betaine anhydrous (Cystadane)

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.