Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Label indication KALYDECO is indicated for the treatment of cystic fibrosis (CF) in patients aged 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data [see Clinical Pharmacology (12.1) and Clinical Studies (14) ] . FDA label
Also known as IVA, VX-770, Kalydeco
Ivacaftor · 24 trials · 4 indications
Absolute change in percent predicted in 1 second FEV1 from baseline through week 24
LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
AE: any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, Inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. AEs with start date or increased severity on or after the first dose of study drug through the end of study participation was considered treatment-emergent.
AE: any untoward medical occurrence in a participants during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after informed consent form is signed. AE includes serious as well as non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, In-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. TEAEs were defined as adverse events with start date or increased severity on and after the first dose of study drug through Week 108.
AE: any adverse change from participant's baseline (pre-treatment) condition, including any adverse experience, abnormal recording/clinical laboratory assessment which occurs during course of study, whether it is considered related to study drug or not. SAE: medical event or condition, which falls into any of following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolonged hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect, important medical event. Related AEs includes all AEs for which the causality was either related to study drug or possibly related to study drug. Data was reported as per the dose received and for overall participants.
AE: any adverse change from participant's baseline (pre-treatment) condition, including any adverse experience, abnormal recording/clinical laboratory assessment which occurs during course of study, whether it is considered related to study drug or not. AE includes both serious and non-serious AE. SAE: medical event or condition, which falls into any of following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolonged hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect, important medical event. Related AEs includes all AEs for which the causality was either related to study drug or possibly related to study drug. Data was reported as per the dose received.
Plasma concentration was reported for ivacaftor and its metabolites (hydroxymethyl ivacaftor \[M1\] and ivacaftor carboxylate \[M6\]) up to 24 hours post-dose on Day 4 (Hour 0 \[pre-dose\] on Day 1 and Day 4; 2, 3, 6, 24 hours post-dose on Day 4). Data was planned to be reported for overall participants in the period.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male subjects 18 years and older and female subjects 16 years and older. The Wang standard was used for male subjects aged 6 to 17 years and for female subjects aged 6 to 15 years. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during study Part 1.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male subjects 18 years and older and female subjects 16 years and older. The Wang standard was used for male subjects aged 6 to 17 years and for female subjects aged 6 to 15 years. Absolute change in percent predicted FEV1 over 24 weeks of ivacaftor treatment (from Week 12 \[Part 1: Treatment Period 2\] through Week 36 \[Part 2\]) was reported for subjects who received ivacaftor in Part 1: Treatment Period 2, as per planned analysis. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during Part 1: Treatment Period 2.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male subjects 18 years and older and female subjects 16 years and older. The Wang standard was used for male subjects aged 6 to 17 years and for female subjects aged 6 to 15 years.
Adverse Event: any untoward medical occurrence in a participant during the study, including any unfavorable and unintended sign, symptom, or disease whether or not it was considered to be study drug related. This included any newly occurring event or previous condition that increased in severity or frequency after obtaining informed consent and assent (where applicable). SAE: medical event or condition, which resulted in any of following, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolonged hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect, important medical event. Non-Serious AEs included all AEs except SAEs.
Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.
Safety of ivacaftor will be determined by number of participants with adverse events (including serious adverse events).
Number of participants with abnormal serum chemistry values compared to screening values will also be used to determine safety of ivacaftor.
Safety of ivacaftor will also be determined by number of participants with abnormal changes in their screening hematology values.
Number of participants with abnormal changes in their screening ECGs is another factor that will be used to evaluate the safety of ivacaftor.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years. Data was to be reported for each cycle (Cycle 1 and Cycle 2) and as per drug treatment, for overall participants and as per genotype (residual function mutation and mRNA splice site mutation).
Lung clearance index (LCI) is a measure of ventilation inhomogeneity that is derived from a multiple-breath washout test. The LCI was calculated as the number of lung volume turnovers (cumulative expired volume divided by the functional residual capacity \[FRC\]) required to reduce end-tidal SF6 concentration to 1/40th of the starting value.
Subjects inhaled hyperpolarized helium-3 (3He) gas mixed with nitrogen to make a total volume of approximately one-third forced vital capacity (FVC) to a maximum of 1 liter and hold their breath for 20 seconds or less. Rapid magnetic resonance imaging (MRI) was performed during inhalation/exhalation and/or breath-hold. Areas of decreased ventilation were observed as ventilation defects that are visualized as decreased (and/or absent) 3He intensity in 3He-MRI. The total ventilation defect was defined as the ratio of total ventilation defect volume (L) to total lung volume (L), expressed as a percentage. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug in VX-770 treatment phase (Day 15 to 42).
Subjects were asked to inhale hyperpolarized 3 He gas mixed with nitrogen to make a total volume of approximately one-third forced vital capacity (FVC) to a maximum of 1 liter and hold their breath for 20 seconds or less. Rapid MRI was performed during inhalation/exhalation and/or breath-hold. Areas of decreased ventilation were observed as ventilation defects that are visualized as decreased (and/or absent) 3He intensity in 3He MRI. The total ventilation defect was defined as the ratio of total ventilation defect volume (L) to total lung volume (L), expressed as a percentage. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug in Part B (48 weeks).
Adverse event data were collected up to the follow-up visit (5 to 9 days after last dose of study drug). Serious adverse events that were ongoing at the follow-up visit were followed until the event resolved, returned to baseline, or was determined to be a stable or chronic condition.
Adverse event data were collected up to the follow-up visit (5 to 9 days after last dose of study drug). Serious adverse events that were ongoing at the follow-up visit were followed until the event resolved, returned to baseline, or was determined to be a stable or chronic condition.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
PK Parameters - maximum observed plasma concentrations (Cmax) and area under the concentration versus time curve (AUC),
PK parameters - maximum observed plasma concentrations (Cmax) and area under the concentration versus time curve (AUC),
| Arm | Type | Description |
|---|---|---|
| Ivacaftor | EXPERIMENTAL | There is only one arm to this study. The two sisters with Cystic Fibrosis will both receive Ivacaftor for 6 months for their treatment. |
| Ivacaftor Arm | EXPERIMENTAL | Participants less than (\<) 24 months of age and weighing 5 to less than (\<) 7 kilogram (kg) received 25 mg IVA every 12 hours (q12h), 7 to \<14 kg received 50 mg IVA q12h, and those weighing 14 to \<25 kg received 75 mg IVA q12h. Participants more than or equal (\>=) 24 months of age and weighing \<14 kg received 50 mg IVA q12h, and those weighing more than or equal to (\>=)14 kg received 75 mg IVA q12h in the treatment period for up to 96 weeks. Doses were determined based on safety and pharmacokinetic (PK) data from parent study, age and weight. |
| Observational Arm | NO_INTERVENTION | - |
| Sequence 1: First Ivacaftor (IVA) Then Placebo | EXPERIMENTAL | Participants received IVA 150 milligram (mg) every 12 hours (q12h) for 8 weeks in treatment period 1 followed by placebo matched to IVA for 8 weeks in treatment period 2. A washout period of 8 weeks was maintained between the 2 treatment periods. |
| Sequence 2: First Placebo Then IVA | EXPERIMENTAL | Participants received placebo matched to IVA for 8 weeks in treatment period 1 followed by IVA 150 mg q12h for 8 weeks in treatment period 2. A washout period of 8 weeks was maintained between the 2 treatment periods. |
| Part A: 3 to <24 months | EXPERIMENTAL | Participants weighing 5 to less than (\<) 7 kilogram (kg) received 25 milligram (mg) IVA, 7 to \<14 kg received 50 mg IVA, and those weighing 14 to \<25 kg received 75 mg IVA administered every 12 hours (q12h) on Days 1 through 3 and 1 morning dose on Day 4. |
| Part B + A/B:1 to < 24 months | EXPERIMENTAL | Participants 4 to \<6 months of age and weighing greater than or equal to (≥) 5 kg received 25 mg IVA q12h. At 6 months of age and older, participants weighing 5 to \<7 kg received 25 mg IVA, 7 to \<14 kg received 50 mg IVA, and those weighing 14 to \<25 kg received 75 mg IVA q12h for 24 weeks on Part B. For Part A/B, participants 1 to \<4 months weighing 3 kg to \<5 kg received an initial low dose of 5.7 mg q12h IVA and those weighing ≥5 kg received 11.4 mg q12h IVA for the first 15 days of IVA treatment. Doses were maintained or adjusted upward at Day 15 and based on weight and/or age once they reached 4 months of age. |
| Ivacaftor (Run-in Period) | EXPERIMENTAL | Ivacaftor 150 milligram (mg) tablet orally every 12 hours for 4 weeks. |
| VX-661 + Ivacaftor (Active comparator period) | EXPERIMENTAL | VX-661 100 mg and ivacaftor 150 mg fixed-dose combination tablet orally once daily in the morning and ivacaftor 150 mg tablet orally once daily in the evening for 8 weeks. |
| Ivacaftor monotherapy (Active comparator period) | ACTIVE_COMPARATOR | Ivacaftor 150 mg tablet orally every 12 hours as monotherapy for 8 weeks. |
| Observational | NO_INTERVENTION | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet, orally, q12h in the previous Study 110 (NCT01614457) or Study 111 (NCT01614470), were observed (did not receive study drug) in this Study 112 (NCT01707290) for up to 2 years. |
| Part 1: Ivacaftor First, Then Placebo | EXPERIMENTAL | Ivacaftor 150 milligram (mg) tablet orally twice daily for 8 weeks in treatment period 1 followed by placebo matched to ivacaftor tablet orally twice daily for 8 weeks in treatment period 2. Washout out period of 4 to 8 weeks was maintained between each treatment period. |
| Part 1: Placebo First, Then Ivacaftor | EXPERIMENTAL | Placebo matched to ivacaftor tablet orally twice daily for 8 weeks in treatment period 1 followed by ivacaftor 150 mg tablet orally twice daily for 8 weeks in treatment period 2. Washout out period of 4 to 8 weeks was maintained between each treatment period. |
| Part 2: Ivacaftor | EXPERIMENTAL | Ivacaftor 150 mg tablet orally twice daily for 16 weeks. |
| Placebo | PLACEBO_COMPARATOR | Placebo matched to Ivacaftor tablet orally twice daily for 24 weeks. |
| VX-770 | EXPERIMENTAL | VX-770 (ivacaftor) 150 milligram (mg) tablet orally twice daily (q12h). |
| 150 mg Ivacaftor q12h | EXPERIMENTAL | Subjects who received 150 mg of ivacaftor q12h for up to 48 weeks. |
| Ivacaftor, Placebo, Ivacaftor, Placebo (IPIP) | EXPERIMENTAL | During the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 \[(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)\] and Cycle 2 \[(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)\] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor. |
| Ivacaftor, Placebo, Placebo, Ivacaftor (IPPI) | EXPERIMENTAL | During the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 \[(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)\] and Cycle 2 \[(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)\] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor. |
| Placebo, Ivacaftor, Ivacaftor, Placebo (PIIP) | EXPERIMENTAL | During the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 \[(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)\] and Cycle 2 \[(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)\] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor. |
| Placebo, Ivacaftor, Placebo, Ivacaftor (PIPI) | EXPERIMENTAL | During the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 \[(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)\] and Cycle 2 \[(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)\] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor. |
| Treatment Sequence 1 | EXPERIMENTAL | Ivacaftor administered in Treatment Period 1 and placebo administered in Treatment Period 2. |
| Treatment Sequence 2 | EXPERIMENTAL | Placebo administered in Treatment Period 1 and ivacaftor administered in Treatment Sequence 2. |
| Ivacaftor Group A | EXPERIMENTAL | Subjects in Part 1 who first received 25 mg or 75 mg of ivacaftor every 12 hours (q12h) for 14 days, then crossed over to receive the alternate dose for another 14 days. |
| Ivacaftor Group B | EXPERIMENTAL | Subjects in Part 1 who first received 75 mg or 150 mg of ivacaftor q12h for 14 days then crossed over to receive the alternate dose for another 14 days. |
| Ivacaftor Group C | EXPERIMENTAL | Subjects in Part 2 who received 150 mg or 250 mg of ivacaftor q12h for 28 days. |
| Part A: Pooled Placebo (Except Cohort A7) | PLACEBO_COMPARATOR | Participants without CF who received single dose of placebo matched to VX-445 in Cohort A1 to A5. |
| Part A: VX-445 (Except Cohort A7) | EXPERIMENTAL | Participants without CF who received single ascending dose of VX-445 tablet starting from 20 milligrams (mg) to 360 mg in Cohort A1 to A5. |
| Part A: VX-445 (Cohort A7) | EXPERIMENTAL | Participants without CF who received single dose of VX-445 100 mg tablet on Day 1 in fasted state and on Day 7 in fed state, followed by VX-445 20 mg intravenous (IV) injection on Day 13 in fed state in Cohort A7. |
| Part B: Pooled Placebo (Cohort B1 to B4) | PLACEBO_COMPARATOR | Participants without CF who received multiple doses of placebo matched to VX-445 once daily (qd) for 10 days in Cohort B1 to B4. |
| Part B: VX-445 (Cohort B1 to B4) | EXPERIMENTAL | Participants without CF who received VX-445 tablet qd for 10 days in Cohort B1 (60 mg), B2 (120 mg), B3 (240 mg) and B4 (340 mg). |
| Part C: Pooled Placebo (Cohort C1 to C3) | PLACEBO_COMPARATOR | Participants without CF who received placebo matched to VX-445/TEZ/IVA triple combination (TC) qd in the morning and placebo matched to IVA in the evening for 14 days. |
| Part C: VX-445/TEZ/IVA TC (Cohort C1 to C3) | EXPERIMENTAL | Participants without CF who received VX-445 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in Cohort C1; VX-445 280 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in Cohort C2 and VX-445 100 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in Cohort C3 for 14 days. |
| Part D: Placebo | PLACEBO_COMPARATOR | Participants with CF, F/MF genotype who received placebo matched to VX-445/TEZ/IVA TC qd in the morning and placebo matched to IVA qd in the evening for 4 weeks in the TC treatment period. |
| Part D: VX-445/TEZ/IVA TC - Low Dose | EXPERIMENTAL | Participants with CF, F/MF genotype who received VX-445 50 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the TC treatment period. |
| Part D: VX-445/TEZ/IVA TC - Medium Dose | EXPERIMENTAL | Participants with CF, F/MF genotype who received VX-445 100 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the TC treatment period. |
| Part D: VX-445/TEZ/IVA TC - High Dose | EXPERIMENTAL | Participants with CF, F/MF genotype who received VX-445 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the TC treatment period. |
| Part E: TEZ/IVA | ACTIVE_COMPARATOR | Following run-in period of 4 weeks with TEZ/IVA, participants with CF, F/F genotype who received TEZ 100 mg qd/IVA 150 mg q12h and placebo matched to VX-445 for 4 weeks in the TC treatment period. |
| Part E: VX-445/TEZ/IVA TC | EXPERIMENTAL | Following run-in period of 4 weeks with TEZ/IVA, participants with CF, F/F genotype who received VX-445 200 mg qd/TEZ 100 mg qd /IVA 150 mg q12h for 4 weeks in the TC treatment period. |
| Part F: Placebo | PLACEBO_COMPARATOR | Participants with CF, F/MF genotype who received placebo matched to VX-445/TEZ/VX-561 for 4 weeks in the TC treatment period. |
| Part F: VX-445/TEZ/VX-561 TC | EXPERIMENTAL | Participants with CF, F/MF genotype who received VX-445 200 mg qd/TEZ 100 mg qd/VX-561 150 mg qd for 4 weeks in the TC treatment period. |
| Cohort 1 | ACTIVE_COMPARATOR | participants in Cohort 1 will be administered ivacaftor alone followed by ivacaftor with concomitant ciprofloxacin. |
| Cohort 2 | EXPERIMENTAL | Participants in Cohort 2 will be administered VX-661 in combination with ivacaftor followed by VX-661 in combination with ivacaftor and concomitant ciprofloxacin |
| Group A | EXPERIMENTAL | approximately 12 male and female subjects with moderate hepatic impairment |
| Group B | EXPERIMENTAL | approximately 12 healthy male and female subjects |
| desipramine | EXPERIMENTAL | - |
| Midazolam | EXPERIMENTAL | - |
| Rosiglitazone | EXPERIMENTAL | - |
| Fluconazole | EXPERIMENTAL | - |
| Rifampin | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| Ivacaftor | DRUG | Subjects will be treated with ivacaftor for 6 months and followed for 7 months and will undergo assessments along the way to measure sweat chloride and sputum amounts. |
| IVA | DRUG | Granules for oral administration. |
| Placebo | DRUG | Placebo matched to IVA tablet. |
| Tezacaftor/Ivacaftor | DRUG | - |
| Ivacaftor 150 MG | DRUG | Ivacaftor is a CFTR potentiator |
| Placebo-matched-to-ivacaftor tablet | DRUG | Orally every 12 hours up to 4 weeks. |
| VX-770 | DRUG | Tablet. |
| Ivacaftor 25 mg/75 mg | DRUG | 25 mg or 75 mg q12h for a total of 28 days (Part 1) |
| Ivacaftor 75 mg/150 mg | DRUG | 75 mg or 150 mg q12h for a total of 28 days (Part 1) |
| Ivacaftor 150 mg or 250 mg | DRUG | 150 mg or 250 mg of ivacaftor q12h for 28 days (Part 2) |
| TEZ/IVA | DRUG | TEZ/IVA fixed-dose combination for oral administration. |
| VX-445 | DRUG | VX-445 tablet for oral administration. |
| Matched Placebo | DRUG | Matched placebo. |
| TEZ | DRUG | Tablet for oral administration. |
| VX-561 | DRUG | Tablet for oral administration. |
| VX-661 | DRUG | - |
| ciprofloxacin | DRUG | - |
| Rifampin | DRUG | In Period 2, subjects will receive a daily oral dose of rifampin 600 mg on Days 1 through 10 |
Inclusion Criteria: * Subjects are \>18 years of age and able to provide informed consent. * Subjects reside in the US and are willing to be treated with ivacaftor. * Subjects have the splicing mutation of interest. * Subjects are willing and able to perform requirements of the study. Exclusion Cr...
Top 6 of 7 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | Alyftrek, Trikafta, Ivacaftor, VX-581, VX-272 |
| BiomX Inc. | PHGE | 1 | PHASE2 | BX004 |
| 4D Molecular Therapeutics, Inc. | FDMT | 1 | PHASE2 | 4D-710 |
| Arcturus Therapeutics Holdings, Inc. | ARCT | 1 | PHASE2 | ARCT-032 |
| Krystal Biotech, Inc. | KRYS | 1 | PHASE1 | KB407 |
| Roivant Sciences Ltd. | ROIV | 1 | PHASE1 | SP-101 |
Ivacaftor is a small molecule studied for cystic fibrosis and chronic obstructive pulmonary disease, including chronic bronchitis. In cystic fibrosis it has been evaluated in patients with specific CFTR mutations, such as the 3849 + 10KB C to T or D1152H mutations, and in children under 24 months of age with an approved ivacaftor-responsive mutation.
Ivacaftor targets CFTR, the cystic fibrosis transmembrane conductance regulator, and is classified as a positive modulator. By acting on this protein, the drug is designed to modulate CFTR function in patients whose cystic fibrosis is driven by particular CFTR mutations. Its clinical program also includes a Phase 2 study in chronic obstructive pulmonary disease with chronic bronchitis.
Ivacaftor is developed by Vertex Pharmaceuticals Incorporated, which trades under the ticker VRTX. Vertex is the sponsor behind the clinical trial program for the drug across cystic fibrosis and chronic obstructive pulmonary disease.
Ivacaftor is an investigational drug that has been studied across Phase 1, Phase 2, and Phase 3 clinical trials. Its recorded development stage is Phase 2, and the program includes 18 trials in total, of which 17 are completed and none are currently active.
Ivacaftor trials include NCT03277196, a completed Phase 3 study of long-term treatment in cystic fibrosis patients under 24 months of age with an approved ivacaftor-responsive mutation, and NCT03085485, a completed Phase 2 study in chronic obstructive pulmonary disease and chronic bronchitis. Other completed studies include NCT03068312 in cystic fibrosis and NCT03227471.
Yes, IVA is a common abbreviation for ivacaftor. The drug is also referred to by dose-based names including ivacaftor 25 mg/75 mg and ivacaftor 150 mg. These names all refer to the same CFTR positive modulator developed by Vertex Pharmaceuticals.