WARNING: Life-threatening allergic reactions have occurred in some patients during VIMIZIM® (elosulfase alfa) infusions and up to 3 hours after infusion. Patients with acute respiratory illness may be at increased risk and require additional monitoring.


  1. VIMIZIM [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2014.
  2. Wood TC, Harvey K, Beck M, et al. Diagnosing mucopolysaccharidosis IVA. J Inherit Metab Dis. 2013;36(2):293-307. doi:10.1007/s10545-013-9587-1.
  3. Data on file. BioMarin Pharmaceutical Inc.
  4. 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. doi:10.1016/j.ymgme.2013.01.021.
  5. Tomatsu S, Montaño AM, Oikawa H, et al. Mucopolysaccharidosis type IVA (Morquio A disease): clinical review and current treatment: a special review. Curr Pharm Biotechnol. 2011;12(6):931-945. doi:1389-2010/11 $58.00+.00.
  6. Bank RA, Groener JEM, van Gemund JJ, et al. Deficiency in N-acetylgalactosamine-6-sulfate sulfatase results in collagen perturbations in cartilage of Morquio syndrome A patients. Mol Genet Metab. 2009;97(3):196-201. doi:10.1016/j.ymgme.2009.03.008.
  7. Taylor KR, Gallo RL. Glycosaminoglycans and their proteoglycans: host-associated molecular patterns for initiation and modulation of inflammation. FASEB J. 2006;20(1):9-22.
  8. Clarke LA, Winchester B, Giugliani R, et al. Biomarkers for the mucopolysaccharidoses: Discovery and clinical utility. Mol. Genet. Metab. 2012. doi:10.1016/j.ymgme.2012.05.003
  9. Northover H, Cowie RA, Wraith JE. Mucopolysaccharidosis type IVA (Morquio syndrome): a clinical review. J Inherit Metab Dis. 1996;19(3):357-365.
  10. 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 Rehab Med. 2010;119-127. doi:10.3233/PRM-2010-0114.
  11. American Thoracic Society Committee on Proficiency Standards for Clinical Pulmonary Function Laboratories. ATS statement: guidelines for the six-minute walk test. Am J Respir Crit Care Med. 2002;166(1):111-117. doi:10.1164/rccm.166/1/111.
  12. Beck M, Glössl J, Grubisic A, Spranger J. Heterogeneity of Morquio disease. Clin Genet. 1986;29(4):325-331.
  13. Montaño AM, Tomatsu S, Gottesman GS, Smith M, Orii T. International Morquio A Registry: clinical manifestation and natural course of Morquio A disease. J Inherit Metab Dis. 2007;30(2):165-174. doi:10.1007/s10545-007-0529-7.
  14. Palandurkar K, Thakur S, Agrawal U, Goyal MM, Basak A. The diagnosis of Morquio disease correlating the clinical, radiological and biochemical findings: a case series. J Clin Diagn Res. 2011;5(8):1641-1645.
  15. Dvorak-Ewell M, Wendt D, Hague C, et al. Enzyme replacement in a human model of mucopolysaccharidosis IVA in vitro and its biodistribution in the cartilage of wild type mice. PLoS One. 2010;5(8):e12194. doi:10.1371/journal.pone.0012194.
  16. Coutinho MF, Lacerda L, Alves S. Glycosaminoglycan storage disorders: a review. Biochem Res Int. 2012;2012:471325. doi:10.1155/2012/471325.
  17. Hendriksz CJ, Al-Jawad M, Berger KI, et al. Clinical overview and treatment options for non-skeletal manifestations of mucopolysaccharidosis type IVA (published online ahead of print February 23, 2012). J Inherit Metab Dis. doi:10.1007/s10545-012-9459-0.
  18. Fukuda S, Tomatsu S, Masue M, et al. Mucopolysaccharidosis type IVA: N-acetylgalactosamine-6-sulfate sulfatase exonic point mutations in classical Morquio and mild cases. J Clin Invest. 1992;90(3):1049-1053.
  19. Yeung AH, Cowan MJ, Horn B, Rosbe KW. Airway management in children with mucopolysaccharidoses. Arch Otolaryngol Head Neck Surg. 2009;135(1):73-79. doi:10.1001/archoto.2008.515.
  20. Ireland MA, Rowlands DB. Mucopolysaccharidosis type IV as a cause of mitral stenosis in an adult. Br Heart J. 1981;46(1):113-115.
  21. John RM, Hunter D, Swanton RH. Echocardiographic abnormalities in type IV mucopolysaccharidosis. Arch Dis Child. 1990;65(7):746-749.
  22. Pelley CJ, Kwo J, Hess DR. Tracheomalacia in an adult with respiratory failure and Morquio syndrome. Respir Care. 2007;52(3):278-282.
  23. Pritzker MR, King RA, Kronenberg RS. Upper airway obstruction during head flexion in Morquio’s disease. Am J Med. 1980;69(3):467-470.
  24. Semenza GL, Pyeritz RE. Respiratory complications of mucopolysaccharide storage disorders. Medicine. 1988;67(4):209-219.
  25. Gulati MS, Agin MA. Morquio syndrome: a rehabilitation perspective. J Spinal Cord Med. 1996;19(1):12-16. doi:10.1159/000202621.
  26. Holzgreve W, Gröbe H, von Figura K, Kresse H, Beck H, Mattei JF. Morquio syndrome: clinical findings in 11 patients with MPS IVA and 2 patients MPS IVB. Hum Genet. 1981;57(4):360-365.
  27. Danes BS. Corneal clouding in the genetic mucopolysaccharidoses: a cell culture study. Clin Genet. 1973;4(1):1-7.
  28. Couprie J, Denis P, Guffon N, Reynes N, Masset H, Beby F. Ocular manifestations in patients affected by Morquio syndrome (MPS IV). J FrOphtalmol. 2010;33(9):617-622. doi:10.1016/j.jfo.2010.09.008.
  29. Cahane M, Treister G, Abraham FA, Melamed S. Glaucoma in siblings with Morquio syndrome. Br J Ophthalmol. 1990;74(6):382-383.23Hendriksz CJ, Al-Jawad M, Berger KI, et al. Clinical overview and treatment options for non-skeletal manifestations of mucopolysaccharidosis type IVA (published online ahead of print February 23, 2012). J Inherit Metab Dis. doi:10.1007/s10545-012-9459-0.
  30. Kinirons MJ, Nelson J. Dental findings in mucopolysaccharidosis type IV A (Morquio’s disease type A). Oral Surg Oral Med Oral Pathol. 1990;70(2):176-179.
  31. Geiger R, Strasak A, Treml B, et al. Six-minute walk test in children and adolescents. J Pediatr. 2007;150(4):395-399. doi:10.1016/j.jpeds.2006.12.052.
  32. Lehman TJ, Miller N, Norquist B, Underhill L, Keutzer J. Diagnosis of the mucopolysaccharidoses. Rheumatology. 2011;50 Suppl 5:v41-8.
  33. Tomatsu S, Fukuda S, Masue M, et al. Morquio disease: isolation, characterization and expression of full-length cDNA for human N-acetylgalactosamine-6-sulfate sulfatase. BiochemBiophys Res Commun. 1991;181(2):677-683.

Important Safety Information

Life-threatening anaphylactic reactions have occurred in some patients during VIMIZIM® (elosulfase alfa) infusions. Anaphylaxis, presenting as cough, erythema, throat tightness, urticaria, flushing, cyanosis, hypotension, rash, dyspnea, chest discomfort, and gastrointestinal symptoms (e.g., nausea, abdominal pain, retching, and vomiting) in conjunction with urticaria have been reported to occur during VIMIZIM infusions, regardless of duration of the course of treatment. Closely observe patients during and after VIMIZIM administration and be prepared to manage anaphylaxis. Inform patients of the signs and symptoms of anaphylaxis and have them seek immediate medical care should symptoms occur. Patients with acute respiratory illness may be at risk of serious acute exacerbation of their respiratory compromise due to hypersensitivity reactions, and require additional monitoring.

Due to the potential for anaphylaxis, appropriate medical support should be readily available when VIMIZIM is administered and for an appropriate period of time following administration. In clinical trials, cases of anaphylaxis occurred as early as 30 minutes from the start of infusion and up to three hours after infusion, and as late into treatment as the 47th infusion.

In clinical trials, hypersensitivity reactions have been observed as early as 30 minutes from the start of infusion but as late as six days after infusion. Frequent symptoms of hypersensitivity reactions (occurring in more than 2 patients) included anaphylactic reactions, urticaria, peripheral edema, cough, dyspnea, and flushing.

Because of the potential for hypersensitivity reactions, administer antihistamines with or without antipyretics prior to infusion. Management of hypersensitivity reactions should be based on the severity of the reaction and include slowing or temporary interruption of the infusion and/or administration of additional antihistamines, antipyretics, and/or corticosteroids for mild reactions. However, if severe hypersensitivity reactions occur, immediately stop the infusion of VIMIZIM and initiate appropriate treatment.

Consider the risks and benefits of re-administering VIMIZIM following a severe reaction.

Patients with acute febrile or respiratory illness at the time of VIMIZIM infusion may be at higher risk of life-threatening complications from hypersensitivity reactions. Careful consideration should be given to the patient’s clinical status prior to administration of VIMIZIM and consider delaying the VIMIZIM infusion.

Sleep apnea is common in MPS IVA patients. Evaluation of airway patency should be considered prior to initiation of treatment with VIMIZIM. 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 acute reaction, or extreme drowsiness/sleep induced by antihistamine use.

Spinal or cervical cord compression (SCC) is a known and serious complication of MPS IVA and may occur as part of the natural history of the disease. In clinical trials, SCC was observed both in patients receiving VIMIZIM and patients receiving placebo. Patients with MPS IVA should be monitored for signs and symptoms of SCC (including back pain, paralysis of limbs below the level of compression, urinary and fecal incontinence) and given appropriate clinical care.

All patients treated with VIMIZIM 2 mg/kg once per week in the placebo-controlled trial developed anti-drug antibodies. The relationship between the presence of neutralizing antibodies and long-term therapeutic response or occurrence of anaphylaxis or other hypersensitivity reactions could not be determined.

VIMIZIM should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. It is not known if VIMIZIM is present in human milk. Exercise caution when administering VIMIZIM to a nursing mother. There is a Morquio A Registry that collects data on pregnant women and nursing mothers with MPS IVA who are treated with VIMIZIM. Contact for information and enrollment.

Safety and effectiveness in pediatric patients below 5 years of age have not been established and are currently being evaluated. In clinical trials, the most common adverse reactions (≥10%) occurring during infusion included pyrexia, vomiting, headache, nausea, abdominal pain, chills, and fatigue. The acute reactions requiring intervention were managed by either temporarily interrupting or discontinuing infusion, and administering additional antihistamine, antipyretics, or corticosteroids.

To report SUSPECTED ADVERSE REACTIONS contact BioMarin Pharmaceutical Inc. at 1-866-906-6100, or FDA at 1-800-FDA-1088 or go to


BioMarin Pharmaceutical Inc.

Novato, CA 94949

BioMarin Medical Information: 1-800-983-4587

To report suspected adverse events email

BioMarin RareConnections™: 1-866-906-6100