Amino acid formula with vitamins and minerals without methionine and supplemented with arachidonic acid and docosahexaenoic acid

PBAC's latest decision on Amino acid formula with vitamins and minerals without methionine and supplemented with arachidonic acid and docosahexaenoic acid: Recommended with restriction (2023). Considered for Dietary management of pyridoxine non-responsive homocystinuria (HCU), expected to be used in infants and young children from 6 months to 5 years of age.

Eligible population

Infants and young children from 6 months to 5 years of age with pyridoxine non-responsive homocystinuria.

Therapy area

Metabolic

Line of therapy

Not applicable

Evidence base

Cost-minimisation

Primary endpoint

Cost-minimisation

Comparator

HCU gel

Economic model

Cost-minimisation

Eligible patients/year

1

ICER note

Cost-minimisation analysis; no ICER calculated by design.

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