In a 24-week clinical trial, people who took VIMIZIM improved their endurance as measured by the 6-minute walk test (6MWT)1,2
MOR-004: a phase 3, double-blind, randomized, placebo-controlled trial (N=176)2,3
MOR-004: a phase 3, double-blind, randomized, placebo-controlled trial (N=176)2,3
Treatment with VIMIZIM® (elosulfase alfa) 2 mg/kg/wk demonstrated:
- Improvement in 6MWT was seen as early as 12 weeks2
- 22.5-meter improvement in 6MWT over placebo at 24 weeks (P=0.0174)1,2
The 6MWT is a validated measure of endurance in the clinical setting that evaluates the functional reserves of the cardiovascular, pulmonary, or musculoskeletal systems.5
Learn about the overall pivotal phase 3 study design.
VIMIZIM demonstrated positive results across many efficacy measures at 24 weeks6
Although statistical significance was only observed in the 6MWT results, improvement was seen in the majority of exploratory efficacy endpoints. The sample size for the study was selected to be sufficient to detect the impact of treatment on only the primary and secondary endpoints. Tertiary endpoints were intended to be exploratory in nature.2
FET, forced expiratory time; FEV1, forced expiratory volume in 1 second; FIVC, forced inspiratory vital capacity; FVC, forced vital capacity; MPS HAQ, Mucopolysaccharidoses Health Assessment Questionnaire; MVV, maximum voluntary ventilation; 3MSCT, 3-minute stair-climbing test; Z-score, how far a measurement deviates from the average.
Adverse reactions
- In clinical trials, the most common adverse reactions (≥10%) occurring during infusion included pyrexia, vomiting, headache, nausea, abdominal pain, chills, and fatigue
- Serious and severe reactions were associated with VIMIZIM, including hypersensitivity reactions as well as life-threatening allergic reactions (anaphylaxis)
- In the clinical trials, anaphylaxis occurred during VIMIZIM infusions and up to 3 hours after the infusion
References: 1. VIMIZIM [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2019. 2. Hendriksz CJ, Burton B, Fleming TR, et al. Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study. J Inherit Metab Dis. 2014;37(6):979-990. 3. Hendriksz CJ, Parini R, AlSayed MD, et al. Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome. Mol Genet Metab. 2016;119(1-2):131-143. 4. Data on file. BioMarin Pharmaceutical, Inc. 5. Harmatz P, Mengel KE, Giugliani R, et al. The Morquio A Clinical Assessment Program: baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects. Mol Genet Metab. 2013;109(1):54-61. 6. Hendriksz CJ, Giugliani R, Harmatz P, et al. Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial. Mol Genet Metab. 2015;114(2):178-185.