Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
BMN 110 Weekly · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Placebo | PLACEBO_COMPARATOR | - |
| BMN 110 Weekly | EXPERIMENTAL | - |
| BMN 110 Every Other Week | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| BMN 110 Weekly | DRUG | BMN 110 Weekly: Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours once a week. |
| Placebo | DRUG | Intravenous infusion of placebo solution at a volume equivalent to that needed for 2.0 mg/kg dose of BMN 110 administered over a period of approximately 4 hours once a week. |
| BMN 110 Every Other Week | DRUG | BMN 110 Every Other Week: Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week and infusions of placebo on alternating weeks. |
Inclusion Criteria: * At least 5 years of age. * Documented clinical diagnosis of MPS IVA based on clinical signs and symptoms of MPS IVA and documented reduced fibroblast or leukocyte GALNS enzyme activity or genetic testing confirming diagnosis of MPS IVA. * Willing and able to provide written, s...
BMN 110 Weekly is an investigational small molecule being studied for the treatment of Mucopolysaccharidosis IV A (MPS IV A), also known as Morquio A syndrome. It is developed by BioMarin Pharmaceutical Inc. (BMRN) and is currently in Phase 3 clinical development for this rare disease indication.
BMN 110 Weekly is a small molecule therapy. The specific molecular target of BMN 110 Weekly is not disclosed in the available clinical trial information. The drug is being evaluated for its efficacy and safety in patients with Mucopolysaccharidosis IV A (Morquio A syndrome).
BMN 110 Weekly is developed by BioMarin Pharmaceutical Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol BMRN. BioMarin is conducting Phase 3 clinical trials to evaluate the drug for the treatment of Mucopolysaccharidosis IV A (Morquio A syndrome).
BMN 110 Weekly is in Phase 3 clinical development. It is an investigational drug and has not been approved by regulatory authorities. Two Phase 3 trials have been completed, including a double-blind, placebo-controlled study and a long-term extension study, both in patients with MPS IV A.
BMN 110 Weekly has been studied in two completed Phase 3 trials. The first, NCT01275066, was a double-blind, placebo-controlled study in 177 patients with MPS IV A. The second, NCT01415427, was a long-term extension study enrolling 173 patients. Both trials included patients aged 5 years and older across multiple countries.
BMN 110 Weekly is the investigational name for the drug also known as elosulfase alfa. It is developed by BioMarin Pharmaceutical Inc. for the treatment of Mucopolysaccharidosis IV A (Morquio A syndrome). The drug has been evaluated in Phase 3 clinical trials under the name BMN 110.