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ORKAMBI (Vertex Pharmaceuticals Australia Pty Ltd)
Product name
ORKAMBI
Date registered
Evaluation commenced
Decision date
Approval time
221 working days (255)
Active ingredients
lumacaftor/ivacaftor
Registration type
EOI
Indication
ORKAMBI (granules) is now also indicated for the treatment of cystic fibrosis (CF) in patients age 2 to 5 years who are homozygous for the F508del mutation in the CFTR gene.
Registration process
Orphan drug
Sponsors receive a fee waiver to help bring medicines for a small population to market.