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Vertex to Present New Data on ALYFTREK® at the North American Cystic Fibrosis Conference

VRTX
  • Interim data from the ALYFTREK open-label extension study in children aged 2 to 5 years shows recovery of exocrine pancreatic function in some participants, allowing discontinuation of pancreatic enzyme replacement therapy (PERT).
  • The study involved 48 children in a PERT discontinuation substudy, with 37.5% successfully stopping PERT for a mean duration of 8.6 weeks at the time of interim analysis.
  • Mean fecal elastase-1 (FE-1) levels increased by 110.2 µg/g from baseline to 239.7 µg/g, with 48.6% of children reaching the pancreatic sufficiency threshold of ≥200 µg/g.
  • Across the full enrolled cohort of 66 children, 50% were not on PERT at the time of the data cut, suggesting reversal of prior pancreatic exocrine organ failure.
  • The use of ALYFTREK in children with cystic fibrosis aged 2 to 5 years remains investigational as it is currently indicated for patients aged 6 years and older.
  • Additional abstracts presented at the North American Cystic Fibrosis Conference covered long-term benefits of vanzacaftor/tezacaftor/deutivacaftor in reducing IV antibiotic use, natural history of exocrine pancreatic function in infants, & the lifetime treatment burden of PERT.
  • ALYFTREK carries a Boxed Warning for drug-induced liver injury & liver failure, requiring liver function test monitoring prior to initiation, monthly for the first 6 months, every 3 months for the next 12 months, & annually thereafter.
  • ALYFTREK is not recommended for patients with moderate hepatic impairment & should not be used in patients with severe hepatic impairment, while TRIKAFTA requires dosage reduction & close monitoring in moderate impairment cases.
  • Warnings include risks of hypersensitivity reactions, intracranial hypertension, neuropsychiatric events including suicidal thoughts, & cataracts in pediatric patients treated with TRIKAFTA.
  • Concomitant use of strong or moderate CYP3A inducers is not recommended due to decreased drug exposure, while moderate or strong CYP3A inhibitors require dose reductions for both ALYFTREK & TRIKAFTA.
  • Serious adverse reactions more frequent with ALYFTREK than ELX/TEZ/IVA included influenza, increased AST, increased GGT, depression, & syncope.
  • Vertex is a global biotechnology company with approved therapies for cystic fibrosis, sickle cell disease, & other serious conditions, headquartered in Boston with international operations in London.