Potential disease stabilization was shown
in 11 of 12 patients
Patients taking MEPSEVII had an overall positive response in the
patient-specific multi-domain responder index (MDRI) score
Scores were based on change from baseline at active treatment week 24
- -1 = Decline equal to or greater than minimally important difference (MID)
- 0 = Change less than the MID
- +1 or greater = Improvement equal to or greater than the MID
- Ability to perform physical function assessments was evaluated in 12 patients with highly variable disease
- At active treatment week 24, MDRI response showed:
- Overall positive mean change of +0.5 (±0.8 SD) (P=0.0527)
- Clinical improvement in disease manifestations, including mobility, fine motor function, and fatigue
- Some patients were too young or cognitively impaired to perform all tests
- 1 patient with -1 MDRI scores had an acute viral illness on the week 24 visit
- Positive domains outnumbered negative by 3:1
- Due to heterogeneity of disease in the study population, some assessments were hindered by cognitive and physical impairments
Indication and Usage
MEPSEVII is a lysosomal enzyme replacement therapy indicated for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome).
MEPSEVII has shown clinical improvement in various disease manifestations, including mobility, fatigue and fine motor function, in MPS VII patients based on the totality of clinical data.
Important Safety Information Hypersensitivity Reactions Including Anaphylaxis: Hypersensitivity reactions have been reported with enzyme replacement therapies including MEPSEVII, although no severe reactions have occurred when MEPSEVII was infused according to directions. Administer MEPSEVII according to the recommended infusion rate schedule. Observe patients during and after the infusion. If a severe hypersensitivity reaction occurs, immediately stop the infusion and initiate appropriate medical treatment. Pre-treatment with non-sedating antihistamines with or without antipyretics is recommended prior to the start of infusion.
Adverse Reactions Most common adverse reactions (incidence ≥10%) were infusion-associated reactions including anaphylactoid reaction, pruritus, local infusion site reactions such as infusion site extravasation and infusion site swelling, diarrhea and rash. Please see full Prescribing Information, for additional important safety information.
Important Safety Information Hypersensitivity Reactions Including Anaphylaxis: Hypersensitivity reactions have been reported with enzyme replacement therapies including MEPSEVII, although no severe reactions have occurred when MEPSEVII was infused according to directions. Administer MEPSEVII according to the recommended infusion rate schedule. Observe patients during and after the infusion. If a severe hypersensitivity reaction occurs, immediately stop the infusion and initiate appropriate medical treatment. Pre-treatment with non-sedating antihistamines with or without antipyretics is recommended prior to the start of infusion.
Adverse Reactions Most common adverse reactions (incidence ≥10%) were infusion-associated reactions including anaphylactoid reaction, pruritus, local infusion site reactions such as infusion site extravasation and infusion site swelling, diarrhea and rash. Please see full Prescribing Information, for additional important safety information.