• 
• 
• 
• 
• 
• 

FAQ’s

1. What is Morquio A?

Morquio A (Mucopolysaccharidosis IVA or MPS IVA) is a disorder caused by deficient activity of an enzyme called N acetylgalactosamine 6 sulfatase (GALNS). Deficiency of GALNS causes a buildup of a large molecule called keratan sulfate (KS) inside the cells of many different tissues in the body. This excessive storage of KS causes systemic skeletal abnormalities and joint abnormalities, which limit mobility and endurance which can result in short stature.

Malformation of the chest can impair respiratory function. Abnormalities of the vertebrae and ligaments can cause cervical spinal instability and potentially spinal cord compression. Other symptoms may include hearing loss, corneal clouding, and heart valvular disease.

The first symptoms of Morquio A often become evident in the first five years of life. Depending on the severity of disorder age of diagnosis will vary.

2. Who is BioMarin and why are we developing a treatment for MPS IVA?

• BioMarin’s mission is to develop and commercialize innovative biopharmaceuticals for serious diseases and medical conditions.
• BioMarin is a leader in the MPS field with two enzyme replacement therapies already on the market: Aldurazyme^® (laronidase) for MPS I (a joint venture with Genzyme) and Naglazyme^® (galsulfase) for MPS VI (please see important safety information below).
• Our proven experience in developing enzyme replacement therapies for MPS disorders will allow us to efficiently move our MPS IVA program forward
• Visit our website (http://www.biomarinpharm.com) to learn more about BioMarin and our treatment development programs.

3. When will the clinical trials start?

BioMarin initially intends to conduct two studies.

• A Clinical Assessment Study (no treatment) of MPS IVA patients started in October, 2008. This study is called MorCAP or MOR-001.
• A Phase 1/2 clinical study (MOR-002) was initiated in April 2009. The Phase 1/2 study will evaluate the safety of an investigational ERT. Depending on the outcome of the Phase 1/2, BioMarin hopes to initiate a Phase 3 study. It is too early to tell if and when this Phase 3 trial might take place.

4. What is the objective of BioMarin’s Clinical Assessment Study (MorCAP) for MPS IVA?

The assessment study (called MorCAP) is a study designed to better understand the disorder. The primary objective of the study is to measure limits in endurance and respiratory function in untreated MPS IVA patients and better characterize the spectrum of symptoms and biochemical abnormalities in MPS IVA. Characterization of clinical impairments across a large patient population is expected to improve understanding of the disorder and make it easier to design effective clinical trials in the future.

5. Should I participate in MorCAP?

The decision to participate in MorCAP or any other clinical trial is a decision that should be made by each person with the advice and guidance of their physician. By taking part in MorCAP you can be part of the process of gathering information that can aid in understanding MPS IVA and how to treat it.

Get more information about clinical trials from the U.S. Food and Drug Administration (FDA) at the link below.
http://www.fda.gov/ForConsumers/byAudience/ForPatientAdvocates/HIVandAIDSActivities/
ucm117893.htm

6. Will the treatment be available for all patients?

A limited number of patients will participate in the clinical trials of the investigational ERT. The Phase 1/2 study is designed to evaluate the safety and determine the optimal dose of the treatment. A Phase 3 study may be initiated after evaluating outcomes of the Phase 1/2 study.

 

NAGLAZYME

Naglazyme is indicated for patients with mucopolysaccharidosis VI (MPS VI).
Naglazyme has been shown to improve walking and stair-climbing capacity.

Important Safety Information

The most common adverse events in patients treated with Naglazyme were headache, fever, joint pain, vomiting, upper respiratory infections, abdominal pain, diarrhea, ear pain, cough, and ear infections. Severe reactions included swollen blood vessels, low blood pressure, difficulty breathing, respiratory distress, stopping breathing, and hives. The most common symptoms of infusion reactions included fever, chills/shakes, headache, rash, and mild to moderate hives. Nausea, vomiting, elevated blood pressure, chest pain, abdominal pain, malaise, and joint pain were also reported. No patients discontinued Naglazyme treatment because of reactions. Nearly all patients developed antibodies as a result of the treatment, but the level of immune response did not correlate with the severity of the adverse reaction. Because antihistamine use may increase the risk of stopping breathing, airways should be checked to ensure they are not blocked or obstructed. Treatment may be delayed if you have a fever or respiratory illness.

Please see patient product information at www.naglazyme.com. If you have any questions about this information, please talk with your doctor.

 

ALDURAZYME

ALDURAZYME® (laronidase) is indicated for patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms. The risks and benefits of treating mildly affected patients with the Scheie form have not been established.

ALDURAZYME has been shown to improve pulmonary function and walking capacity. ALDURAZYME has not been evaluated for effects on the central nervous system manifestations of the disorder.

Important Safety Information

 

Life-threatening anaphylactic reactions have been observed in some patients during or up to 3 hours after ALDURAZYME infusions. Reactions have included: respiratory failure, respiratory distress, stridor, tachypnea, bronchospasm, airway obstruction, hypoxia, hypotension, bradycardia, and urticaria. Interventions have included: resuscitation, mechanical ventilatory support, emergency tracheotomy, hospitalization, and treatment with inhaled beta-adrenergic agonists, epinephrine, and intravenous corticosteroids.

In clinical trials and postmarketing safety experience with ALDURAZYME, approximately 1% of patients experienced severe or serious allergic reactions. In patients with MPS I, pre-existing upper airway obstruction may have contributed to the severity of some reactions. Due to the potential for severe allergic reactions, appropriate medical support should be readily available when ALDURAZYME is administered. Because of the potential for recurrent reactions, some patients who experience initial severe reactions may require prolonged observation. The risks and benefits of re-administering ALDURAZYME following an anaphylactic or severe allergic reaction should be considered.

Patients with an acute illness at the time of ALDURAZYME infusion may be at greater risk for infusion-related reactions. Careful consideration should be given to the patient’s clinical status prior to administration of ALDURAZYME.

Patients should receive antipyretics and/or antihistamines prior to infusion. If an infusion reaction occurs, regardless of pretreatment, decreasing the infusion rate, temporarily stopping the infusion, and/or administration of additional antipyretics and/or antihistamines may ameliorate the symptoms.

The most common adverse reactions associated with ALDURAZYME treatment in the clinical studies were upper respiratory tract infection, rash, and injection site reaction The most common adverse reactions requiring intervention were infusion-related reactions involving flushing, fever, headache, and rash.

In postmarketing experience with ALDURAZYME, severe and serious infusion-related reactions have been reported, some of which were life-threatening. The most frequently reported adverse reactions included: chills, vomiting, nausea, arthralgia, diarrhea, tachycardia, abdominal pain, blood pressure increased, and oxygen saturation decreased.

Approximately 91% of patients treated with ALDURAZYME in clinical studies were positive for antibodies to laronidase. The clinical significance of antibodies to ALDURAZYME is not known, including the potential for product neutralization. Adverse events should be reported promptly to Genzyme Medical Information at 800-745-4447, option 2. ALDURAZYME is available by prescription only. To learn more, please see the full prescribing information (PDF) including boxed warning, visit www.ALDURAZYME.com or contact Genzyme at 1-800-745-4447.