Homocystinuria
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Sign up freeTop drugs studied for this disease
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EMA approval · 2026-03-10EMA Authorised: Amversio (betaine) — SERB SA
The European Medicines Agency has authorized Amversio as a betaine product for homocystinuria, with SERB SA as the marketing authorization h…
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CLINICAL_TRIALS trial_result · 2026-02-19Clinical Trial Update: Evaluation of the Express Plus Range [NA]
A completed clinical trial evaluated Vitaflo's Express Plus Range for multiple metabolic disorders including phenylketonuria, homocystinuria…
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CLINICAL_TRIALS research · 2026-02-12Clinical Trial Update: Proof of Concept Creatine Supplementation for Homocystinuria Study [NA]
A proof-of-concept clinical trial evaluating creatine supplementation for homocystinuria is currently active but not recruiting.
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CLINICAL_TRIALS trial_result · 2025-04-11Clinical Trial Update: A Phase 3 Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE) [Phase 3]
This Phase 3 long-term extension study, ENSEMBLE, aims to assess the long-term safety and efficacy of pegTibatinase in participants aged 5 t…
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CLINICAL_TRIALS research · 2025-04-10Clinical Trial Update: Health Related Quality of Life (HrQoL) in Classical Homocystinuria (CBS Deficiency)
This clinical trial is investigating health-related quality of life in classical homocystinuria (CBS deficiency) and is currently recruiting…
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CLINICAL_TRIALS research · 2025-04-09Clinical Trial Update: A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment [Phase 3]
Phase 3 study evaluating peg-tibatinase versus placebo for classical homocystinuria due to CBS deficiency; the trial status is active but no…
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EMA approval · 2024-12-12EMA Authorised: Cystadane (Betaine anhydrous) — Recordati Rare Diseases
EMA has authorised Cystadane (betaine anhydrous) for homocystinuria, with Recordati Rare Diseases as the marketing authorization holder.
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CLINICAL_TRIALS research · 2024-11-22Clinical Trial Update: Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE) [Phase 1, Phase 2]
Pegtibatinase is being studied in a Phase 1/Phase 2 trial for homocystinuria caused by cystathionine beta-synthase deficiency, sponsored by …
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CLINICAL_TRIALS research · 2024-08-13Clinical Trial Update: Incidence and Risk Factors of Ocular Complications Among Patients With Homocystinuria
This clinical trial update investigates the incidence and risk factors of ocular complications in patients with homocystinuria and is curren…
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CLINICAL_TRIALS trial_result · 2023-02-03Clinical Trial Update: Betaine METABOLISM OF PATIENTS With Homocystinuria [Phase 2]
Phase 2 clinical trial update on betaine metabolism in patients with homocystinuria; study status completed.
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FDA approval · 2022-01-28FDA Approved: BETAINE ANHYDROUS — Original Application (Eton Pharmaceuticals, Inc.)
The FDA approved an original generic application for betaine anhydrous, manufactured by Eton Pharmaceuticals. It is indicated for the treatm…
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FDA approval · 2021-11-23FDA Approved: BETAINE — Original Application (Cosette Pharmaceuticals, Inc.)
FDA approved Cosette Pharmaceuticals' Betaine based on an Original Application (ANDA) for its use in treating homocystinuria.
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FDA approval · 2018-11-06FDA Approved: CYSTADANE — Supplemental Application (Recordati Rare Diseases)
The FDA approved a supplemental application for CYSTADANE (betaine) submitted by Recordati Rare Diseases.
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CLINICAL_TRIALS trial_result · 2018-07-09Clinical Trial Update: Oxidative Stress Markers In Inherited Homocystinuria And The Impact Of Taurine [Phase 1, Phase 2]
Phase 1/2 trial evaluating oxidative stress markers in inherited homocystinuria and the impact of taurine; results were posted in 2018.
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CLINICAL_TRIALS trial_result · 2017-10-16Clinical Trial Update: Vitamin D3 Treatment and Homocysteine Concentrations Among Overweight Reproductive Women [Phase 2]
Phase 2 trial evaluating Vitamin D3 (cholecalciferol) treatment and its effect on homocysteine levels in overweight reproductive-age women; …
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FDA approval · 2010-04-05FDA Approved: CYSTADANE — Supplemental Application (Recordati Rare Diseases)
FDA approved a supplemental application for Cystadane (betaine) from Recordati Rare Diseases.
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CLINICAL_TRIALS trial_result · 2009-02-18Clinical Trial Update: Homocystinuria: Treatment With N-Acetylcysteine [Phase 2]
A Phase 2 clinical trial evaluating N-Acetylcysteine for treatment of homocystinuria has completed.