NAGLAZYME® (galsulfase) safety information

With all medications, there is a chance of side effects. This is true of NAGLAZYME® (galsulfase) too.

Possible allergic reactions

Severe and life‐threatening allergic reactions can occur during NAGLAZYME infusions and up to 24 hours after infusion. Typical signs of an allergic reaction include shock, difficulty breathing, wheezing, swelling of the throat, and low blood pressure. If a severe allergic reaction occurs during infusion, the infusion should be stopped immediately and you should receive medical attention. Contact your doctor or get medical help right away if you develop any severe symptoms after infusion.

Infusion‐associated reactions

Because NAGLAZYME is given by infusion, there are reactions that may happen as a result of the infusion. In clinical trials, some people experienced serious and severe infusion reactions like hives, chest pain, rash, abdominal pain, difficulty breathing, swelling, fever, and eye irritation. You should receive medication such as antihistamines before NAGLAZYME infusions to reduce the risk of infusion‐associated reactions.

If an infusion‐associated reaction occurs, the infusion should be slowed or stopped and you may be given additional medication. For most patients, these problems went away when the infusion was stopped or slowed down. In some cases, patients were given another medicine, such as an antihistamine or pain reliever, to help.

Learn more about possible infusion‐associated reactions

Side effects

In clinical studies, the most common side effects of NAGLAZYME were rash, pain, hives, fever, itching, chills, headache, nausea, vomiting, abdominal pain, and difficulty breathing. The most common side effects requiring medical attention are infusion‐associated reactions.

Learn about more serious NAGLAZYME side effects (also known as adverse events)

View NAGLAZYME Warnings and Precautions

Infusion‐associated reactions

Despite routine pretreatment with antihistamines, infusion‐associated reactions, some severe, occurred in 33 of 59 patients treated with NAGLAZYME. The most common symptoms of drug‐related infusion‐associated reactions were pyrexia, chills, rash, urticaria, dyspnea, nausea, vomiting, pruritus, erythema, abdominal pain, hypertension, and headache. Respiratory distress, chest pain, hypotension, angioedema, conjunctivitis, tremor, and cough were also reported. Infusion‐associated reactions began as early as week 1 and as late as week 146 of NAGLAZYME treatment.

Symptoms typically abated with slowing or temporary interruption of the infusion and administration of additional antihistamines, antipyretics, and occasionally corticosteroids. Most patients were able to complete their infusions. Subsequent infusions were managed with a slower rate of NAGLAZYME administration, treatment with additional prophylactic antihistamines, and, in the event of a more severe reaction, treatment with prophylactic corticosteroids. Twenty‐three of 33 patients (70%) experienced recurrent infusion‐associated reactions during multiple infusions, although not always in consecutive weeks.

Adverse events

The most common serious adverse events related to the use of NAGLAZYME occurred during infusions and included apnea, pyrexia, and respiratory distress. Severe adverse events included chest pain, dyspnea, laryngeal edema, and conjunctivitis. The most common adverse events in clinical studies were rash, pain, urticaria, pyrexia, pruritus, chills, headache, nausea, vomiting, abdominal pain, and dyspnea. The most common adverse events requiring interventions are infusion‐associated reactions.

The table below enumerates adverse events reported during the 6‐month placebo‐controlled trial that occurred more frequently in the NAGLAZYME group than in the placebo group. Each event listed occurred in at least 2 more patients treated with NAGLAZYME than in placebo‐treated patients. Observed adverse events in the phase 1, phase 2, and open‐label extension studies were not different in nature or severity.

Immune‐mediated reactions

Type III immune complex–mediated reactions have been observed with NAGLAZYME, as with other enzyme replacement therapies. If immune‐mediated reactions occur, initiate appropriate medical treatment and consider discontinuation of NAGLAZYME. The risks and benefits of readministering NAGLAZYME following an immune‐mediated reaction should be considered. Some patients have successfully continued to receive NAGLAZYME under close clinical supervision.

List of adverse events recorded during the primary trial of NAGLAZYME

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Warnings and precautions

Life‐threatening anaphylactic reactions have been observed in some patients during NAGLAZYME infusions and up to 24 hours after infusion. If anaphylaxis or other severe allergic reactions occur, immediately discontinue infusion and initiate appropriate treatment, which may include resuscitation, epinephrine, and administering additional antihistamines, antipyretics, or corticosteroids.

Caution should be exercised when administering NAGLAZYME to patients susceptible to fluid volume overload, because congestive heart failure may result. Appropriate medical support and monitoring measures should be readily available during NAGLAZYME infusion. Some patients may require prolonged observation times.

Sleep apnea is common in MPS VI patients and antihistamine pretreatment may increase the risk of an apneic episode. Evaluation of airway patency should be considered prior to 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.

Spinal cord damage may occur due to the natural MPS VI disease process. Signs of spinal cord injury include back pain, loss of bladder and bowel control, numbness, and paralysis. Contact your doctor immediately if you develop any of these symptoms.

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Important Safety Information

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

Severe and life-threatening allergic reactions can occur during NAGLAZYME (galsulfase) infusions and up to 24 hours after infusion. Typical signs of an allergic reaction include shock, difficulty breathing, wheezing, swelling of the throat, and low blood pressure. If a severe allergic reaction occurs during infusion, the infusion should be stopped immediately and you should receive medical attention. Contact your doctor or get medical help right away if you develop any severe symptoms after infusion.

In clinical trials, most patients developed antibodies to NAGLAZYME treatment. There was no clear relationship between antibody formation and the safety or effectiveness of NAGLAZYME.

Serious and severe infusion reactions are associated with NAGLAZYME, including hives, chest pain, rash, abdominal pain, difficulty breathing, swelling, fever, and eye irritation. You should receive medication such as antihistamines before NAGLAZYME infusions to reduce the risk of infusion reactions. If an infusion reaction occurs, the infusion should be slowed or stopped and you may be given additional medication.

The most common side effects of NAGLAZYME seen in clinical trials were rash, pain, hives, fever, itching, chills, headache, nausea, vomiting, abdominal pain and difficulty breathing. The most common side effects requiring medical attention are infusion-related effects.

These are not all of the possible side effects with NAGLAZYME. Talk to your doctor if you have any symptoms that bother you or that do not go away.

NAGLAZYME is a prescription medicine. Before treatment with NAGLAZYME, it is important to discuss your medical history with your doctor. Tell your doctor if you are taking any medication and if you are allergic to any medicines. Your doctor will decide if NAGLAZYME is right for you. If you have questions or would like more information about NAGLAZYME, contact your doctor.

Spinal cord damage may occur due to the natural MPS VI disease process. Signs of spinal cord injury include back pain, loss of bladder and bowel control, numbness, and paralysis. Contact your doctor immediately if you develop any of these symptoms.

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

Please see full Prescribing Information.