Lumacaftor

AllianceRx Walgreens Prime study demonstrates favorable adherence, but high cost for cystic fibrosis patients prescribed modulator medications

Retrieved on: 
Wednesday, November 6, 2019

ORLANDO, Fla., Nov. 6, 2019 /PRNewswire/ --AllianceRx Walgreens Prime and Duquesne University School of Pharmacy recently announced results from research analyzing data from cystic fibrosis (CF) patients prescribed cystic fibrosis transmembrane conductance regulator(CFTR) modulator medications.

Key Points: 
  • ORLANDO, Fla., Nov. 6, 2019 /PRNewswire/ --AllianceRx Walgreens Prime and Duquesne University School of Pharmacy recently announced results from research analyzing data from cystic fibrosis (CF) patients prescribed cystic fibrosis transmembrane conductance regulator(CFTR) modulator medications.
  • Researchers wanted to understand if patients took their medications as prescribed, as well as the financial impact of the medications on patients.
  • Since the introduction of the first CFTR modulator in 2012 and subsequent CFTR modulator approvals, these medications have significantly changed the treatment of CF.
  • Of the 3,482 patients using CFTR modulator therapies, 50.8% of patients were on lumacaftor/ivacaftor followed by tezacaftor/ivacaftor (24.8%) and ivacaftor (24.4%).

Vertex Announces New Access Agreement with Scottish Government for ORKAMBI® (lumacaftor/ivacaftor) and SYMKEVI® (tezacaftor/ivacaftor)

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Thursday, September 12, 2019

Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced that eligible cystic fibrosis patients living in Scotland will now have access to ORKAMBI (lumacaftor/ivacaftor) and SYMKEVI (tezacaftor/ivacaftor) in combination with ivacaftor following the signing of an access agreement.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced that eligible cystic fibrosis patients living in Scotland will now have access to ORKAMBI (lumacaftor/ivacaftor) and SYMKEVI (tezacaftor/ivacaftor) in combination with ivacaftor following the signing of an access agreement.
  • It means that approximately 400 eligible cystic fibrosis patients in Scotland now have access to ORKAMBI or SYMKEVI.
  • Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.
  • Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

Vertex to Present Data at ECFS Conference on Potential Impact of Early Treatment and Long-Term Treatment with CFTR Modulators on the Underlying Cause of CF

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Tuesday, June 4, 2019

KALYDECO (ivacaftor) is the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene.

Key Points: 
  • KALYDECO (ivacaftor) is the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene.
  • People with CF who have specific mutations in the CFTR gene are currently indicated for KALYDECO in different countries across North America, Europe and other International markets.
  • Patients should talk to their doctor to learn if they have an indicated CFTR gene mutation.
  • KALYDECO (ivacaftor), ORKAMBI (lumacaftor/ivacaftor), SYMDEKO/SYMKEVI (tezacaftor/ivacaftor and ivacaftor), and VX-445 (elexacaftor) were discovered by Vertex as part of this collaboration.

Two Phase 3 Studies of the Triple Combination of VX-445, Tezacaftor and Ivacaftor Met Primary Endpoint of Improvement in Lung Function (ppFEV1) in People with Cystic Fibrosis

Retrieved on: 
Wednesday, March 6, 2019

We look forward to submitting global regulatory applications for one of these triple combination regimens for both patient populations later this year.

Key Points: 
  • We look forward to submitting global regulatory applications for one of these triple combination regimens for both patient populations later this year.
  • The study randomized 405 patients, and 403 patients received at least one dose of either the VX-445 triple combination regimen or triple placebo.
  • All of the 107 patients who received either the triple combination of VX-445, tezacaftor and ivacaftor or placebo, tezacaftor and ivacaftor completed the 4-week triple combination treatment period.
  • KALYDECO (ivacaftor), ORKAMBI (lumacaftor/ivacaftor), SYMDEKO (tezacaftor/ivacaftor and ivacaftor), VX-659 and VX-445 were discovered by Vertex as part of this collaboration.

Vertex Announces European Commission Approval for ORKAMBI® (lumacaftor/ivacaftor) for Treatment of Children with Cystic Fibrosis Aged 2 to 5 Years Old with Most Common Form of the Disease

Retrieved on: 
Monday, January 21, 2019

By treating the underlying cause of disease early, we can potentially modify its course and offer patients the chance of improved outcomes.

Key Points: 
  • By treating the underlying cause of disease early, we can potentially modify its course and offer patients the chance of improved outcomes.
  • Lumacaftor/ivacaftor is already approved in the EU for the treatment of CF in patients aged 6 and older who have two copies of the F508del mutation.
  • Cystic fibrosis is a rare, life-shortening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.
  • Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.