Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as recombinant human N-acetylgalactosamine-6-sulfatase, N-acetylgalactosamine-6-sulfatase, N-acetylgalactosamine-6-sulfate, sulfatase, galactose-6-sulfatase, GALNS, enzyme replacement therapy, ERT
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 being developed for mucopolysaccharidosis IVA, also known as Morquio A syndrome or MPS IVA. This is a rare inherited lysosomal storage disorder. BioMarin Pharmaceutical Inc. is developing the small molecule therapy, and clinical studies have enrolled patients with MPS IVA in the United States, United Kingdom, Italy, Taiwan, and Australia.
BMN 110 is developed by BioMarin Pharmaceutical Inc., which trades on Nasdaq under the ticker BMRN. The company has sponsored the clinical studies of BMN 110 in patients with mucopolysaccharidosis IVA, including trials conducted in Australia, the United States, Italy, Taiwan, and the United Kingdom.
BMN 110 has been studied in Phase 1, Phase 2, and Phase 3 clinical trials. The Phase 1 study NCT00884949 evaluated safety, tolerability, and efficacy in subjects with MPS IVA. A Phase 2 study and a Phase 3B study in mucopolysaccharidosis IVA have also been completed.
BMN 110 has been evaluated in completed clinical trials including NCT00884949, a Phase 1 study of safety, tolerability, and efficacy in MPS IVA; NCT01515956, a Phase 2 study in pediatric patients under 5 years of age with Morquio A syndrome; and NCT01966029, a Phase 3B study in Australian patients with mucopolysaccharidosis IVA.
Across the completed clinical studies of BMN 110, 28 patients have been enrolled. The Phase 1 study NCT00884949 enrolled 20 subjects, the Phase 2 pediatric study NCT01515956 enrolled 15 patients under 5 years of age, and the Phase 3B study NCT01966029 enrolled 13 patients in Australia.
BMN 110 is an investigational therapy that has been studied in completed Phase 1, Phase 2, and Phase 3 clinical trials for mucopolysaccharidosis IVA. BioMarin Pharmaceutical Inc. is the developer. The clinical program includes studies in pediatric patients under 5 years of age and in Australian patients with Morquio A syndrome.