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BMN 110- Weekly · 1 trial · 3 indications
Efficacy was assessed by changes from baseline in 6-minute walk test
Efficacy was assessed by changes from baseline in 6-minute walk test
| Arm | Type | Description |
|---|---|---|
| BMN 110 Weekly | EXPERIMENTAL | BMN 110 Weekly: In Part 1, patients will receive an intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours once a week. |
| BMN 110 Every Other Week | EXPERIMENTAL | BMN 110 Every Other Week: In Part 1, patients will receive an 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 will receive infusions of placebo on alternating weeks. |
| Name | Type | Description |
|---|---|---|
| BMN 110 - Weekly | DRUG | In Part 1, patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg/qw administered over a period of approximately 4 hours once a week. In Part 2, patients will continue to receive 2.0 mg/kg of BMN 110 every week, with no placebo. |
| BMN 110 - Every Other Week | DRUG | In Part 1, patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week. Patients randomized to the 2.0 mg/kg/qow arm will receive infusions of placebo on alternating weeks, to mask active drug weeks. In Part 2, patients will receive 2.0 mg/kg of BMN 110 every week, with no placebo. |
Inclusion Criteria: * Must have completed MOR-004 * Is willing and able to provide written, signed informed consent. Or in the case of patients under the age of 18 (or other age as defined by regional law or regulation), provide written assent (if required) and have written informed consent, signed...
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.