Betaine

Overview

Betaine is a methyl group donor that functions in the normal metabolic cycle of methionine. It is a naturally occurring choline derivative commonly ingested through diet, with a role in regulating cellular hydration and maintaining cell function. Homocystinuria is an inherited disorder that leads to the accumulation of homocysteine in plasma and urine. Currently, no treatments are available to correct the genetic causes of homocystinuria. However, in order to normalize homocysteine levels, patients can be treated with vitamin B6 (pyridoxine), vitamin B12 (cobalamin), folate and specific diets. Betaine reduces plasma homocysteine levels in patients with homocystinuria. Although it is present in many food products, the levels found there are insufficient to treat this condition. The FDA and EMA have approved the product Cystadane (betaine anhydrous, oral solution) for the treatment of homocystinuria, and the EMA has approved the use of Amversio (betaine anhydrous, oral powder).

Indication

Betaine is indicated for the treatment of homocystinuria in pediatric and adult patients to decrease elevated homocysteine blood levels. Included within the category of homocystinuria are deficiencies or defects in cystathionine beta-synthase (CBS), 5,10-methylenetetrahydrofolate reductase (MTHFR), and cobalamin cofactor metabolism (cbl).

Associated Conditions

Homocystinuria
Homocystinuria Due to Cystathionine Beta-Synthase Deficiency
Homocystinuria due to MTHFR deficiency
Homocystinuria due to cobalamin cofactor metabolism (cbl) defect

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
The Therapeutic Effect of Betaine in Syringomyelia	2024/03/13	NCT06308367	Phase 2	Recruiting	Xuanwu Hospital, Beijing
Comparison of Gum® HydralTM Moisturizing Gel and Biotene® Oral Gel in Dry Mouth Sensation Reduction	2022/04/22	NCT05342272	Phase 3	Completed	Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia
Betaine METABOLISM OF PATIENTS With Homocystinuria	2015/03/31	NCT02404337	Phase 2	Completed	Assistance Publique - Hôpitaux de Paris
Choline Dehydrogenase and Sperm Function: Effects of Betaine	2014/04/24	NCT02122211	Phase 1	Completed	University of North Carolina, Chapel Hill
Metabolic Effects of Betaine Supplementation	2013/09/25	NCT01950039	Phase 2	Completed	Joslin Diabetes Center
Betaine and Peroxisome Biogenesis Disorders	2013/04/24	NCT01838941	Phase 3	Completed	McGill University Health Centre/Research Institute of the McGill University Health Centre
A Preliminary Study of Choline and Betaine Supplementation Among Adults Exposed to Arsenic in Bangladesh	2012/12/17	NCT01749982	Phase 4	Completed	Columbia University
Pilot Study of Betaine + Combination Antiviral Therapy for Chronic Hepatitis C Genotype 1 Non-responder/Relapsers	2009/04/16	NCT00882193	Early Phase 1	Terminated	University of Nebraska
Betaine in Patients With Nonalcoholic Steatohepatitis	2008/01/07	NCT00586911	Phase 2	Completed	Mayo Clinic
Dietary Supplements for the Treatment of Angelman Syndrome	2006/07/06	NCT00348933	Not Applicable	Completed	University of California, San Diego

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
Cystadane	Rare Disease Therapeutics, Inc.	66621-4000	ORAL	6 g in 1 1	10/10/2010
EnBrace HR	Jaymac Pharma	64661-650	ORAL	500 ug in 1 1	6/9/2019
Betaine Anhydrous for Oral Solution	OAKRUM PHARMA, LLC	72647-900	ORAL	1 g in 1 g	11/10/2022
Cystadane	Recordati Rare Diseases	52276-400	ORAL	1 g in 1 g	1/11/2023
Cystadane	Orphan Europe SARL	52276-401	ORAL	1 g in 1 g	3/5/2018
Betaine Anhydrous For Oral Solution	Lukare Medical, LLC	55792-002	ORAL	1 g in 1 g	5/1/2020
Betaine Anhydrous	Eton Pharmaceuticals, Inc.	71863-115	ORAL	1 g in 1 g	12/18/2023
Betaine	Cosette Pharmaceuticals, Inc.	0713-0352	ORAL	1 g in 1 g	11/4/2022

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
Amversio	SERB SA,Avenue Louise 480,1050 Brussels,Belgium	Authorised	5/5/2022

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
Orthoplex Hydrozyme	19983	Bio Concepts Pty Ltd	Medicine	A	10/3/1991
NATURE'S WEALTH SUPER FAT BURNERS tablet	46341	Nature's Wealth Products	Medicine	A	8/23/1993
Compound Capsules	283037	Australia Natural Foods And Beverages Pty Ltd	Medicine	A	11/29/2016
CYSTADANE betaine 1g/g oral powder bottle	57379	Recordati Rare Diseases Australia Pty Ltd	Medicine	A	9/30/1996
EAGLE TRESOS-B tablet - film coated bottle	15457	Integria Healthcare Australia Pty Ltd	Medicine	A	9/27/1991
EAGLE DIGESTAID tablet film-coated bottle	15464	Integria Healthcare Australia Pty Ltd	Medicine	A	9/27/1991

Health Canada Drug Approvals

Approved Product	Company	DIN	Dosage Form	Strength	Market Date
FORMULA 22158	therapeutic foods co.	00191531	Capsule - Oral	21 MG / CAP	5/18/1994
DIGESTAID TAB	anabolic laboratories inc.	00046728	Tablet - Oral	113 MG	12/31/1964
ENZYME TABLETS	general nutrition canada inc.	02181312	Tablet - Oral	65 MG	10/20/2001
BROMELAIN-PAPAIN	seroyal international inc.	00565504	Tablet - Oral	200 MG	12/31/1981
DYGEST	creative nutrition canada corp.	00665975	Tablet - Oral	90 MG	12/31/1987
BETACOL	therapeutic foods co.	00191388	Capsule - Oral	61 MG	12/15/1989
F-F-C TAB	nutri-west products ltd.	02096846	Tablet - Oral	15 MG / TAB	12/31/1994
BETAINE HCL AND PEPSIN	rheingold food international ltd.	00633690	Tablet - Oral	324 MG	12/31/1985
BETAINE HCL TAB 500MG	gahler enterprises ltd.	01905430	Tablet - Oral	500 MG / TAB	12/31/1991
ULTIMATE TAB	pure life international prods inc.	01946781	Tablet - Oral	25 MG	12/31/1992

CIMA AEMPS Drug Approvals

Approved Product	Company	Registration Number	Pharmaceutical Form	Prescription Type	Status
No CIMA AEMPS (Spain) approvals found for this drug.

Philippines FDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Philippines FDA approvals found for this drug.

Saudi SFDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Saudi SFDA approvals found for this drug.

Malaysia NPRA Drug Approvals

Approved Product	Company	Registration Number	Dosage Form	Strength	Approval Date
No Malaysia NPRA approvals found for this drug.

UK EMC Drug Information

Medicine Name	MA Holder	MA Number	Pharmaceutical Form	Active Ingredient	Authorization Date
No UK EMC drug information found for this drug.

AI-Powered Research

Premium Access