Shots: The two P-III studies involve assessing of VX-445 + tezacaftor + ivacaftor vs triple PBO & PBO + tezacaftor + ivacaftor in 405 & 108 patients with one F508del […]readmore
Tags : Cystic Fibrosis
Shots: The approval is based on P-III ARRIVAL study results assessing Kalydeco in 25 children with CF who have either of 10 mutations in CFTR gene (G551D, G178R, S549N, S549R, […]readmore
Regulatory
Vertex‘s Orkambi (lumacaftor/ivacaftor) Receives Health Canada Approval for Cystic Fibrosis
Shots: The approval is based on P-III study results assessing Orkambi in 60 patients with CF, who have two copies of the F508del CFTR mutation aged 6 to 11 years for 24 wks. […]readmore
Regulatory
Proteostasis’ PTI-428 Receives FDA’s Breakthrough Therapy Designation for Cystic Fibrosis
Shots: The FDA’s BT designation is based on P-II study results assessing PTI-428 (50 mg, qd) vs PBO in 24 CF with F508del mutation on background treatment with Orkambi for […]readmore