Advice
following a second resubmission
betaine anhydrous (Cystadane) is accepted for restricted use within NHS Scotland.
Indication under review: adjunctive treatment of homocystinuria involving deficiencies or defects in cystathionine beta-synthase (CBS), 5,10-methylene-tetrahydrofolate reductase (MTHFR) or cobalamin cofactor metabolism (cbl).
SMC restriction: patients who are not responsive to vitamin B6 treatment.
Limited clinical data confirmed the effectiveness of betaine anhydrous in homocystinuria, There remains some uncertainty about the cost-effectiveness of betaine anhydrous even in the restricted patient group described above, but given the orphan nature of the condition the economic case for use was accepted.
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Medicine details
- Medicine name:
- betaine anhydrous (Cystadane)
- SMC ID:
- 407/07
- Indication:
- Adjunctive treatment of homocystinuria involving deficiencies or defects in cystathionine beta-synthase (CBS), 5,10-methylene-tetrahydrofolate reductase (MTHFR) or cobalamin cofactor metabolism (cbl).
- Pharmaceutical company
- Orphan Europe (UK) Ltd
- BNF chapter
- Nutrition and blood
- Submission type
- Resubmission
- Status
- Restricted
- Date advice published
- 09 August 2010