MEPSEVII® (vestronidase alfa-vjbk) demonstrated improvement or stabilization across multiple measures1
Mobility
Patients treated with MEPSEVII had improved or stabilized mobility1
Respiratory
MEPSEVII demonstrated improvement in pulmonary function after 120 weeks of treatment1
Improvement in pulmonary function from baseline
21% improvement in FVC (% predicted) in 1 patient1
Study 201 - Ex-US investigation (120 weeks): A single-arm, open-label, dose-exploration study outside the United States included 3 patients with MPS VII aged 5 to 25 years. FVC = forced vital capacity.
Mobility in this patient also improved1
- 105-meter improvement in the 6MWT in the same patient
Small heterogeneous patient group1
- Due to the extremely small population of patients with MPS VII, the patient group was heterogeneous
- Clinical end points in some patients were not assessable due to the extent of disease, age, or level of cognition
Study 301 - Randomized Start trial (Baseline to 48 weeks): The efficacy study was a randomized-start, phase 3 clinical study in which 12 patients (4 males, 8 females) aged 8 to 25 years (median age: 14 years) were assessed to evaluate the efficacy and safety of recombinant human ß-glucuronidase (rhGUS) enzyme replacement therapy (ERT) in patients with mucopolysaccharidosis VII (MPS VII).1,2
Study 203 - Open-label, single arm (48 weeks): An open-label, uncontrolled single arm study in which 8 patients below 5 years of age received MEPSEVII at 4 mg/kg every 2 weeks for 48 weeks with an optional 240 weeks of additional dose continuation period.1
Study 201 - Ex-US investigation (120 weeks): A single-arm, open-label, dose-exploration study outside the United States included 3 patients with MPS VII aged 5 to 25 years.1
Small heterogeneous patient group1
- Due to the extremely small population of patients with MPS VII, the patient group was heterogeneous
References: 1. MEPSEVII [package insert]. Novato, CA: Ultragenyx Pharmaceutical Inc; 2020. 2. Data on file. Clinical Study Report UX003-CL301. Ultragenyx Pharmaceutical Inc; January 9, 2017.