Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
rAvPAL-PEG · 4 trials · 1 indication
Plasma Phe
Blood Phe Concentration (Change from Baseline) and Daily Dose in PAL-003 Subjects by Disposition (PAL-003 Population)
Specifically, safety was to be assessed by examining the incidence of all treatment-emergent adverse events reported during the study period and clinically significant changes in vital signs and clinical laboratory results, including development of rAvPAL-PEG antibodies.
| Arm | Type | Description |
|---|---|---|
| rAvPAL-PEG | EXPERIMENTAL | rAvPAL-PEG in varying doses |
| rAvPAL-PEG 0.001 mg/kg | EXPERIMENTAL | Subjects will start on rAvPAL-PEG 0.001 mg/kg |
| rAvPAL-PEG 0.003 mg/kg | EXPERIMENTAL | Subjects will start on rAvPAL-PEG 0.003 mg/kg |
| rAvPAL-PEG 0.01 mg/kg | EXPERIMENTAL | Subjects will start on rAvPAL-PEG 0.01 mg/kg |
| rAvPAL-PEG 0.03 mg/kg | EXPERIMENTAL | Subjects will start on rAvPAL-PEG 0.03 mg/kg |
| rAvPAL-PEG 0.1 mg/kg | EXPERIMENTAL | Subjects will start on rAvPAL-PEG 0.1 mg/kg |
| 0.001 mg/kg | ACTIVE_COMPARATOR | One subcutaneous injection of 0.001 mg/kg of rAvPAL-PEG. |
| 0.003 mg/kg | ACTIVE_COMPARATOR | One subcutaneous injection of 0.003 mg/kg of rAvPAL-PEG. |
| 0.01 mg/kg | ACTIVE_COMPARATOR | One subcutaneous injection of 0.01 mg/kg of rAvPAL-PEG. |
| 0.03 mg/kg | ACTIVE_COMPARATOR | One subcutaneous injection of 0.03 mg/kg of rAvPAL-PEG. |
| 0.1 mg/kg | ACTIVE_COMPARATOR | One subcutaneous injection of 0.1 mg/kg of rAvPAL-PEG. |
| 0.3 mg/kg | ACTIVE_COMPARATOR | One subcutaneous injection of 0.3 mg/kg of rAvPAL-PEG. |
| 1.0 mg/kg | ACTIVE_COMPARATOR | One subcutaneous injection of 1.0 mg/kg of rAvPAL-PEG. |
| Name | Type | Description |
|---|---|---|
| rAvPAL-PEG | DRUG | 0.06 mg/kg/day, 0.1 mg/kg/day, 0.2 mg/kg/day, 0.4 mg/kg/day, 0.6 mg/kg/day, 0.8 mg/kg/day |
| rAvPAL-PEG 0.001 mg/kg | DRUG | In Part 1, the planned starting dose levels is 0.001 mg/kg |
| rAvPAL-PEG 0.003 mg/kg | DRUG | In Part 1, the planned starting dose levels is 0.003 mg/kg |
| rAvPAL-PEG 0.01 mg/kg | DRUG | In Part 1, the planned starting dose levels is 0.01 mg/kg |
| rAvPAL-PEG 0.03 mg/kg | DRUG | In Part 1, the planned starting dose levels is 0.03 mg/kg |
| rAvPAL-PEG 0.1 mg/kg | DRUG | In Part 1, the planned starting dose levels is 0.1 mg/kg |
Inclusion Criteria: * Diagnosis of PKU with both of the following: current blood Phe concentration of ≥ 600 micromol/L at screening and average blood Phe concentration of ≥ 600 micromol/L over the past 3 years, using available data * Evidence that the subject is a non-responder to Kuvan® treatment ...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| BioMarin Pharmaceutical Inc. | BMRN | 5 | PHASE3 | Pegvaliase |
| PTC Therapeutics, Inc. | PTCT | 2 | PHASE3 | PTC923 |
| Sanofi SA Sponsored ADR | SNY | 1 | PHASE1 | SAR444836 |
| Agios Pharmaceuticals, Inc. | AGIO | 1 | PHASE1 | AG-181 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
| Q32 Bio Inc | QTTB | 1 | - | HMI-102 |
rAvPAL-PEG is an investigational therapy being developed for phenylketonuria (PKU), a metabolic disorder. It is a small molecule designed to address the condition in patients aged 16 years and older. The drug is currently in clinical development and has not been approved by regulatory authorities.
rAvPAL-PEG is being developed by BioMarin Pharmaceutical Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol BMRN. BioMarin has conducted multiple clinical trials to evaluate the safety and efficacy of rAvPAL-PEG in patients with phenylketonuria.
rAvPAL-PEG is in Phase 2 clinical development. It has completed four clinical trials, including one Phase 1 study and three Phase 2 studies, all focused on phenylketonuria. The drug remains investigational and is not yet approved for commercial use.
rAvPAL-PEG has been studied in four completed clinical trials in the United States. These include NCT00634660, a Phase 1 safety and tolerability study; NCT00924703, a long-term extension study; NCT00925054, a dose-finding study; and NCT01212744, a daily dosing study. All trials enrolled patients aged 16 years and older with phenylketonuria.
rAvPAL-PEG is not FDA approved. It is an investigational drug that has completed Phase 1 and Phase 2 clinical trials for phenylketonuria. The drug is still in clinical development, and its safety and efficacy have not been fully established for regulatory approval.
rAvPAL-PEG has completed four clinical trials, including one Phase 1 and three Phase 2 studies, all in patients with phenylketonuria. The trials were conducted in the United States with a total enrollment of 149 participants. The drug is not currently in active clinical trials and remains investigational.