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BioMarin RareConnectionsTM helps patients gain access to therapy throughout their treatment journey
WARNING: Life-threatening anaphylactic reactions have occurred in some patients during VIMIZIM infusions. Please see the Important Safety Information and the full Prescribing Information for additional safety information.
BioMarin’s HUB provides patients and clinics with:
- Personalized support—RareConnections Case Managers work one-to-one with patients and their families; RareConnections Field Reimbursement Managers work one-to-one with your clinic
- Product access education and support
- Help for patients prescribed NAGLAZYME® (galsulfase) so they can navigate the insurance process and understand coverage options to gain access to treatment
- Financial assistance programs for eligible patients
- Information about payer requirements your clinic needs to seek access for treatment coverage for patients
- Logistics support—Case Managers can assist with directing the prescription to the appropriate specialty pharmacy and coordinating the shipment to the infusion center or the patient’s home as needed
To enroll your patient in BioMarin RareConnections, complete the NAGLAZYME Patient Enrollment Form (PEF) and the Patient Consent Form (PCF).
Download the NAGLAZYME Coverage Authorization Guide.
A comprehensive resource including sample Letter of Medical Necessity for accessing NAGLAZYME treatment for your patients.
Download the guide 
NAGLAZYME® (galsulfase) Indication and Important Safety Information
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS
VIMIZIM® (elosulfase alfa) Indication and Important Safety Information
WARNING: RISK OF ANAPHYLAXIS
INDICATION
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.
IMPORTANT SAFETY INFORMATION
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS
Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. These reactions have occurred during and up to 24 hours after completion of the NAGLAZYME infusion. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.
Administration of NAGLAZYME should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions, including anaphylaxis.
Initiate NAGLAZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue NAGLAZYME and immediately initiate appropriate medical treatment, including use of epinephrine. In patients who have experienced anaphylaxis or other severe allergic reactions during infusion with NAGLAZYME, caution should be exercised upon rechallenge. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur.
Immune-Mediated Reactions
As with other enzyme replacement therapies, immune-mediated reactions, including membranous glomerulonephritis have been observed. If immune-mediated reactions occur, consider discontinuing Naglazyme administration and initiate appropriate medical treatment. Consider the risks and benefits of re-administering Naglazyme following an immune-mediated reaction. Some patients have successfully been rechallenged and have continued to receive Naglazyme under close clinical supervision.
Risk of Acute Cardiorespiratory Failure
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.
Acute Respiratory Complications Associated with Administration
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.
Infusion Reactions
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.
Spinal or Cervical Cord Compression
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.
Adverse Reactions
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.
To report SUSPECTED ADVERSE REACTIONS, contact BioMarin Pharmaceutical Inc. at 1-866-906-6100, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please see full Prescribing Information.
INDICATION
VIMIZIM® (elosulfase alfa) is indicated for patients with mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).
IMPORTANT SAFETY INFORMATION
Life-threatening anaphylactic reactions have occurred in some patients during VIMIZIM infusions. Anaphylaxis, presenting as cough, erythema, throat tightness, urticaria, flushing, cyanosis, hypotension, rash, dyspnea, chest discomfort, and gastrointestinal symptoms 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 3 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 6 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 includes 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; 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, and 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 MARS@BMRN.com 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 antihistamines, 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 www.fda.gov/medwatch.
Please see accompanying full Prescribing Information, including Boxed Warning.