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.

BioMarin RareConnections™: personalized support to help people with Morquio A gain access to VIMIZIM

BioMarin RareConnections™ is committed to working with patients, physicians, and insurance providers to provide access to VIMIZIM® (elosulfase alfa) therapy and ongoing support. The BioMarin RareConnections team includes an Account Manager, Case Manager, and Clinical Nurse Educator, all dedicated to helping your patients start and stay on VIMIZIM.

Support for physicians

BioMarin RareConnections can provide you and your staff a range of services

  • Access to laboratory testing around diagnosis confirmation
  • Coordination with site for drug delivery logistics
  • Infusion day coordination and logistics
  • Patient support (see below)

Support for patients

BioMarin RareConnections provides patients a wide variety of support services at no cost

  • Education on Morquio A and VIMIZIM
  • Personalized support to navigate the reimbursement process for tests and treatment
  • Additional treatment support such as transportation assistance
  • Help with treatment continuity regardless of life changes

Access to treatment

BioMarin RareConnections Case Managers can help patients and treating physicians navigate the reimbursement process as it pertains to necessary tests and treatment. If a patient has limited or no insurance, BioMarin RareConnections will work with them to find out if they qualify for an assistance program or whether alternative ways to cover infusions need to be explored. If needed, Case Managers can direct patients to third party, nonprofit organizations such as the National Organization for Rare Disorders (NORD) that can potentially help pay for VIMIZIM.

Treatment support

Treatment support is offered for patients receiving infusions at a clinic or in the home setting.

  • For patients receiving infusions at a clinic, case managers can arrange transportation to and from the clinic and ensure that VIMIZIM is delivered to the clinic prior to infusion
  • BioMarin RareConnections will also support the transition to home infusions for each patient, as directed by their physician. This may include finding and training an infusion nurse, ensuring VIMIZIM delivery, and helping the patient with proper storage

Starting on and staying on VIMIZIM

Long­term commitment to treatment can be an issue with chronic therapies such as VIMIZIM. BioMarin RareConnections Case Managers are available to help patients make necessary plans to maintain their weekly infusion schedule, especially in the face of changes such as a new job, new insurance, a move to a different location, or going off to college.

Enrolling in BioMarin RareConnections

In order to benefit from BioMarin RareConnections, patients must sign and return the HIPAA (Health Insurance Portability and Accountability Act of 1996) consent forms. These forms can be obtained by contacting BioMarin RareConnections or by downloading them here.


    Download Adobe Reader

Please contact BioMarin RareConnections if you’d like assistance with either of these steps.

Phone: 1-866-906-6100

Fax: 1­-888­-863-­3361


VIMIZIM overview
Learn more about the treatment supported by BioMarin RareConnections™
VIMIZIM administration instructions
Step-by-step instructions on how to administer VIMIZIM

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