近日，美国FDA批准Ultragenyx Pharmaceutical的新药Mepsevii（vestronidase alfa-vjbk）上市，治疗VII型粘多糖贮积症（MPS VII）。这也是首款获美国FDA批准的新药，用于治疗儿童和成人MPS VII患者的创新疗法。
Mepsevii (Vestronidase Alfa-Vjbk Injection, for Intravenous Use)
MEPSEVII Rx
Generic Name and Formulations:
Vestronidase alfa-vjbk 2mg/mL; soln for IV infusion after dilution; preservative-free.
Company:
Ultragenyx Pharmaceutical Inc.
Indications for MEPSEVII:
Treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome).
Limitations Of use:
Effect on the CNS manifestations of MPS VII has not been determined.
Adults and Children:
Premedicate with a non-sedating antihistamine with or without an antipyretic 30–60mins prior to infusion. Give as IV infusion over 4hrs (infuse first 2.5% of total volume over 1hr, then increase rate as tolerated over next 3hrs); see full labeling. 4mg/kg every 2 weeks.
Warnings/Precautions:
Have appropriate medical support readily available. Should be administered under supervision of healthcare professional. Monitor during and for ≥60mins post-infusion for anaphylaxis; discontinue immediately if a severe systemic reaction occurs. Elderly. Pregnancy. Nursing mothers.
Pharmacological Class:
Recombinant human lysosomal beta glucuronidase.
Adverse Reactions:
Infusion site extravasation, diarrhea, rash, anaphylaxis, infusion site swelling, peripheral swelling, pruritus.
Generic Availability:
NO
How Supplied:
Single-dose vial (5mL)—1

Mepsevii Approved to Treat Rare Genetic Disease
The Food and Drug Administration (FDA) has approved Mepsevii (vestronidase alfa-vjbk) for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome) in pediatric and adult patients. This enzyme replacement therapy is intended to replace the deficient lysosomal enzyme beta-glucuronidase in MPS VII patients.
The approval of Mepsevii was based on a clinical trial program that included 23 patients with MPS VII; the patients ranged in age from 5 months to 25 years. Patients received Mepsevii at doses up to 4mg/kg once every two weeks for up to 164 weeks. Efficacy was primarily assessed through the 6-minute walk test (6MWT) in 10 patients who could perform the test.
After 24 weeks, the mean difference in distance walked relative to placebo was 18 meters. Additional follow-up for up to 120 weeks suggested continued improvement in 3 patients and stabilization in the remaining 7 patients. After 120 weeks of exposure, 1 patient demonstrated a 21% improvement over baseline in forced vital capacity (FVC% predicted) on pulmonary function testing in addition to a 105 meter improvement in the 6MWT. Two other patients with baseline hepatosplenomegaly had reduction in liver volume (24% and 53%) and spleen volume (28% and 47%) after 36 weeks of Mepsevii treatment. The effect of Mepsevii on the central nervous system manifestations of MPS VII has not been determined
The most common side of effects of treatment include infusion site reactions, diarrhea, rash, and anaphylaxis.
Mepsevii is supplied as a carton containing one 10mg/5mL single-dose vial. It is expected to be available later this month.