Amino acid formula (multiple formulations for pku, tyrosinaemia, ga1, mma/pa, hcu, msud)

PBAC's latest decision on Amino acid formula (multiple formulations for pku, tyrosinaemia, ga1, mma/pa, hcu, msud): Recommended with restriction (2020). Considered for Dietary management of proven inborn errors of amino acid metabolism: Phenylketonuria (PKU), Tyrosinaemia (TYR), Glutaric Aciduria Type 1 (GA1), Methylmalonic acidaemia and Propionic acidaemia (MMA/PA), Pyridoxine non-responsive homocystinuria (HCU), and Maple syrup urine disease (MSUD) in infants from birth to 12 months and as supplementary feed up to 3 years of age.

Eligible population

Infants aged birth to 12 months with proven phenylketonuria, tyrosinaemia, glutaric aciduria type 1, methylmalonic acidaemia, propionic acidaemia, or maple syrup urine disease; use as supplementary feed up to 3 years of age.

Therapy area

Metabolic

Line of therapy

Not applicable

Evidence base

Cost-minimisation

Economic model

Not modelled

ICER note

Not modelled; Committee Secretariat formulation change submission with no new economic evaluation

Similar precedents

• Amino acid formula with vitamins and minerals (three formulations: without phenylalanine; without phenylalanine and tyrosine; without valine, leucine and isoleucine)
  similarity 0.83
• Amino acid formula with vitamins and minerals (multiple formulations)
  similarity 0.78
• Amino acid formula with vitamins, minerals and long chain polyunsaturated fatty acids without phenylalanine
  similarity 0.78
• Amino acid formula with vitamins and minerals (msud, tyr, hcu coolers)
  similarity 0.76
• Amino acid formula with vitamins, minerals and long chain polyunsaturated fatty acids, without phenylalanine
  similarity 0.75
• Amino acid formula with fat, carbohydrate, vitamins, minerals and long chain polyunsaturated fatty acids without phenylalanine and supplemented with docosahexaenoic acid
  similarity 0.75