Navigation Links
FDA Approves Drug to Treat Condition That Causes Elevated Ammonia Levels
Date:3/18/2010

SILVER SPRING, Md., March 18 /PRNewswire-USNewswire/ -- The U.S. Food and Drug Administration today approved Carbaglu (carglumic acid) Tablets to treat a condition that results in too much ammonia in the blood.

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

The condition, N-acetylglutamate synthase or NAGS deficiency, is an extremely rare, genetic disorder that can be present in babies soon after birth. NAGS deficiency and the resulting elevated levels of ammonia (hyperammonemia). can be fatal if it is not detected and treated rapidly. DNA testing can confirm the diagnosis of NAGS.

"We are very excited that more drugs are being developed to treat very rare but often devastating genetic disorders" said Janet Woodcock, M.D., director of FDA's Center for Drug Evaluation and Research. "We hope to see continuing progress in this area."

The safety and efficacy of Carbaglu was studied in 23 patients with NAGS who received the treatment for times ranging from six months to 21 years. In these patients, Carbaglu reduced blood ammonia levels within 24 hours and normalized ammonia levels within three days. The majority of those in the study appeared to maintain normal plasma ammonia levels with long-term Carbaglu treatment.

Side effects experienced by those using Carbaglu included vomiting, abdominal pain, fever, tonsillitis, anemia, ear infection, diarrhea, inflammation of the nose and throat, and headache. As with all FDA-approved products, the agency will continue to monitor Carbaglu as it is used to treat hyperammonemia.

Carbaglu should only be administered by a physician experienced in treating metabolic disorders. The recommended initial dose of Carbaglu is 100 to 250 mg/kg/day for treatment of acute hyperammonemia. Use of other ammonia-lowering therapies with Carbaglu during episodes of acute hyperammonemia is recommended. Dosing should be adjusted based on a patient's ammonia levels and symptoms.  

Consumers and health care professionals are encouraged to report adverse events to the FDA's MedWatch program at 800-FDA-1088 or online at www.fda.gov/medwatch/how.htm.

Carbaglu is manufactured by Orphan Europe.

For more information

Rare Diseases Clinical Research Network

http://www.rarediseasesnetwork.org/

National Urea Cycle Disorders Foundation

http://www.nucdf.org/

Office of Rare Diseases, National Institutes of Health

http://rarediseases.info.nih.gov

Media Inquiries: Shelly Burgess, 301-796-4651, shelly.burgess@fda.hhs.gov

Consumer Inquiries: 888-INFO-FDA

SOURCE U.S. Food and Drug Administration

Back to top

RELATED LINKS
http://www.fda.gov/
http://www.nucdf.org
http://www.rarediseasesnetwork.org
http://rarediseases.info.nih.gov

'/>"/>

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

Related medicine technology :

1. FDA Approves Lillys Osteoporosis Drug EVISTA(R) (raloxifene HCl) to Reduce The Risk of Invasive Breast Cancer in Two Populations of Postmenopausal Women
2. Health Canada Approves ATRIPLA(R) (efavirenz 600 mg/emtricitabine 200 mg/ tenofovir disoproxil fumarate 300 mg), the First Once-Daily Single Tablet Regimen for HIV
3. FDA Approves Cymbalta(R) for Maintenance Treatment of Major Depressive Disorder
4. European Commission Approves ATRIPLA(R) (efavirenz 600 mg/emtricitabine 200 mg/tenofovir disoproxil fumarate 300 mg), the First Once-Daily Single Tablet Regimen for Virologically Suppressed Adults With HIV-1 Infection
5. FDA Approves MiddleBrooks Amoxicillin PULSYS for Pharyngitis/Tonsillitis in Adolescents and Adults
6. FDA Approves XYZAL(R) (levocetirizine dihydrochloride) Oral Solution for the Relief of Seasonal and Year Round Allergies and Chronic Idiopathic Urticaria
7. UM Maryland Industrial Partnerships Program Approves 18 Collaborative Research Projects, Worth $4.4 Million, Between University Faculty, State Technology Companies
8. U.S. Food and Drug Administration Approves ABILIFY(R) (aripiprazole) for the Acute Treatment of Manic and Mixed Episodes Associated With Bipolar I Disorder in Pediatric Patients (10 to 17 Years of Age)
9. U.S. Food and Drug Administration Approves ORENCIA(R) (abatacept) for the Treatment of Moderate-to-Severe Polyarticular Juvenile Idiopathic Arthritis (JIA) in Patients Six Years and Older
10. FDA Approves HIFU Clinical Trial for Recurrent Prostate Cancer
11. EPA Approves XDS-CALUX(R) as Alternate Dioxin Test Method 4435
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/24/2016)... SOUTH SAN FRANCISCO, Calif. , June 24, ... GBT ), a biopharmaceutical company developing novel ... with significant unmet needs, today announced the closing ... 6,400,000 shares of common stock, at the public ... the shares in the offering were offered by ...
(Date:6/24/2016)... 2016 The Academy of Managed Care Pharmacy ... that would allow biopharmaceutical companies to more easily share ... formulary and coverage decisions, a move that addresses the ... The recommendations address restrictions in the sharing ... drug label, a prohibition that hinders decision makers from ...
(Date:6/24/2016)... , June 24, 2016 According ... by Type (Standard Pen Needles, Safety Pen Needles), Needle ... GLP-1, Growth Hormone), Mode of Purchase (Retail, Non-Retail) - ... This report studies the market for the forecast period ... reach USD 2.81 Billion by 2021 from USD 1.65 ...
Breaking Medicine Technology:
(Date:6/24/2016)... Angeles, CA (PRWEB) , ... June 24, 2016 , ... ... surgery procedures that most people are unfamiliar with. The article goes on to state ... procedures, but also many of these less common operations such as calf and cheek ...
(Date:6/24/2016)... ... June 24, 2016 , ... Marcy was in a crisis. Her son James, eight, was ... his family verbally and physically. , “When something upset him, he couldn’t control his emotions,” ... He would throw rocks at my other children and say he was going to kill ...
(Date:6/24/2016)... San Francisco, CA (PRWEB) , ... June 24, ... ... at CitiDent, is now offering micro-osteoperforation for accelerated orthodontic treatment. Dr. Cheng has ... , self-ligating Damon brackets , AcceleDent, and accelerated osteogenic orthodontics. , ...
(Date:6/24/2016)... ... June 24, 2016 , ... People across the U.S. ... magazine’s Code Talker Award, an essay contest in which patients and their families pay ... be presented at the 2016 National Society of Genetic Counselors (NSGC) Annual Education Conference ...
(Date:6/24/2016)... (PRWEB) , ... June 24, 2016 , ... ... client, The Grove Investment Group (TGIG), has initiated cultivation and processing operations at ... Las Vegas and Pahrump, Nevada. , Puradigm is the manufacturer of a complete ...
Breaking Medicine News(10 mins):