BioMarin Pharmaceutical Australia

11 PBAC submissions on file, active 2007–2022. 55% have been recommended.

Drug portfolio

Sorted by PBS spend captured. PBS expenditure is credited to the sponsor with the most recent Recommended decision per drug, so biosimilars and originator brands aren't double-counted.

• Sapropterin
  Metabolic · Noted · 2022—
• Elosulfase alfa
  Metabolic · Not recommended · 2016—
• Cerliponase alfa
  Rare disease · Recommended with restriction · 2018—
• Galsulfase
  Rare disease · Not recommended · 2007—
• Pegvaliase
  Metabolic · Recommended with restriction · 2022—
• Vosoritide
  Rare disease · Recommended with restriction · 2022—

Therapy area mix

Activity by year

2007 (1) · 2014 (1) · 2016 (1) · 2018 (3) · 2019 (1) · 2020 (1) · 2022 (3)

Rejection patterns

Across 3 Not recommended decisions on file.

• 1×unclear clinically significant clinical benefit compared to placebo
• 1×unreliable and unacceptably high ICER (more than $200
• 1×000/QALY)
• 1×uncertain clinical trial population applicability
• 1×concerns about duration of economic evaluation