Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Liprotamase Powder for Oral Solution, ALTU-135, LY3031642, TheraCLEC-total
Liprotamase · 5 trials · 3 indications
Number of subjects reporting 1 or more adverse events
The primary endpoint evaluates the difference between treatment arms in change from baseline in coefficient of fat absorption (CFA). As such, descriptive statistics for individual treatment arms are not provided in this measure, but are reported in the secondary endpoints
| Arm | Type | Description |
|---|---|---|
| Part A | EXPERIMENTAL | Liprotamase Powder for Oral Solution in Subjects aged ≥7 years of age |
| Part B | EXPERIMENTAL | Liprotamase Powder for Oral Solution in Subjects aged 28 days to \<7 years |
| Liprotamase | EXPERIMENTAL | Individually-optimized dose to be administered orally |
| porcine (pig) PERT | ACTIVE_COMPARATOR | Individually-optimized dose to be administered orally |
| Placebo | PLACEBO_COMPARATOR | Double Blind Treatment Period: Placebo (microcrystalline cellulose) administered orally with each of three meals and two snacks daily for 6 days. |
| Low Liprotamase Dose | EXPERIMENTAL | Liprotamase in a fixed combination of lipase (5,000 units), protease (5,000 units) and amylase (750 units) administered orally (one Size 5 capsule of liprotamase and five Size 2 capsules of placebo) with each of three meals and two snacks daily for 28 days |
| Mid Liprotamase Dose | EXPERIMENTAL | Liprotamase in a fixed combination of lipase (25,000 units), protease (25,000 units) and amylase (3,750 units) administered orally (one Size 5 capsule of liprotamase, one Size 2 capsule of liprotamase, and four Size 2 capsules of placebo) with each of three meals and two snacks daily for 28 days |
| High Liprotamase Dose | EXPERIMENTAL | Liprotamase in a fixed combination of lipase (100,000 units), protease (100,000 units) and amylase (15,000 units) administered orally (one Size 5 capsule of placebo and five Size 2 capsules of liprotamase) with each of three meals and two snacks daily for 28 days |
| Name | Type | Description |
|---|---|---|
| Liprotamase Powder for Oral Solution | DRUG | Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement therapy |
| Liprotamase | DRUG | oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement |
| porcine (pig) PERT | DRUG | oral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source |
| Placebo | DRUG | Administered orally |
Inclusion Criteria: * For Part A: males or females ≥7 years of age * For Part B: males or females 28 days to \<7 years * Diagnosis of cystic fibrosis based on presentation, genotype and/or sweat chloride * Low fecal elastase * Fair-to-good nutritional status Exclusion Criteria: * History or diagn...
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 |
| Roivant Sciences Ltd. | ROIV | 1 | PHASE2 | SP-101 |
| Krystal Biotech, Inc. | KRYS | 1 | PHASE1 | KB407 |