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Label indication ORKAMBI is indicated for the treatment of cystic fibrosis (CF) in patients aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene. If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of the F508del mutation on both alleles of the CFTR gene. FDA label
Also known as LUM, Orkambi
LUM/IVA · 7 trials · 1 indication
MRI scans assessed semi-quantitatively via a standardized chest MRI scoring system. Each participant had 6 lobes scored using 7 scoring parameters:1) Bronchiectasis/wall thickening 2) Mucus plugging 3) Abscesses/sacculations 4) Consolidations 5) Special findings 6)Mosaic pattern 7) Perfusion abnormalities. For each of 7 parameter, there were scores of 6 lobes (score of each lobe : 0= normal value, 1 = \<50% of lobe involved and 2 = \>=50% of lobe involved). MRI global score was calculated as sum of parameters 1 to 7. MRI total score is ranged from 0-84. Higher score indicate more lobe involvement.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
| Arm | Type | Description |
|---|---|---|
| LUM/IVA | EXPERIMENTAL | Participants weighing 7 to less than (\<) 9 kilograms (kg) received LUM 75 milligrams (mg)/IVA 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 9 to \<14 kg received LUM 100 mg/IVA 125 mg q12h in the treatment period of 96 weeks. Participants weighing greater than or equal to (\>=)14 kg received LUM 150 mg/IVA 188 mg FDC q12h in the treatment period of 96 weeks. |
| Part A: LUM/IVA | EXPERIMENTAL | Participants weighing 7 to less than (\<)10 kilograms (kg) at screening received LUM 75 milligrams (mg)/IVA 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 10 to \<14 kg at screening received LUM 100 mg/IVA 125 mg q12h for 15 days. Participants weighing greater than or equal to (\>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 15 days. |
| Part B: LUM/IVA | EXPERIMENTAL | Participants weighing 7 to \<9 kg at screening received LUM 75 mg/IVA 94 mg FDC q12h and those weighing 9 to \<14 kg received LUM 100 mg/IVA 125 mg q12h for 24 weeks. Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 24 weeks. Doses were adjusted upwards for changes in weight. |
| Lumacaftor/Ivacaftor (LUM/IVA) | EXPERIMENTAL | Part A (\<14 kg): Participants weighing less than (\<) 14 kilograms (kg) at screening received LUM 100 milligram (mg)/IVA 125 mg fixed-dose combination every 12 hours for 15 days in Part A. Part A (\>=14 kg): Participants weighing greater than or equal to (\>=) 14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 15 days in Part A. Part B (\<14 kg): Participants weighing \<14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B. Part B (\>=14 kg): Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B. |
| Treatment Period 1: LUM/IVA to LUM/IVA | EXPERIMENTAL | - |
| Treatment Period 1: Placebo (PBO) to LUM/IVA | EXPERIMENTAL | - |
| Treatment Period 1: Observational Cohort | NO_INTERVENTION | - |
| Treatment Period 2: LUM/IVA | EXPERIMENTAL | - |
| Part 1: Placebo | PLACEBO_COMPARATOR | Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks. |
| Part 1: LUM/IVA | EXPERIMENTAL | Participants weighing less than (\<)14 kilograms (kg) at screening received LUM 100 milligrams (mg)/IVA 125 mg fixed-dose combination (FDC) every 12 hours (q12h) in placebo-controlled period for 48 weeks. Participants weighing greater than or equals to (\>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks. |
| Part 2: Overall LUM/IVA | EXPERIMENTAL | Participants who received either placebo or LUM/IVA in placebo-controlled period administered LUM/IVA (either LUM 100 mg/IVA 125 mg FDC q12h or LUM 150 mg/IVA 188 mg FDC q12h as per their body weight for participants \<6 years of age at week 48 and LUM 200 mg/IVA 250 mg FDC q12h regardless of their body weight for participants \>=6 years of age at week 48) in open-label period for 48 weeks. |
| Treatment Sequence 1 | EXPERIMENTAL | LUM/IVA in Treatment Period 1; washout; placebo in Treatment Period 2 |
| Treatment Sequence 2 | EXPERIMENTAL | Placebo in Treatment Period 1; washout; LUM/IVA in Treatment Period 2 |
| Name | Type | Description |
|---|---|---|
| LUM/IVA | DRUG | LUM/IVA granules for oral administration |
| LUM | DRUG | Fixed Dose Combination (FDC) granules (LUM/IVA). |
| IVA | DRUG | FDC granules (LUM/IVA). |
| Placebo | DRUG | Placebo matched to LUM/IVA for oral administration. |
Key Inclusion Criteria: * Participants From Study VX16-809-122 Part B (Study 122) * Completed the 24-week Treatment Period and the Safety Follow-up Visit in Study 122B * Participants Not From Study 122 * Subjects will be 1 to less than 2 years of age * Homozygous for the F508del mutation (F...
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 |
LUM/IVA is used to treat cystic fibrosis in people who are homozygous for the F508del mutation in the CFTR gene. It is a small molecule combination therapy developed for this biomarker-selected patient population. The clinical program has studied it in patients from 12 months of age and older.
LUM/IVA is developed by Vertex Pharmaceuticals Incorporated, which trades on the Nasdaq under the ticker VRTX. Vertex is the sponsor of the clinical trials evaluating the combination in cystic fibrosis.
LUM/IVA has been studied in Phase 3 clinical development for cystic fibrosis. Seven trials are registered in the program, all of which are completed, including Phase 3 studies in infants and young children. No trials are currently listed as active.
The LUM/IVA program includes completed trials such as NCT04235140, a Phase 3 long-term safety study in participants 12 to under 24 months of age, NCT03601637, a Phase 3 safety and pharmacokinetic study in participants 1 to under 2 years, and NCT03125395, a Phase 3 rollover safety study in subjects aged 2 years and older.
Yes, LUM is an alternative name for LUM/IVA. Both refer to the same lumacaftor and ivacaftor combination product developed by Vertex Pharmaceuticals for cystic fibrosis.
The registered LUM/IVA clinical program has enrolled a total of 549 participants across seven completed trials. These studies were randomized, double-blind, and controlled, and enrolled patients with cystic fibrosis who are homozygous for the F508del mutation.