FAQs

Back to: "The successful effort to develop Myozyme^®"...

1. Is the movie Extraordinary Measures a true story?

The movie Extraordinary Measures is inspired by the story of John Crowley, a father who has two children with Pompe disease. In the late 1990s and early 2000s, Crowley collaborated with a scientist named William Canfield in an effort to develop a treatment for Pompe disease. Their company, called Novazyme, was acquired by Genzyme in 2001. The movie is based on a book called “The Cure” by author Geeta Anand, which presents an account of John Crowley’s efforts to develop a treatment for Pompe disease.

2. Is the drug called ‘Special Medicine’ in the movie really Myozyme?

Myozyme is the only approved treatment for Pompe disease. Genzyme originally focused efforts to develop a treatment for Pompe disease on four different drug candidates, one of which was the experimental drug developed by Crowley and Canfield. Genzyme compared all 4 candidates carefully in 2002 in an analysis nicknamed “The Mother of All Experiments” and determined that the product (Myozyme) being developed internally at Genzyme in collaboration with researchers at Duke University and the Erasmus Medical Center was the most promising. The research efforts for the other product candidates, including the drug originally developed by Crowley and Canfield, were eventually discontinued.

Genzyme continues its research efforts into a second generation drug called “neo-GAA.” Neo-GAA has some similarities to the earlier Novazyme treatment candidate developed by Bill Canfield.

3. Does Extraordinary Measures accurately represent the development of Myozyme?

Not completely, though the “Special Medicine” portrayed in the movie is no doubt Myozyme, which received approval in 2006. It also does not accurately represent the efforts of so many other heroes involved with the development of Myozyme, including patients. As the producers say, their movie is “inspired” by actual events and many parts are fictional.

4. Who developed Myozyme?

Myozyme was developed based on research conducted by Genzyme and in collaboration with researchers at Duke University and the Erasmus Medical Center in The Netherlands. The effort to develop Myozyme involved heroic efforts by hundreds of Genzyme employees working together with researchers, patients, families and healthcare professionals around the world. It is through their combined efforts that the research that led to the development of Myozyme, the first ever treatment for Pompe disease, was completed.

5. Are some elements of the movie based on facts?

Yes. The movie is based on the life events of John Crowley, a father of two children with Pompe, and William Canfield (called Robert Stonehill in the movie), a scientist. Crowley and Canfield did join forces to work on the development of a treatment for Pompe at a company called Novazyme that was acquired by Genzyme in 2001. The movie also includes many accurate reflections of the challenges that patients and families affected by Pompe face every day.

6. Was Genzyme involved in the development of Extraordinary Measures?

No, Genzyme does not have a financial interest in this film and was not directly involved in any aspect of its production. Our hope is that this story will help more people learn about Pompe disease and the challenges faced by Pompe patients, their families, and the dedicated treatment professionals who work to help them. For more than 10 years, Genzyme has worked to raise awareness of Pompe disease and to support the Pompe community, and the company is especially proud to have led the effort to develop the first and only treatment for Pompe disease.

7. How was Myozyme developed?

Myozyme was developed based on research conducted at Genzyme in collaboration with Duke University and Erasmus Medical Center in The Netherlands. The clinical trials for Myozyme were conducted at academic centers around the world. The clinical research program to develop Myozyme included two global clinical research studies conducted at seven study sites over three years. These were some of the first clinical trials ever conducted involving Pompe patients.

Genzyme completed the regulatory filings for Myozyme in the US and EU in 2005 and Myozyme was approved as the first product for the treatment of Pompe in the US and the EU in early 2006.

For additional information about the development of Myozyme, visit www.genzyme.com. For more information about Pompe disease, visit www.pompe.com.

8. How does Myozyme work?

Myozyme is a recombinant form of the body’s acid alpha-glucosidase (GAA) enzyme. Myozyme is administered via intravenous (IV) infusion every two weeks and replaces the deficient naturally occurring GAA enzyme in the body, travelling through the bloodstream to the muscles and breaking down the glycogen that causes damage when it builds up in cells.

9. Are any of the main characters from the movie still associated with Genzyme?

Bill Canfield continues to work as a researcher at Genzyme.

Important Information about MYOZYME^®
(alglucosidase alfa)

MYOZYME (alglucosidase alfa) is indicated for use in patients with Pompe disease (GAA deficiency). MYOZYME has been shown to improve ventilator-free survival in patients with infantile-onset Pompe disease as compared to an untreated historical control, whereas use of MYOZYME in patients with other forms of Pompe disease has not been adequately studied to assure safety and efficacy.

Risk of Anaphylaxis and Allergic Reactions: Life-threatening and severe allergic reactions have included anaphylactic shock, cardiac arrest, respiratory distress, hypotension, bradycardia, hypoxia, bronchospasm, throat tightness, dyspnea, angioedema, and urticaria. In clinical trials and postmarketing safety experience with MYOZYME, approximately 1% of patients developed anaphylactic shock and/or cardiac arrest during MYOZYME infusion that required life-support measures. In clinical trials and expanded access programs with MYOZYME, approximately 14% of patients treated with MYOZYME have developed allergic reactions that involved at least 2 of 3 body systems, cutaneous, respiratory, or cardiovascular systems. (Please see WARNINGS section of the Full Prescribing Information.)

Risk of Acute Cardiorespiratory Failure: Acute cardiorespiratory failure requiring intubation and inotropic support has been observed up to 72 hours after infusion with MYOZYME in infantile-onset Pompe disease patients with underlying cardiac hypertrophy, possibly associated with fluid overload with intravenous administration of MYOZYME. (Please see Full Prescribing Information for appropriate infusion volumes.)

Risk of Cardiac Arrhythmia and Sudden Cardiac Death During General Anesthesia for Central Venous Catheter Placement: Caution should be used when administering general anesthesia for the placement of a central venous catheter in infantile-onset Pompe disease patients with cardiac hypertrophy.

Infusion Reactions: The most common adverse reactions requiring intervention were infusion-related reactions which occurred in 20 of 39 (51%) of patients treated with MYOZYME in clinical studies. Some reactions were severe. Severe infusion reactions reported in more than 1 patient in clinical studies and the expanded access program included: fever, decreased oxygen saturation, tachycardia, cyanosis, and hypotension. Other infusion reactions reported in more than one patient in clinical studies and the expanded access program included: rash, flushing, urticaria, fever, cough, tachycardia, decreased oxygen saturation, vomiting, tachypnea, agitation, increased blood pressure/hypertension, cyanosis, irritability, pallor, pruritus, retching, rigors, tremor, hypotension, bronchospasm, erythema, face edema, feeling hot, headache, hyperhidrosis, increased lacrimation, livedo reticularis, nausea, periorbital edema, restlessness, and wheezing.

Some patients were pre-treated with antihistamines, antipyretics and/or steroids. Infusion reactions occurred in some patients after receiving antipyretics, antihistamines, or steroids. Infusion reactions may occur at any time during, or up to 2 hours after, the infusion of MYOZYME, and are more likely with higher infusion rates.

In addition to the infusion reactions reported in clinical trials and expanded access programs, the following have been reported during postmarketing use of MYOZYME: cardiac arrest, pharyngeal edema, peripheral edema, chest pain, chest discomfort, muscle spasm, fatigue and conjunctivitis.

Patients with advanced Pompe disease may have compromised cardiac and respiratory function, which may predispose them to a higher risk of severe complications from infusion reactions. Therefore, these patients should be monitored more closely during administration of MYOZYME. Patients with an acute underlying illness at the time of MYOZYME infusion appear to be at greater risk for infusion reactions. Careful consideration should be given to the patient’s clinical status prior to administration of MYOZYME.

Immune Mediated Reactions: Severe cutaneous and systemic immune mediated reactions have been reported in postmarketing experience with MYOZYME in at least 2 patients. Patients should be monitored for the development of systemic immune complex-mediated reactions involving skin and other organs while receiving MYOZYME.

Immunogenicity: The majority of patients in clinical trials developed antibodies to treatment with MYOZYME. The effect of antibody development on the long term efficacy of MYOZYME is not fully understood. There is an observation that some patients, who develop high and sustained anti-alglucosidase alfa antibody titers, including those who possess 2 null mutations, have a poorer clinical response.

MYOZYME is available by prescription only.

Adverse reactions should be reported promptly to Genzyme Medical Information at 800-745-4447, option 2. To learn more, please see the Full Prescribing Information or contact Genzyme Medical Information at 1-800-745-4447, option 2.

Megan Assink, who was diagnosed with Pompe disease when she was 6 weeks old, received Myozyme as part of our clinical development of this Pompe therapy.

About Pompe disease

Pompe disease is a rare and progressive neuromuscular disorder affecting both children and adults. It affects an estimated 5,000 to 10,000 patients worldwide.

In patients with Pompe, an enzyme known as acid alpha-glucosidase (GAA) is either missing or in short supply. GAA is responsible for the breakdown of glycogen, a form of sugar stored in muscle cells throughout the body. In patients with Pompe, glycogen builds up in cells in the body, weakening and damaging muscles.

As it progresses, Pompe can cause a range of serious health problems including affecting breathing and mobility.

Beginning in 1998, hundreds of employees from Genzyme departments and locations around the world worked on the Myozyme program to make it a success. Above, a group of employees from a variety of teams in Cambridge, including Clinical and Regulatory, who worked on the Myozyme program.

The development of Myozyme would have been impossible without the participation of patients and their families.

Please see Important Safety Information below, including boxed warning

Please see full PI including boxed warning Download Prescribing Information (PDF)

 

Additional Resources

• Key milestones in Genzyme’s effort to develop Myozyme (PDF)
• Frequently asked questions
• "Hollywood Treatment" - Boston Globe

Related Links

For more information, please consult these sources:

• Acid Maltase Deficiency Association
• Duke Medicine
• International Pompe Association
• Muscular Dystrophy Association
• Myozyme.com
• National Organization for Rare Disorders
• Pompe.com
• Pompe Center ErasmusMC
• United Pompe Foundation

Genzyme Media Relations

• Lori Gorski, 617-768-9344