep 22, 2027

8026284*PEDMar 22, 2028

9173851Jun 17, 2034

9233077Jun 17, 2034

9233077*PEDDec 17, 2034

9173851*PEDDec 17, 2034

10143665Aug 16, 2036

10905662Aug 16, 2036

10548859Aug 16, 2036

10328037Aug 16, 2036

10143665*PEDFeb 16, 2037

10548859*PEDFeb 16, 2037

10328037*PEDFeb 16, 2037

10905662*PEDFeb 16, 2037

Procysbi

Status by Indication[3][1]

Competitive Landscape

Trials by Indication[1]

Cystinosis3 trials

Mitochondrial Disease2 trials

Major Depressive Disorder1 trial

ART1 trial

Absorption1 trial

Asthma1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

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