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The Long-Term Impact of NAGLAZYME® (galsulfase) on MPS VI

Regardless of phenotype or age, NAGLAZYME demonstrated consistent and sustained improvement in a 10‐year Resurvey Study

The Resurvey Study was performed to examine the long‐term impact of NAGLAZYME® (galsulfase) on disease progression. This 10‐year follow-up obtained data on medical histories and clinical assessments (n=59) of NAGLAZYME‐treated and untreated patients who participated in an original Survey Study establishing demographics and clinical progression of patients with MPS VI. Throughout the study period, NAGLAZYME‐treated patients received therapy for an average of 6.8 years. The findings in the Resurvey Study were consistent with the results of clinical trials of long‐term use of NAGLAZYME in the treatment of MPS VI.1

NAGLAZYME therapy resulted in improved endurance across all age groups1

Improvements in endurance in patients who completed the 6-minute walk test (6MWT) at follow-up1

Endurance graph
  • Among 54 NAGLAZYME-treated patients, 46 were able to complete the 6MWT; they walked 21% farther than at baseline, a mean improvement of 65.7 m1
  • The subgroup of adult patients (≥18 years old) showed a sustained 22% improvement in endurance1
  • Results from the Resurvey Study are consistent with improvement demonstrated in the phase 3 trial1,2

As exemplified by these clinical trial data, the walk test has emerged as a proven, trusted, and relatively simple method of measuring improved endurance in patients with MPS VI. The test demonstrates how performance impairment is a measure of disease progression.3

The mean 6MWT distance for the full NAGLAZYME enzyme replacement therapy (ERT) group (n=54) changed from 304.0 ± 108.4 m at baseline to 320.4 ± 195.7 m at 10 years, an increase of 16.4 ± 155.9 m.1

Regardless of age, NAGLAZYME improved pulmonary function at the 10‐year follow‐up1

Pulmonary function across all age groups in the NAGLAZYME-treated patients1

Pulmonary function graph

The mean change in forced vital capacity (FVC) from baseline was 0.37 L (P<0.0001) in the NAGLAZYME‐treated group (n=48) and −0.70 L in the untreated group (n=3). The mean change in forced expiratory volume in 1 second (FEV1) from baseline was 0.21 L (P=0.001) in both the treated <13‐year‐old and ≥13‐year‐old age group patients (n=47) and −0.60 L in the untreated group (n=3).1

Across ages and phenotypes, uGAG levels normalized with NAGLAZYME1

Urinary GAG levels in all NAGLAZYME‐treated patients1

Urinary GAG levels graph

auGAG levels >200 μg/mg are associated with rapidly progressing disease; uGAG levels ≤200 μg/mg are associated with slowly progressing disease.4

  • uGAG levels decreased 87.9% from a mean of 321.34 ± 199.86 µg/mg creatinine at baseline to levels below 100 µg/mg1
  • 100% of the patients (n=55) treated with NAGLAZYME had uGAG levels <100 µg/mg creatinine at 10-year follow-up1
    • This includes 33 patients who presented with the rapidly progressing phenotype at baseline1
    • Results from the Resurvey Study are consistent with the decrease in uGAG levels seen in the phase 3 trial1,5

Regardless of phenotype, NAGLAZYME slows disease progression

Slowly progressing patients benefit from NAGLAZYME1

Resurvey Study endurance results (6MWT) of patients with uGAG levels ≤200 μg/mg1

  • MPS VI patients with uGAG levels <100 µg/mg showed a 19% improvement in endurance1
  • Patients with uGAG levels of 100-200 µg/mg showed an 11% improvement in endurance1

NAGLAZYME slowed disease progression in patients with the rapidly progressing phenotype

  • 6MWT and pulmonary function remained stable in patients with baseline uGAG levels >200 µg/mg1

Learn more about the role compliance plays in MPS VI management »


  1. Giugliani R, Lampe C, Guffon N, et al. Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux–Lamy syndrome)—10-year follow-up of patients who previously participated in an MPS VI Survey Study. Am J Med Genet A. 2014;164A(8):1953-1964. doi:10.1002/ajmg.a.36584.
  2. Harmatz P, Giugliani R, Schwartz I, et al; MPS VI Phase 3 Study Group. Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study. J Pediatr. 2006;148(4):533-539. doi:10.1016/j.jpeds.2005.12.014.
  3. McDonald A, Steiner R, Kuehl K, Turbeville S. Clinical utility of endurance measures for evaluation of treatment in patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). J Pediatr Rehabil Med. 2010;3(2):119-127. doi:10.3233/PRM-2020-0114.
  4. Swiedler SJ, Beck M, Bajbouj M, et al. Threshold effect of urinary glycosaminoglycans and the walk test as indicators of disease progression in a survey of subjects with mucopolysaccharidosis VI (Maroteaux–Lamy syndrome). Am J Med Genet A. 2005;134A(2):144-150. doi:10.1002/ajmg.a.30579.
  5. Harmatz P, Giugliani R, Schwartz IVD, et al; MPS VI Study Group. Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase. Mol Genet Metab. 2008;94(4):469-475. doi:10.1016/j.ymgme.2008.04.001.


Life-threatening anaphylactic reactions and severe allergic reactions have been observed in some patients during NAGLAZYME infusions and up to 24 hours after infusion. If these reactions occur, immediate discontinuation of NAGLAZYME is recommended and appropriate medical treatment should be initiated, which may include resuscitation, epinephrine, administering additional antihistamines, antipyretics or corticosteroids. In patients who have experienced anaphylaxis or other severe allergic reactions during infusion with NAGLAZYME, caution should be exercised upon rechallenge; appropriately trained personnel and equipment for emergency resuscitation (including epinephrine) should be available during infusions.

As with other enzyme replacement therapies, immune-mediated reactions, including membranous glomerulonephritis have been observed. In clinical trials, nearly all patients developed antibodies as a result of treatment with NAGLAZYME; however, the analysis revealed no consistent predictive relationship between total antibody titer, neutralizing or IgE antibodies, and infusion-associated reactions, urinary glycosaminoglycan (GAG) levels, or endurance measures.

Caution should be exercised when administering NAGLAZYME to patients susceptible to fluid volume overload because congestive heart failure may result. Consider a decreased total infusion volume and infusion rate when administering NAGLAZYME to these patients.

Consideration to delay NAGLAZYME infusion should be given when treating patients who present with an acute febrile or respiratory illness. Sleep apnea is common in MPS VI patients and antihistamine pretreatment may increase the risk of apneic episodes. Evaluation of airway patency should be considered prior to the initiation of treatment. Patients using supplemental oxygen or continuous positive airway pressure (CPAP) during sleep should have these treatments readily available during infusion in the event of an infusion reaction, or extreme drowsiness/sleep induced by antihistamine use.

Pretreatment with antihistamines with or without antipyretics is recommended prior to the start of infusion to reduce the risk of infusion reactions. If infusion reactions occur, decreasing the infusion rate, temporarily stopping the infusion, or administering additional antihistamines and/or antipyretics is recommended.

During infusion, serious adverse reactions included laryngeal edema, apnea, pyrexia, urticaria, respiratory distress, angioedema, and anaphylactoid reaction; severe adverse reactions included urticaria, chest pain, rash, abdominal pain, dyspnea, apnea, laryngeal edema, and conjunctivitis. The most common adverse events (≥10%) observed in clinical trials in patients treated with NAGLAZYME were rash, pain, urticaria, pyrexia, pruritus, chills, headache, nausea, vomiting, abdominal pain and dyspnea. The most common adverse reactions requiring interventions are infusion-related reactions.

Spinal/cervical cord compression is a known and serious complication that is expected to occur during the natural course of MPS VI. Signs and symptoms of spinal/cervical cord compression include back pain, paralysis of limbs below the level of compression, and urinary or fecal incontinence. Patients should be evaluated for spinal/cervical cord compression prior to initiation of NAGLAZYME to establish a baseline and risk profile. Patients treated with NAGLAZYME should be regularly monitored for the development or progression of spinal/cervical cord compression and be given appropriate clinical care.

To report SUSPECTED ADVERSE REACTIONS, contact BioMarin Pharmaceutical Inc. at 1-888-906-6100, or FDA at 1-800-FDA-1088 or

Please see full Prescribing Information.

NAGLAZYME® (galsulfase) is indicated for patients with mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome). NAGLAZYME has been shown to improve walking and stair-climbing capacity.