Navigation Links
FDA Approves First-of-its-Kind Drug to Treat Rare Blood Disorder

The U.S. Food and Drug Administration (FDA) today approved Soliris (eculizumab), the first product for the treatment of paroxysmal nocturnal hemoglobinuria// (PNH), a rare type of blood disorder that can lead to disability and premature death. Soliris is classified as an Orphan Drug and is a new molecular entity containing an ingredient not previously marketed in the United States.

"This product is important in that it offers a treatment other than blood transfusion that may help this small population of patients who are often very ill," said Steven Galson, M.D., M.P.H., director, Center for Drug Evaluation and Research, FDA. "This approval is one of multiple examples of how the orphan products program can benefit the public health with urgently needed products that would otherwise not be commercially available."

PNH, which usually develops in adults, is a disease characterized by red blood cells that develop abnormally. Once the abnormal cells are present in the bloodstream, naturally occurring proteins (called the complement system) designed to destroy bacteria and other infection-causing organisms break these cells down. This leads to abnormally darkened urine and, more importantly, causes anemia. Depending upon the severity of the disorder, patients with PNH may have pain, fatigue and debilitating weakness, the need for frequent blood transfusions, blood clots, and life-threatening or fatal strokes, heart attacks and intestinal disease.

Soliris does not cure PNH, but treats the breakdown of red blood cells, the most common characteristic of PNH. Soliris acts to block the complement system activity, including the destruction of PNH red blood cells.

FDA based its approval on the company's randomized, double-blind, placebo-controlled clinical study of 87 patients with PNH and a series of other clinical studies. The controlled study showed that over a 26-week period half of the participants receiving Soliris had stab
'"/>




Page: 1 2

Related medicine news :

1. FDA Approves Contraceptive Ring
2. FDA Approves New HIV Drug, Viread
3. FDA Approves Birth Control Skin Patch
4. FDA Approves New Arthritis Drug, Bextra
5. FDA Approves Elidel Cream for Eczema
6. FDA Approves New Anti-Clotting Drug
7. FDA Approves New surgery Treatment for Farsightedness
8. FDA Approves Anti-Cancer Cell Drug - Zevalin
9. FDA Approves Remodulin for Pulmonary Hypertension
10. World Health Organisation Takes a Historic Decision and Approves Treaty to Discourage Smoking
11. World Health Organisation Takes a Historic Decision and Approves Treaty to Discourage Smoking
Post Your Comments:
*Name:
*Comment:
*Email:
TAG: FDA Approves First its Kind Drug Treat Rare Blood Disorder

(Date:10/31/2014)... 2014 — A team from the Broad Institute, ... of California, Santa Cruz, was awarded one of ... Pilot contracts with the goal of building a ... Cancer Genome Atlas (TCGA) and other datasets by ... in one cloud environment. This co-location will enable ...
(Date:10/31/2014)... At the start of 2014, numerous news ... monoxide poisoning in their homes. Now, as 2015 (and ... Missouri-based heating and cooling company is taking a more ... the home heating season with the assurance that their ... Nov. 1, 2014, E and Q Heating and Cooling ...
(Date:10/31/2014)... apnea may make it hard for you to remember simple ... your house keys, a small study suggests. Tests on ... -- called spatial memory -- was impaired when sleep apnea ... of sleep weren,t affected. REM sleep is the deepest level ... for the first time that sleep apnea, an increasingly common ...
(Date:10/31/2014)... lab device to give cancer researchers an unprecedented microscopic ... spread through the body, causing more than 90 percent ... tumor cells travel, the device could uncover new ways ... the university,s Whiting School of Engineering and its Institute ... new system recently in the journal Cancer Research ...
(Date:10/31/2014)... scientists have found a drug combination that can trigger ... the way for new treatments, according to research that ... (NCRI) Cancer Conference in Liverpool next week*. , When ... chain of events culminating in self destruction. But cancer ... immortal. This means that cells grow out of control ...
Breaking Medicine News(10 mins):Health News:Broad Institute, Univ. of California team awarded NCI Cancer Genomics Cloud Pilot contract 2Health News:Broad Institute, Univ. of California team awarded NCI Cancer Genomics Cloud Pilot contract 3Health News:Many Homeowners Missing Key Safety Risks with Home Heating;Missouri-based Company E and Q Announces New Safety Check 2Health News:Many Homeowners Missing Key Safety Risks with Home Heating;Missouri-based Company E and Q Announces New Safety Check 3Health News:Sleep Apnea May Steal Some of Your Memory: Study 2Health News:Viewing cancer on the move: New device yields close-up look at metastasis 2Health News:Viewing cancer on the move: New device yields close-up look at metastasis 3Health News:Scientists trigger self-destruct switch in lung cancer cells 2
... America, Inc. (OTC Bulletin Board: CBAI), the umbilical cord blood ... ) focused on bringing the life saving potential of ... announce that between February 15, 2009, and today, it has ... percent. This follows the announcement on Feb. 12, 2009 ...
... Stars Perform to Raise Awareness of Neurological DiseaseEAST RUTHERFORD, N.J., April ... Foundation: WHO: Anthony Galde of Wicke ... Research Foundation WHAT: Curtain Call for a Caus ... & the Dystonia Medical Research Foundation host the 2nd Annual ...
... The United States Marine Corps launches its inaugural ... city opens its doors to the nearly 2,000 Marines ... Chicagoland area. "Established to recognize the contributions of ... which they came, Marine Week also showcases the rich ...
... arthritis (RA) is a systemic disease or an autoimmune disorder ... the thin layer of cells called the synovium that lines ... are swollen joints and crippling stiffness, particularly of the hands ... and also impedes mobility and quality of life. RA cannot ...
... vaccines and critical-care biopharmaceuticals announces exclusive sanofi pasteur Fluzone® agreement for ... ... (Vocus) April 6, 2009 -- As a trusted partner ... is pleased to announce that sanofi pasteur has named FFF the ...
... Lens Boot Camp™ - a first-of-its-kind training program to help refractive ... that its first boot camp session will be held on June ... ... Camarillo, CA (PRWEB) April 6, 2009 -- Premium Lens Boot Camp™ ...
Cached Medicine News:Health News:Cord Blood Announces $1.75 Million Reduction in Long Term Debt 2Health News:Broadway Comes to New Jersey 2Health News:Marine Corps Launches Inaugural Marine Week in Chicago, May 11-17 2Health News:Marine Corps Launches Inaugural Marine Week in Chicago, May 11-17 3Health News:Rheumatoid Arthritis Therapeutics in Thailand 2Health News:Rheumatoid Arthritis Therapeutics in Thailand 3Health News:FFF Named the Exclusive Distributor of Fluzone for GPOs & Announces New Fluzone Return Policy 2Health News:Premium Lens Boot Camp™ Helps Refractive Surgeons Grow Their Premium IOL Practice 2
(Date:10/31/2014)... 30, 2014 Electronic prescribing or ... of prescription related information between prescriber, dispenser ... a physician and nurse practitioner to transmit ... error occurs during pharmacy operations. E-Prescribing system ... provides complete medical lists, conducts all security ...
(Date:10/31/2014)... Oct. 31, 2014  AbbVie (NYSE: ... third quarter ended Sept. 30, 2014. ... of our business with double-digit growth from HUMIRA ... grew nearly 14 percent in the quarter excluding ... executive officer, AbbVie.  "We exceeded our outlook for ...
(Date:10/30/2014)... MAHWAH, N.J. , Oct. 30, 2014 ... PGA TOUR and Champions Tour, is rounding out its ... Cup Championship in Scottsdale, AZ. ... PGA TOUR and Champions Tour that the brand has ... destination designed to educate golf fans on the importance ...
Breaking Medicine Technology:E-Prescribing Market - Global Industry Analysis, Size, Share, Growth, Trends and Forecast, 2013 - 2019 2AbbVie Reports Third-Quarter 2014 Financial Results 2AbbVie Reports Third-Quarter 2014 Financial Results 3AbbVie Reports Third-Quarter 2014 Financial Results 4AbbVie Reports Third-Quarter 2014 Financial Results 5AbbVie Reports Third-Quarter 2014 Financial Results 6AbbVie Reports Third-Quarter 2014 Financial Results 7AbbVie Reports Third-Quarter 2014 Financial Results 8AbbVie Reports Third-Quarter 2014 Financial Results 9AbbVie Reports Third-Quarter 2014 Financial Results 10AbbVie Reports Third-Quarter 2014 Financial Results 11AbbVie Reports Third-Quarter 2014 Financial Results 12AbbVie Reports Third-Quarter 2014 Financial Results 13AbbVie Reports Third-Quarter 2014 Financial Results 14AbbVie Reports Third-Quarter 2014 Financial Results 15AbbVie Reports Third-Quarter 2014 Financial Results 16AbbVie Reports Third-Quarter 2014 Financial Results 17AbbVie Reports Third-Quarter 2014 Financial Results 18AbbVie Reports Third-Quarter 2014 Financial Results 19AbbVie Reports Third-Quarter 2014 Financial Results 20AbbVie Reports Third-Quarter 2014 Financial Results 21AbbVie Reports Third-Quarter 2014 Financial Results 22AbbVie Reports Third-Quarter 2014 Financial Results 23AbbVie Reports Third-Quarter 2014 Financial Results 24Stryker Mobility Zone Makes Final Stop Of 2014 At Charles Schwab Cup Championship 2Stryker Mobility Zone Makes Final Stop Of 2014 At Charles Schwab Cup Championship 3Stryker Mobility Zone Makes Final Stop Of 2014 At Charles Schwab Cup Championship 4
... Show Increased Enzyme Levels in Two Most ... /PRNewswire/ -- Amicus,Therapeutics, a biopharmaceutical company developing ... a range of human genetic diseases, announced ... preclinical studies of,Plicera(TM) (isofagomine tartrate, AT2101) for ...
... the Targeted,Anticancer Therapies (TAT 2007) and the ... Phase 1 Trial Design, SOUTH SAN FRANCISCO, ... Inc. , presented,non-clinical data on its development-stage ... on targeted anticancer,medicines. Findings from in vitro ...
Cached Medicine Technology:Amicus Therapeutics Presents Preclinical Data from Studies of,Plicera for Gaucher Disease 2Amicus Therapeutics Presents Preclinical Data from Studies of,Plicera for Gaucher Disease 3Sunesis Pharmaceuticals Presents SNS-314 Aurora Kinase Inhibitor,Data Showing Broad Anti-Tumor Activity, Dosing Flexibility 2Sunesis Pharmaceuticals Presents SNS-314 Aurora Kinase Inhibitor,Data Showing Broad Anti-Tumor Activity, Dosing Flexibility 3
Cordis DUCOR Catheters are available in a broad variety of French sizes and configurations. These catheters combine an atraumatic tip with either a braided or non-braided body....
The advanced center-of-the-arc system allows an infinite choice of trajectories. The system's lightweight durable frame provides accuracy, simplicity, patient mobility and accessibility....
The CRW system epitomizes simplicity, quality assurance, and versatility....
Used for complex diagnostic and interventional procedures....
Medicine Products: