Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
BMN 110 · 3 trials · 5 indications
The analyses of safety will include all patients who receive any study drug. All safety data will be summarized descriptively. All AEs will be coded using the Medical Dictionary for Regulatory Activities (MedDRA). The incidence of AEs will be summarized by System Organ Class (SOC), Preferred Term (PT), relationship to study drug, and severity. Patient listings will be provided for SAEs, deaths, and AEs leading to study drug discontinuation, or study withdrawals. All AE summaries will include only treatment-emergent AEs (TEAEs) reported during the study period (AEs that occur after the first study drug infusion). Infusion-associated AEs will be summarized by SOC, PT and severity. The following safety measures will be summarized descriptively: concomitant medications, clinical laboratory tests, vital signs, ECGs, immunogenicity results, analgesic medication use, results from routine physical examinations (including standard neurologic examinations), and cervical spine imaging.
Number of Participants Experiencing Adverse Events
The primary objective of the study was to evaluate the safety of weekly infusions of BMN 110 administered in escalating doses to subjects with MPS IVA. The safety variable incidence of TEAE is summarized.
| Arm | Type | Description |
|---|---|---|
| Treatment | EXPERIMENTAL | All patients receive treatment with BMN 110 |
| BMN 110 Weekly | EXPERIMENTAL | - |
| BMN 110 | EXPERIMENTAL | Within-patient Dose-Escalation |
| Name | Type | Description |
|---|---|---|
| BMN 110 | DRUG | All pateints receive treatment with BMN 110 |
Inclusion Criteria: 1. Diagnosed with MPS IVA as confirmed by a documented GALNS enzymatic test (GALNS activity in affected range, beta-galactosidase and a second lysosomal sulfatase activity within normal range). 2. Age 12 months or older. 3. Willing and able to provide written, signed informed co...
BMN 110 is used for the treatment of Mucopolysaccharidosis IVA, also known as Morquio A Syndrome. This is a rare genetic disorder. The drug is being developed as a potential therapy for this condition.
BMN 110 is being developed by BioMarin Pharmaceutical Inc., a biopharmaceutical company. The company's stock ticker is BMRN. BioMarin is focused on developing therapies for rare diseases.
BMN 110 is in clinical development. It has completed trials in Phase 1, Phase 2, and Phase 3. However, it is not yet approved and remains an investigational drug. The most advanced completed trial was a Phase 3 study.
BMN 110 has been studied in several clinical trials. NCT00884949 was a Phase 1 study in the United Kingdom. NCT01515956 was a Phase 2 study in pediatric patients. NCT01966029 was a Phase 3 study in Australia. All trials have been completed.
BMN 110 is also known as elosulfase alfa. It is a drug being developed by BioMarin Pharmaceutical Inc. for the treatment of Mucopolysaccharidosis IVA, also called Morquio A Syndrome.