A Cystic Fibrosis Drug: VX-770

School of Science and Technology

Nazarbayev University

Luiza Niyazmetova
October 11, 2011
Outline
 Introduction
 Structure and properties of VX-770
 Stages of development and study
 Clinical application
 Conclusion
VX-770. Introduction.
 Developed by Vertex Pharmaceuticals company to
treat abnormal gene and its protein CFTR (Cystic
fibrosis transmembrane regulator)
 Cystic Fibrosis – inherited chronic disorder:
 defective gene and protein cause producing very
thick mucus in lungs and digestive tract which leads
to infections, inability to break down and absorb food
properly (patients are underweight)
 Status of development: not completed
 VX-770 was chosen among 228,000 tested chemicals
Structure and properties of VX-770
 CFTR potentiator
 C H N O Ivacaftor (alternative name of VX-
24 28 2 3
770)
 Used for patients with G551D and F508del

mutation
Stages of development
 Pre-Discovery→ Discovery→ Pre-clinical testing
 → Clinical trials:

1. Phase 1: 50 individuals (both healthy and

patients) were involved in testing, developing the
dosage
2. Phase 2: combination of VX-770 and VX-809
(CFTR corrector) to treat people with 2 copies of
F508del mutation (62 patients involved)
3. Phase 3 (completed): 161 patients (6-12 years
old) with the mutation G551 D, were involved
4. October 2011- drug will be submitted to the US
FDA (Food and Drug Administration)
5. If accepted → next stage - Manufacturing
Clinical application
VX-770 is used for:
 Cystic Fibrosis caused by mutations G551D and

F508del
 Increase functioning of defective CFTR protein by

enhancing the transport of ions across the membrane

Results (according to clinical tests on a group of
patients):
 Improved lung functions

 Improved functions of digestive system (gaining of

weight by 3.1 kg on average)

 Reduction of salt amount in sweat

 Lower rate of hospitalization

Conclusion
 VX-770 is designed to treat patients with cystic
fibrosis caused by either G551D or F508del
mutation
 Phase 3 of clinical trials has been completed, the

drug will be checked by committee and will start

to be manufactured
 VX-770 helped to lower the hospitalizing among

patients involved in phase 3 of clinical testing

 October 2011 the drug will be submitted to FDA

to get the permission for manufacturing VX-770

Reference list
FAQs About VX-770. (2011). Retrieved from
http://www.cff.org/research/ClinicalResearch/FAQs/VX-770/
Ivacaftor. Image. (2011). Retrieved from http://t0.gstatic.com/images?
q=tbn:ANd9GcQACYb6aJlhegtylt1wcO8pLaOnhtWzUMFJs6FusA9n8qbx
1UqL
VX-770 (Cystic Fibrosis). (2011). Retrieved from
http://www.vrtx.com/current-projects/drug-candidates/vx-770.html
Vertex Pharmaceuticals, Inc. (2011). Retrieved from
http://www.biotechnologyevents.com/node/2399
Vertex Pharmaceuticals Initiates Phase I Development for VX-770 in
Cystic Fibrosis. (2006). Retrieved from
http://www.cff.org/UploadedFiles/aboutCFFoundation/NewsEvents/20
06NewsArchive/770_Phase1_FINAL_PR3.doc.pdf
Vertex's New Cystic Fibrosis Drug Treats the Cause, Not Just the
Symptoms. (2011). Retrieved from
http://www.dailyfinance.com/2011/02/24/vertex-new-cystic-
fibrosis-drug-vx770-treats-cause-not-symptoms/