Cystadane

(Betaine)

Phase 2Small molecule

RRECORDATI GROUP

Cystadane is one of 82 small molecule programs targeting Homocystinuria. It is currently in Phase 2.

Indications

Total Trials

Active Trials

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Pipeline

Status by Indication[3][1]

Indication

HomocystinuriaActive, not recruiting

HepatitisActive, not recruiting

Chronic HepatitisActive, not recruiting

SyringomyeliaActive, not recruiting

Primary HyperoxaluriaActive, not recruiting

Angelman SyndromeActive, not recruiting

HomocystinuriaPhase 2

Active, not recruiting

HepatitisPhase 2

Active, not recruiting

Chronic HepatitisPhase 1

Active, not recruiting

SyringomyeliaPhase 2

Active, not recruiting

Primary HyperoxaluriaPhase 2

Active, not recruiting

Angelman SyndromePreclinical

Active, not recruiting

Competition

Competitive Landscape

624 competitors in Chronic Hepatitis

#DrugCompanyPhaseTrials

Cystadane

RRECORDATI GROUP

Phase 2

2 2

PEG-Intron

Merck & Co.

Preclinical

43 3

Sovaldi

Gilead

Preclinical

34 4

Virazole

CConrado Roberto Hoffmann Filho

Preclinical

199 5

Incivek

Vertex Pharmaceuticals

Preclinical

10 6

Intron a

Sanofi

Preclinical

7 7

Roferon-a

Roche

Preclinical

3 8

Medrol

Pfizer

Preclinical

1 9

Dasabuvir

AbbVie

Preclinical

7 10

SOF/VEL

Gilead

Preclinical

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

Timeline

Expected Milestones

Q1 2028

Phase 2Recruiting

Syringomyelia

NCT06308367

Q4 2035

Phase 2Not yet recruiting

Hepatitis

NCT07276204

Clinical

Trials by Indication[1]

Chronic Hepatitis2 trials

Chronic Hepatitis C Non-Responder Study With AdoMet and Betaine

Completed·UUniversity Hospital, Basel, Switzerland·Phase 2/3·NCT00310336·30 enrolled

Pilot Study of Betaine + Combination Antiviral Therapy for Chronic Hepatitis C Genotype 1 Non-responder/Relapsers

Terminated·UUniversity of Nebraska·Phase 1·NCT00882193·2 enrolled

Primary Hyperoxaluria1 trial

Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria

Completed·MMayo Clinic·Phase 2·NCT00283387·15 enrolled

AIN1 trial

Metabolic Effects of Betaine Supplementation

Completed·JJoslin Diabetes Center·Phase 2·NCT01950039·28 enrolled

Syringomyelia1 trial

The Therapeutic Effect of Betaine in Syringomyelia

Recruiting·XXuanwu Hospital, Beijing·Phase 2·NCT06308367·30 enrolled

Peroxisome Biogenesis Disorders1 trial

Betaine and Peroxisome Biogenesis Disorders

Completed·MMcGill University Health Centre/Research Institute of the McGill University Health Centre·Phase 3·NCT01838941·12 enrolled

4 more indications

Angelman syndrome1 trial

Dietary Supplements for the Treatment of Angelman Syndrome

Completed·UUniversity of California, San Diego·N/A·NCT00348933·90 enrolled

Adult1 trial

A Preliminary Study of Choline and Betaine Supplementation Among Adults Exposed to Arsenic in Bangladesh

Completed·—·Phase 4·NCT01749982·60 enrolled

Homocystinuria1 trial

Betaine METABOLISM OF PATIENTS With Homocystinuria

Completed·AAssistance Publique - Hôpitaux de Paris·Phase 2·NCT02404337·12 enrolled

Hepatitis1 trial

Betaine vs. Placebo for Serologically Diagnosed Metabolic Dysfunction-associated Steatohepatitis (MASH)

Not yet recruiting·SSouthern California Institute for Research and Education·Phase 2·NCT07276204·70 enrolled

Regulatory

FDA Approval Timeline[4]

Oct 25, 1996

CYSTADANE

NDA NDA020576

Sources

References

[1]
ClinicalTrials.gov — 10 trial records for Betaine. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Betaine compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Betaine. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA020576). U.S. Food & Drug Administration.
[5]
SEC EDGAR — RECORDATI GROUP company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 2[1]
Company: RRECORDATI GROUP

Cystadane

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q1 2028

Q4 2035

Trials by Indication[1]

Chronic Hepatitis2 trials

Primary Hyperoxaluria1 trial

AIN1 trial

Syringomyelia1 trial

Peroxisome Biogenesis Disorders1 trial

Angelman syndrome1 trial

Adult1 trial

Homocystinuria1 trial

Hepatitis1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Related News