Cystadane® (betaine anhydrous for oral solution) has long been a cornerstone in the treatment of homocystinuria, a rare genetic disorder characterized by elevated levels of homocysteine in the blood and urine. Approved by the U.S.

Food and Drug Administration (FDA) in 1996, Cystadane marked a significant advancement in managing this condition, which, if left untreated, can lead to serious complications such as cardiovascular disease, skeletal abnormalities, and developmental delays.​

Cystadane Clinical-Pharmacology

When administered in recommended oral dosage to children or adults, Cystadane acts as a methyl group donor in the remethylation of homocysteine to methionine in patients with homocystinuria.

As a result, toxic blood levels of homocysteine are reduced in these patients, usually to 20-30 percent or less of pre-treatment levels.

Elevated homocysteine blood levels are associated with clinical problems such as a cardiovascular thrombosis, osteoporosis, skeletal abnormalities, and optic lens dislocation. Plasma levels of homocysteine were decreased in nearly all patients treated with betaine.

In observational studies without concurrent controls, clinical improvement was reported by the treating physicians in about three-fourths of patients taking betaine.

Many of these patients were also taking other therapies such as vitamin B6 (pyridoxine), vitamin B12 (cobalamin), and folate with variable biochemical responses. In most cases studied, adding betaine resulted in a further reduction of homocysteine.

Betaine has also been demonstrated to increase low plasma methionine and S-adenosylmethionine (SAM) levels in patients with MTHFR deficiency and cbl defect.

Cystadane Innovation In The Field Of Generics

Cosette Pharmaceuticals also entered the market with its AB-rated generic version, further expanding access to treatment. These developments underscore a growing commitment within the pharmaceutical industry to address the needs of patients with rare diseases by providing cost-effective and accessible therapies.​

As the availability of generic options increases, healthcare providers and patients can collaborate to choose the most suitable and affordable treatment plans.

The introduction of these generics not only enhances patient access but also fosters a competitive market that may lead to further innovations in the treatment of homocystinuria.

In A Word

The introduction of a generic option underscores the importance of accessibility in treating rare diseases.

With multiple therapeutic choices now available, patients and healthcare providers can collaborate to select the most suitable treatment plan based on efficacy, cost, and individual patient needs.​

As research continues and new therapies emerge, the management of homocystinuria is poised to become more effective and patient-centered.

The ongoing commitment of pharmaceutical companies to develop and distribute treatments like Cystadane and its generics plays a crucial role in improving the quality of life for those affected by this rare disorder.

About DengYueMed – HK Drug Wholesale Distributor

As a legally compliant drug import and export company, DengYueMed is certified by the pharmacy & poisons board of Hong Kong — you can verify our qualification on their official website.

Our efforts to improve the affordability of Cystadane drug aim to ensure that more patients can benefit from this important medication.

HK DengYue provides detailed medicine information, transparent pricing, and responsive support to ensure a smooth and reliable buying experience.

Feel free to reach out anytime to discuss your needs or ask questions about the medicine. We welcome you to contact us for a consultation.