Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
BMN 701 · 1 trial · 1 indication
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
| Arm | Type | Description |
|---|---|---|
| BMN 701 | EXPERIMENTAL | IV infusion |
| Name | Type | Description |
|---|---|---|
| BMN 701 | BIOLOGICAL | GILT-tagged recombinant human GAA |
Inclusion criteria: * Patient has been diagnosed with Pompe Disease prior to or during the screening period based on 2 GAA gene mutations and either: endogenous GAA activity \<75% of the lower limit of the normal adult range reported by the testing laboratory, as assessed in cultured skin fibroblas...
BMN 701 is an investigational monoclonal antibody being developed for the treatment of Pompe disease, a rare genetic disorder. It is intended for patients with late-onset Pompe disease. The drug is currently in clinical development and has not been approved by regulatory authorities.
BMN 701 is being developed by BioMarin Pharmaceutical Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol BMRN. The company is focused on developing therapies for rare diseases, and BMN 701 is one of its investigational candidates for Pompe disease.
BMN 701 is in Phase 1 clinical development. A Phase 1 study has been completed to evaluate its safety, tolerability, pharmacokinetics, and pharmacodynamics in patients with late-onset Pompe disease. The drug remains investigational and is not yet approved for commercial use.
BMN 701 has been studied in one clinical trial, identified as NCT01230801. This was a Phase 1 study titled 'Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease.' The trial enrolled 22 participants and has been completed.
BMN 701 is a monoclonal antibody designed to target and treat Pompe disease. While the specific molecular target is not disclosed, the drug is intended to address the underlying enzyme deficiency characteristic of the condition. It is being studied for its ability to improve outcomes in patients with late-onset Pompe disease.