Navigation Links
FDA Approves New Treatment for Late-Onset Pompe Disease
Date:5/25/2010

SILVER SPRING, Md., May 25 /PRNewswire-USNewswire/ -- The U.S. Food and Drug Administration approved Lumizyme (alglucosidase alfa) for patients ages 8 years and older with late-onset (non-infantile) Pompe disease, a rare genetic disorder.

(Logo: http://www.newscom.com/cgi-bin/prnh/20090824/FDALOGO)

Pompe disease occurs in an estimated 1 in every 40,000 to 300,000 births. Its primary symptom is heart and skeletal muscle weakness, progressing to respiratory weakness and death from respiratory failure.

In Pompe disease, a gene mutation prevents the body from making an enzyme, or making enough of the enzyme called acid alpha-glucosidase (GAA), necessary for proper muscle functioning. GAA is used by the heart and muscle cells to convert a form of sugar called glycogen into energy. Without the enzyme action, glycogen builds up in the cells and, ultimately, weakens the heart and muscles.

Lumizyme is believed to work by replacing the deficient GAA, thereby reducing the accumulated glycogen in heart and skeletal muscle cells.

"Pompe disease is a devastating condition without the appropriate treatment," said Julie Beitz, M.D., director of the Office of Drug Evaluation III in FDA's Center for Drug Evaluation and Research. "The approval of Lumizyme will provide an important treatment for patients diagnosed later in life with Pompe disease."  

Lumizyme is being approved with a risk evaluation and mitigation strategy (REMS). It will only be available through a restricted distribution system called the Lumizyme ACE (Alglucosidase Alfa Control and Education) Program to ensure that it is used by the correct patient group.  

Lumizyme will carry a Boxed Warning because of the risk of anaphylaxis, severe allergic reactions, and immune-mediated reactions.

Currently, the only other treatment for Pompe disease available in the United States is Myozyme, which is also manufactured by Genzyme at its manufacturing facilities in Framingham and Allston Landing, Mass. Myozyme has been in short supply due to limited manufacturing capacity. The manufacturer reserved Myozyme to treat infants and children with Pompe disease because younger patients generally have a much more aggressive form of the disease.

Some adult patients in the U.S. received Lumizyme under a temporary access program. The approval of Lumizyme will ensure that treatment is available for all U.S. adult Pompe patients in need of treatment. Lumizyme is manufactured at Genzyme facilities in Ireland and Belgium.

Lumizyme's safety and effectiveness have not been evaluated in patients with infantile-onset Pompe disease or in patients ages 8 years and younger with late-onset disease. These patients should be treated with Myozyme, not Lumizyme.  

The safety and efficacy of Lumizyme are based on a clinical study in 90 patients, ages 10 years to 70 years, with late-onset Pompe disease. The most commonly reported side effects for Lumizyme were infusion-related reactions and included severe allergic reactions, hives, diarrhea, vomiting, shortness of breath, itchy skin, skin rash, neck pain, partial hearing loss, flushing, pain in extremities, and chest discomfort.

Myozyme and Lumizyme are marketed by Cambridge, Mass.-based Genzyme.

For more information:

Pompe Disease Information Page – National Institute of Neurological Disorders and Stroke

http://www.ninds.nih.gov/disorders/pompe/pompe.htm

Drugs@FDA

http://www.fda.gov/Drugs/default.htm

Media Inquiries: Karen Riley, 301-796-4674, karen.riley@fda.hhs.gov

Consumer Inquiries: 888-INFO-FDA


'/>"/>
SOURCE U.S. Food and Drug Administration
Copyright©2010 PR Newswire.
All rights reserved

Related medicine technology :

1. FDA Approves Cefepime for Use In B. Brauns DUPLEX(R) System
2. U.S. FDA Approves Natazia(TM), a New Oral Contraceptive for the Prevention of Pregnancy
3. FDA Approves New Combination Product Oral Contraceptive
4. FDA Approves Labeling Change for Vyvanse(R) (lisdexamfetamine dimesylate) Capsules CII to Include Supplemental Data Demonstrating Significant Efficacy at Two Hours Through 14 Hours After Administration in Adults with ADHD
5. FDA Approves PROVENGE(R) for the Treatment of Men with Advanced Prostate Cancer
6. FDA Approves New Device for Adults with Severe and Persistent Asthma
7. FDA Approves Drug to Treat Condition That Causes Elevated Ammonia Levels
8. FDA Approves First of Its Kind, Fully Implantable (Surgically), Invisible, Prosthetic Hearing Restoration Device
9. FDA Approves First Totally Implanted Hearing System
10. FDA Approves Watsons TRELSTAR(R) 22.5 mg, the First and Only 6-Month Intramuscular GnRH Agonist for the Palliative Treatment of Advanced Prostate Cancer
11. FDA Approves Name Change for Heartburn Drug Kapidex
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:5/26/2016)... -- Since its commercial introduction in 2005, ... science tool for conducting genetic studies in a range ... its new report that the industry sits on the ... a range of new applications in the clinical diagnostics ... ) , Since the Human Genome ...
(Date:5/25/2016)...  Zymo Research Corp. announced today the final ... that help researchers obtain the most accurate and ... rapid growth of the study of microbiomes has ... methods to improve the reproducibility and quality of ... every step of the measurement process including collection ...
(Date:5/25/2016)... , May 25, 2016 Digital ... issuance to it by the US Patent and ... company,s technology includes proprietary processes for electronic opt-­in ... health and wellness programs, HIPAA compliance and otherwise. ... "Our technology allows ...
Breaking Medicine Technology:
(Date:5/27/2016)... ... May 27, 2016 , ... ... in the pharmaceutical, medical and food industries. Aside from its GMP accreditation, Validation ... providing proof of successfully certified products, services and staff. , Validation Center is ...
(Date:5/26/2016)... , ... May 26, 2016 , ... W.S. Badger ... has been honored with a 2016 When Work Works Award for its use of ... of the national When Work Works project administered by the Families and Work Institute ...
(Date:5/26/2016)... ... May 26, 2016 , ... Georgia State University College ... academic programs. , Answering to the increasing demand for curricular specializations, the Certificate ... and environmental and land use law. ,  , “The demand for lawyers with ...
(Date:5/26/2016)... ... 2016 , ... The Woodlands at John Knox Village , Florida’s first ... for living and healing, celebrated its grand opening, today. The Woodlands at John Knox ... by Empowered Staff. , “This is an incredibly fulfilling time for John Knox Village ...
(Date:5/26/2016)... ... May 26, 2016 , ... MadgeTech will be showcasing its line of ... Warner, New Hampshire at the MadgeTech headquarters. With products sold in more than 100 ... government agencies, including NASA. , In 2012, NASA strategically set up 17 RHTemp101A ...
Breaking Medicine News(10 mins):