Drugs / OLACAFTOR
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT03486236 | Jul → Sep 2016 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 21 trial · 1 met primary | ||||||
| Phase 2 | NCT02951182 | Oct 2016 → Aug 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
News releases announcing trial results or a regulatory action · 4
| Date | Issuer | Release |
|---|---|---|
| 2018-06-07 | Vertex Pharmaceuticals Incorporated | Results Vertex Data Presented at European Cystic Fibrosis Society (ECFS) Conference Demonstrate Early and Long-Term Disease-Modifying Potential of Treating the Underlying Cause of CF vrtx.com ↗
Data from the ongoing Phase 3, open-label ARRIVALstudy presented at an oral session and published online today in The Lancet Respiratory Medicine show that treatment with KALYDECO® (ivacaftor) resulted in substantial decreases in mean sweat chloride as well as improvements to multiple efficacy endpoints, suggesting the potential to preserve pancreatic function and modify the course of CF beginning in children as young as one year of age. |
| 2018-02-12 | Vertex Pharmaceuticals Incorporated | Regulatory FDA Approves SYMDEKOTM (tezacaftor/ivacaftor and ivacaftor) to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older with Certain Mutations in the CFTR Gene vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) approved SYMDEKO™ (tezacaftor/ivacaftor and ivacaftor) for treating the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or who have at least one mutation that is responsive to tezacaftor/ivacaftor. |
| 2017-11-03 | Vertex Pharmaceuticals Incorporated | Results Vertex Announces Presentations of Data at North American Cystic Fibrosis Conference that Demonstrate Important Progress Toward Goal of Helping All People with CF vrtx.com ↗
All studies showed statistically significant improvements in lung function (ppFEV1) with a triple combination regimen in these patients. |
| 2017-07-18 | Vertex Pharmaceuticals Incorporated | Results Vertex Announces Positive Phase 1 & Phase 2 Data from Three Different Triple Combination Regimens in People with Cystic Fibrosis Who Have One F508del Mutation and One Minimal Function Mutation (F508del/Min) vrtx.com ↗
Phase 2 data showed mean absolute improvements in ppFEV1 of 9.7 and 12.0 percentage points for VX-152 and VX-440, respectively, in triple combination with tezacaftor and ivacaftor in F508del/Min patients |
All press releases naming this drug 5 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | OLACAFTOR | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297649 ↗ |
| Known as | VX-440 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02951182 ↗ |
| Action | Activate | “Cystic fibrosis transmembrane conductance regulator positive modulator” CHEMBL4297649 ↗ |
| Modality | Small molecule | — CHEMBL4297649 ↗ |
| Target | CFTR | “Cystic fibrosis transmembrane conductance regulator positive modulator” CHEMBL4297649 ↗ |