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Also known as N-acetylgalactosamine-6-sulfatase, N-acetylgalactosamine-6-sulfate sulfatase, galactose-6-sulfatase, GALNS, enzyme replacement therapy, ERT, recombinant human N-acetylgalactosamine-6-sulfatase, rhGALNS
BMN 110 Weekly · 2 trials · 4 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. |
| Placebo | PLACEBO_COMPARATOR | - |
| 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. |
| 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: * 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 developed by BioMarin Pharmaceutical Inc. for mucopolysaccharidosis IV A, also called Morquio A syndrome or MPS IV A. It is a rare disease therapy that has been studied in Phase 3 clinical trials. BioMarin trades on Nasdaq under the ticker BMRN.
BMN 110 Weekly is being developed for mucopolysaccharidosis IV A, also known as Morquio A syndrome or MPS IV A. This is a rare inherited disorder classified within the rare disease therapeutic area. The clinical program enrolled patients aged 5 years and older, with both males and females eligible for the studies.
BMN 110 Weekly is developed by BioMarin Pharmaceutical Inc., a biopharmaceutical company that trades on Nasdaq under the ticker BMRN. BioMarin sponsored the Phase 3 clinical program evaluating the small molecule in patients with mucopolysaccharidosis IV A, also called Morquio A syndrome.
BMN 110 Weekly has been evaluated in Phase 3 clinical development. Two Phase 3 trials are recorded, both listed as completed, including a double-blind, placebo-controlled efficacy and safety study and a long-term extension study. The program is not currently listed with active trials.
BMN 110 Weekly was studied in two completed Phase 3 trials. NCT01275066 was a double-blind, placebo-controlled study of efficacy and safety in MPS IV A with 177 patients. NCT01415427 was a long-term efficacy and safety extension study in Morquio A syndrome with 173 patients. Both trials enrolled participants aged 5 years and older across numerous countries.
Yes, BMN 110 Weekly, BMN 110 - Weekly, and BMN 110- Weekly refer to the same investigational therapy from BioMarin Pharmaceutical Inc. The alternate spellings appear in trial and program records, but they describe the same small molecule candidate being studied in mucopolysaccharidosis IV A, also known as Morquio A syndrome.