phenylketonuria
Metabolic · 2014–2026
49decisions
23medicines
5carried consumer input
1,046individual submissions
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| glycomacropeptide formula with long chain polyunsaturated fatty acids and docosahexaenoic acid and low in phenylalanine Dietary management of phenylketonuria (PKU) in children from 3 years of age and adults. | Mar 2026 | Recommended with restriction | unclear |
| pegvaliase Treatment of patients aged 16 years and older with phenylketonuria (PKU) who have inadequate blood phenylalanine control (baseline blood Phe level above 600… | Mar 2026 | Deferred | 19 individuals |
| amino acid formula with vitamins and minerals without phenylalanine Dietary management of phenylketonuria (PKU), particularly for those requiring a lower-calorie option. Suitable for use from age 4, predominantly for adults and… | Mar 2024 | Recommended | none received |
| amino acid formula with vitamins, minerals and long chain polyunsaturated fatty acids without phenylalanine Dietary management of phenylketonuria (PKU) in infants. | Mar 2024 | Recommended | none received |
| amino acid formula with fat, carbohydrate, vitamins and minerals without phenylalanine Dietary management of phenylketonuria (PKU) in children aged 3 years and older, and adults with PKU requiring amino acid supplementation. | Nov 2024 | Recommended | none received |
| glycomacropeptide and essential amino acids with vitamins and minerals Dietary management of phenylketonuria (PKU). | Nov 2024 | Recommended with restriction | none received |
| glycomacropeptide and essential amino acids with vitamins and minerals Phenylketonuria (PKU) — continuation of listing for new formulations of glycomacropeptide and essential amino acids nutritional products. | Mar 2023 | Recommended | none received |
| sapropterin Hyperphenylalaninemia (HPA) due to phenylketonuria (PKU) in adult patients who are sapropterin-responsive. | Jul 2022 | Recommended with restriction | 102 individuals Disorders Association · PKU Association of NSW |
| glycomacropeptide formula with amino acids and low phenylalanine Treatment of phenylketonuria (PKU) in children from 3 years of age and adults. | Mar 2022 | Recommended with restriction | input received |
| glycomacropeptide formula with amino acids, vitamins, minerals, trace elements, carbohydrate, fat and low phenylalanine Treatment of phenylketonuria (PKU) in children and adults. | Mar 2022 | Recommended | none received |
| amino acid formula with vitamins and minerals without phenylalanine Phenylketonuria (PKU). This is a continuation of the existing PBS listing with a change in the source of Vitamin A from retinyl acetate to retinyl palmitate. | Nov 2022 | Recommended | none received |
| glycomacropeptide formula with long chain polyunsaturated fatty acids and docosahexaenoic acid and low in phenylalanine Dietary management of patients with phenylketonuria (PKU). | Nov 2022 | Recommended | none received |
| amino acid formula with carbohydrate, vitamins, minerals and trace elements without phenylalanine, supplemented with docosahexaenoic acid Dietary management of phenylketonuria (PKU) or hyperphenylalaninaemia in children from 10 years of age or adults. | Jul 2020 | Not recommended | none received |
| amino acid formula with vitamins and minerals without phenylalanine Dietary management of phenylketonuria (PKU). | Mar 2020 | Recommended with restriction | none received |
| amino acid formula with vitamins and minerals, low phenylalanine and supplemented with docosahexaenoic acid and arachidonic acid Dietary management of phenylketonuria (PKU) in older infants and young children. | Jul 2019 | Recommended | none received |
| glycomacropeptide formula with docosahexaenoic acid and low phenylalanine Dietary management of phenylketonuria (PKU). | Jul 2019 | Recommended | none received |
| tyrosine with carbohydrate Dietary management of phenylketonuria (PKU) in patients aged 3 years and over. | Jul 2019 | Recommended with restriction | none received |
| amino acid formula with carbohydrate without phenylalanine Dietary management of phenylketonuria (PKU). | Nov 2019 | Recommended | none received |
| glycomacropeptide and essential amino acids with vitamins and minerals Phenylketonuria (PKU). The submission requested a formulation change to include docosahexaenoic acid (DHA), lower phenylalanine levels, and other changes to… | Nov 2019 | Recommended with restriction | none received |
| amino acid formula with vitamins, minerals and long chain polyunsaturated fatty acids, without phenylalanine Dietary management of phenylketonuria (PKU) in infants. | Jul 2018 | Recommended with restriction | none received |
| sapropterin Treatment of hyperphenylalaninemia (HPA) caused by phenylketonuria (PKU) in sapropterin-responsive patients. | Mar 2018 | Deferred | 919 individuals Metabolic Dietary Disorders Association |
| glycomacropeptide and essential amino acids with vitamins and minerals Treatment of phenylketonuria (PKU). | Nov 2018 | Recommended with restriction | unclear |
| glycomacropeptide formula with docosahexaenoic acid with low phenylalanine Treatment of phenylketonuria (PKU) in patients aged 3 years or older. | Nov 2018 | Recommended | unclear |
| sapropterin Treatment of hyperphenylalaninaemia (HPA) caused by phenylketonuria (PKU). Initial responsiveness testing and continuing treatment in patients under 18 years… | Nov 2018 | Recommended with restriction | 6 individuals |
| amino acid formula with vitamins and minerals, without phenylalanine Dietary management of phenylketonuria in patients aged four years and above (for the liquid formulation) and eight years and above (for the powder formulation). | Jul 2017 | Recommended with restriction | none received |
| glycomacropeptide formula with long chain polyunsaturated fatty acids and docosahexaenoic acid and low phenylalanine Dietary management of phenylketonuria. | Jul 2017 | Recommended with restriction | none received |
| amino acid formula with vitamins and minerals, low phenylalanine and supplemented with docosahexaenoic acid and arachidonic acid Treatment of phenylketonuria (PKU) in patients aged 6 months to 5 years as a concentrated, low volume protein substitute for the weaning process. | Mar 2017 | Recommended with restriction | none received |
| amino acid formula with vitamins and minerals without phenylalanine Dietary management of phenylketonuria (PKU). | Mar 2017 | Recommended | none received |
| amino acid formula with vitamins and minerals without phenylalanine Dietary management of phenylketonuria (PKU). | Mar 2017 | Recommended | none received |
| amino acid formula with carbohydrate, vitamins, minerals and trace elements without phenylalanine, supplemented with docosahexaenoic acid Dietary management of phenylketonuria (PKU) or hyperphenylalaninaemia in children from 10 years of age or adults. | Nov 2017 | Not recommended | none received |
| glycomacropeptide and essential amino acids with vitamins and minerals Treatment of Phenylketonuria (PKU) in patients requiring medical foods for protein supplementation. | Nov 2017 | Recommended | none received |
| glycomacropeptide and essential amino acids with vitamins and minerals Phenylketonuria (PKU) in children from 12 months, pregnant or lactating women, and adults. | Jul 2016 | Recommended with restriction | none received |
| Amino acid formula with fat, carbohydrate, vitamins, minerals and long chain polyunsaturated fatty acids without phenylalanine and supplemented with docosahexaenoic acid Phenylketonuria (PKU) in infants. | Mar 2016 | Recommended | none received |
| amino acid formula with carbohydrate, vitamins, minerals and trace elements without phenylalanine Phenylketonuria. A nutritional formula for dietary management of PKU in patients aged 6 months to 10 years. | Mar 2016 | Recommended | none received |
| Glycomacropeptide and essential amino acids Treatment of phenylketonuria (PKU). | Nov 2016 | Recommended with restriction | none received |
| Glycomacropeptide and essential amino acids with vitamins and minerals Phenylketonuria (PKU) in patients 10 years and older. | Nov 2016 | Recommended with restriction | none received |
| Amino acid formula without phenylalanine Treatment of phenylketonuria (PKU) in adult patients. | Nov 2016 | Recommended | none received |
| amino acid formula with vitamins and minerals without phenylalanine Management of phenylketonuria (PKU). PKU Air is an amino acid formula without phenylalanine for dietary management of this inherited metabolic disorder. | Mar 2015 | Recommended with restriction | no section in document |
| amino acid formula with vitamins and minerals Nutritional management of phenylketonuria (PKU), tyrosinaemia (TYR), and maple syrup urine disease (MSUD) in children. | Mar 2015 | Recommended | no section in document |
| glycomacropeptide and essential amino acids with vitamins and minerals Phenylketonuria (PKU) — a new ready-to-drink oral liquid dose presentation (10 g and 15 g protein per 250 mL carton). | Mar 2015 | Recommended with restriction | no section in document |
| amino acid formula with vitamins and minerals without phenylalanine Phenylketonuria (PKU). A medical food for the nutritional management of patients with PKU. | Nov 2015 | Recommended | none received |
| amino acid formula with vitamins and minerals without phenylalanine Phenylketonuria (PKU). Amino acid formula for oral administration as a nutritional product for patients with phenylketonuria. | Nov 2015 | Recommended with restriction | none received |
| amino acid formula with vitamins and minerals without phenylalanine Phenylketonuria (PKU). A nutritional product for dietary management of patients with PKU across the age range of 6 months to 10 years. | Nov 2015 | Deferred | none received |
| amino acid formula with vitamins, minerals and long chain polyunsaturated fatty acids without phenylalanine Phenylketonuria in infants requiring oral amino acid formula without phenylalanine. | Nov 2015 | Deferred | none received |
| amino acid formula without phenylalanine Treatment of phenylketonuria (PKU) in children and adults. | Nov 2015 | Recommended with restriction | none received |
| glycomacropeptide and essential amino acids with vitamins and minerals Phenylketonuria (PKU) — a pack size upgrade from 28 sachets to 30 sachets per pack. | Nov 2015 | Recommended with restriction | none received |
| amino acid formula with vitamins and minerals without phenylalanine Treatment of patients with phenylketonuria (PKU). The submission sought to inform the PBAC of additional flavour variants and minor nutritional upgrades within… | Jul 2014 | Noted | no section in document |
| amino acid formula with vitamins and minerals without phenylalanine Nutritional support for phenylketonuria (PKU). Upgrade in nutritional formula, flavour and packaging. | Nov 2014 | Deferred | no section in document |
| amino acid formula with vitamins and minerals without phenylalanine Phenylketonuria (PKU). A nutritional formula for patients requiring management of PKU through dietary protein restriction and phenylalanine avoidance. | Nov 2014 | Deferred | no section in document |
Who spoke
Disorders Association · PKU Association of NSW · Metabolic Dietary Disorders Association
What the PBS pays
$281M in government benefit over 2024–25, across 281,009 services.
This is spend on the medicines considered for this condition, not spend on the disease. A medicine used for several conditions is counted in full under each, so figures across conditions add to more than national PBS expenditure.
| Medicine | Government benefit |
|---|---|
| amino acid formula with vitamins, minerals and long chain polyunsaturated fatty acids, without phenylalanine | $21.4M |
| amino acid formula with carbohydrate, vitamins, minerals and trace elements without phenylalanine, supplemented with docosahexaenoic acid | $20.3M |
| amino acid formula with fat, carbohydrate, vitamins, minerals and long chain polyunsaturated fatty acids without phenylalanine and supplemented with docosahexaenoic acid | $20.3M |
| glycomacropeptide formula with amino acids, vitamins, minerals, trace elements, carbohydrate, fat and low phenylalanine | $20.3M |
| amino acid formula with vitamins and minerals, low phenylalanine and supplemented with docosahexaenoic acid and arachidonic acid | $19.4M |
| amino acid formula with carbohydrate, vitamins, minerals and trace elements without phenylalanine | $18M |
| amino acid formula with fat, carbohydrate, vitamins and minerals without phenylalanine | $18M |
| sapropterin | $17.3M |
| Amino acid formula with vitamins and minerals (three formulations: without phenylalanine; without phenylalanine and tyrosine; without valine, leucine and isoleucine) | $17.2M |
| amino acid formula with vitamins and minerals, without phenylalanine | $17M |
| amino acid formula with vitamins, minerals and long chain polyunsaturated fatty acids without phenylalanine | $17M |
| amino acid formula with vitamins and minerals without phenylalanine | $17M |
Appraised elsewhere
NICE, in England and Wales, has appraised 1 technology for this condition, recommending 1 . A different committee, a different population and a different price: this is what has been looked at there, not a verdict on what happened here.
| Technology | Appraisal | Outcome | Date |
|---|---|---|---|
| sapropterinSapropterin for treating hyperphenylalaninaemia in phenylketonuria | TA729 | recommended restricted | 2021-09 |
Conditions are classified by a language model from the indication text of each submission, so an individual label here can be wrong. Consumer input is read from the document's own consumer comments section: 5 of 49 decisions since 2014 carried some. Silence means no comment reached the committee through that facility, not that nobody was affected. More on how this was built.