Navigation Links
FDA Approves Kalydeco to Treat Rare Form of Cystic Fibrosis
Date:1/31/2012

SILVER SPRING, Md., Jan. 31, 2012 /PRNewswire-USNewswire/ -- The U.S. Food and Drug Administration today approved Kalydeco (ivacaftor) for the treatment of a rare form of cystic fibrosis (CF) in patients ages 6 years and older who have the specific G551D mutation in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene.

(Logo: http://photos.prnewswire.com/prnh/20090824/FDALOGO)

CF is a serious genetic disorder affecting the lungs and other organs that ultimately leads to an early death. It is caused by mutations (defects) in a gene that encodes for a protein called CFTR that regulates ion (such as chloride) and water transport in the body. The defect in chloride and water transport results in the formation of thick mucus that builds up in the lungs, digestive tract and other parts of the body leading to severe respiratory and digestive problems, as well as other complications such as infections and diabetes.

CF, which affects about 30,000 people in the United States, is the most common fatal genetic disease in the Caucasian population. About 4 percent of those with CF, or roughly 1,200 people, are believed to have the G551D mutation.

"Kalydeco is an excellent example of the promise of personalized medicine – targeted drugs that treat patients with a specific genetic makeup," said FDA Commissioner Margaret A. Hamburg, M.D. "The unique and mutually beneficial partnership that led to the approval of Kalydeco serves as a great model for what companies and patient groups can achieve if they collaborate on drug development."

The FDA reviewed and approved Kalydeco in approximately three months under the agency's priority review program that is designed to expedite the review of drugs. The priority review program uses a six-month review, instead of the standard 10 months, for drugs that may offer significant advances in treatment over available therapy.

Ka
'/>"/>

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

Page: 1 2 3

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:7/10/2014)... 10, 2014  Valeant Pharmaceuticals International, Inc. (NYSE: ... has completed the sale to Galderma of all rights ... held by Valeant for $1.4 billion in cash, pursuant ... recently completed its acquisition of Galderma. ... products to a company that is firmly committed to ...
(Date:7/10/2014)... -- CVS Caremark Corporation (NYSE: CVS ) today ... dividend of $0.275 (27.5 cents) per share on the corporation,s ... to holders of record on July 21, 2014. ... to helping people on their path to better health as ... States . Through the company,s more than 7,600 CVS/pharmacy ...
(Date:1/15/2014)... 2014 Most osteoporosis patients want a choice in ... survey* sponsored by Mission Pharmacal Company. The survey, conducted ... online support community, revealed that 74 percent of osteoporosis ... a form other than a pill or a tablet. ...
Breaking Medicine Technology:Mission Pharmacal Conducts Online Survey of Osteoporosis Patients 2Mission Pharmacal Conducts Online Survey of Osteoporosis Patients 3
... Conn.--(BUSINESS WIRE)--Jun 11, 2007 - Neurogen,Corporation (Nasdaq: NRGN) ... studies for NG2-73, the Company's lead compound,for the ... SLEEP,2007 Annual Meeting of the Associated Professional Sleep ... studies,with NG2-73 in chronic insomnia patients are currently ...
... offers five questions to help women,learn about breast cancer ... – According to a new,GfK Roper Public Affairs survey ... the majority (76,percent) of women surveyed said they know ... unaware of the important recent,progress made in treatment. Fewer ...
Cached Medicine Technology:Neurogen Proprietary Insomnia Compound Data Presented at Associated,Professional Sleep Societies Annual Meeting 2Neurogen Proprietary Insomnia Compound Data Presented at Associated,Professional Sleep Societies Annual Meeting 3Neurogen Proprietary Insomnia Compound Data Presented at Associated,Professional Sleep Societies Annual Meeting 4Women Well-Informed About Breast Cancer, Yet Lacking Knowledge,About Current Treatments, New Survey Finds 2Women Well-Informed About Breast Cancer, Yet Lacking Knowledge,About Current Treatments, New Survey Finds 3
(Date:7/11/2014)... NY (PRWEB) July 11, 2014 ... ) investigation, Bernstein Liebhard LLP has been closely ... & Drug Administration’s (FDA) Obstetrics and Gynecology ... ways to mitigate the cancer risks associated with ... gynecological surgeries. According to a report published by ...
(Date:7/11/2014)... (PRWEB) July 11, 2014 Utilizing the ... own, Dr. William Koenig, has topped a record number ... he has performed over 400 successful procedures without the ... Koenig is well known across the country for his ... safety record has long been the foundation for numerous ...
(Date:7/11/2014)... (HealthDay News) -- U.S. health officials have concluded that ... live anthrax during a safety mishap last month. ... officials have announced a moratorium on the transfer of ... In a report issued Friday, the CDC outlined ... a similar incident doesn,t occur again. Last month, as ...
(Date:7/11/2014)... 11, 2014 According to a new ... Market by Communication Technology (ZigBee, Z-Wave, Bluetooth, Wi-Fi, NFC, ... Automotive & Transportation, Agriculture) & Geography - Global Trends ... Internet of Things market was worth $1029.5 Billion in ... 2020, at an estimated CAGR of 4.08% from 2014 ...
(Date:7/11/2014)... As reported by the New York ... significant number of severe adverse events such as renal ... death from the highly-promoted and costly immune-system drug, Acthar, ... report , a regulatory filing revealed that the total ... almost 14% of prescriptions, up from 9.1% in 2011. ...
Breaking Medicine News(10 mins):Health News:Morcellator Lawsuit News: Bernstein Liebhard LLP Notes Financial Ties Between FDA Advisory Panel Members and Manufacturers of Power Morcellators 2Health News:Morcellator Lawsuit News: Bernstein Liebhard LLP Notes Financial Ties Between FDA Advisory Panel Members and Manufacturers of Power Morcellators 3Health News:Morcellator Lawsuit News: Bernstein Liebhard LLP Notes Financial Ties Between FDA Advisory Panel Members and Manufacturers of Power Morcellators 4Health News:Rochester Surgeon Hits Milestone with Keller Funnel 2Health News:CDC Issues Tough Report on Anthrax Scare 2Health News:Internet of Things Technology & Application Market Worth $1423.09 Billion by 2020 – New Report by MarketsandMarkets 2Health News:Internet of Things Technology & Application Market Worth $1423.09 Billion by 2020 – New Report by MarketsandMarkets 3Health News:Internet of Things Technology & Application Market Worth $1423.09 Billion by 2020 – New Report by MarketsandMarkets 4Health News:Acthar’s Deadly Adverse Events Revealed 2Health News:Acthar’s Deadly Adverse Events Revealed 3
... ... groundbreaking topic of Alzheimer,s prevention. , ... Naples, FL (PRWEB) November 12, 2009 -- Integrative neurologist, ... Advanced Neurotechnologies on the topic of Alzheimer,s disease prevention. The conference will be held ...
... ... Policy, Compliance, Risk, SOX, Internal Audits and IT Governance programs for the healthcare ... ... market leader in the enterprise-wide Governance, Risk and Compliance (“GRC”) solutions, ...
... ... AMD Patients with New Epimacular Brachytherapy System , ... Fremont, CA and Pisa, Italy (PRWEB) November 12, 2009 ... the commercialization of the VIDON® ANV® Therapy System, the company,s novel device for treating ...
... 12 ULURU Inc. (NYSE Alternext: ULU), ... a portfolio of wound management and oral care products, today ... shares of its common stock at a price per share ... investors, resulting in gross proceeds of approximately $1.5 million. , ...
... WARSAW, Ind., Nov. 12 Zimmer Holdings, ... has filed with the Securities and Exchange Commission an automatic shelf ... an unspecified amount of equity and debt securities and that it ... senior notes of a benchmark size. , The Company intends ...
... a pioneer in the development of novel products to ... has announced that it has filed its first IND ... Drug Administration (FDA). CEQ508, an orally administered ... implicated in the formation of colonic polyps and in ...
Cached Medicine News:Health News: Integrative Neurologist Breaks Ground in Alzheimer's Prevention 2Health News:Amedisys Selects MetricStream for Integrated Enterprise-wide GRC 2Health News:NeoVista, Inc. Announces First Neovascular AMD Patient Treated with VIDION ANV Therapy System in Europe Following Commercialization 2Health News:NeoVista, Inc. Announces First Neovascular AMD Patient Treated with VIDION ANV Therapy System in Europe Following Commercialization 3Health News:ULURU Inc. to Raise $1.5 Million in Registered Direct Offering 2Health News:Zimmer Holdings, Inc. Announces Offering of Senior Notes 2Health News:Cequent Files its First IND with FDA: CEQ508, a tkRNAi Drug Candidate in Oncology 2Health News:Cequent Files its First IND with FDA: CEQ508, a tkRNAi Drug Candidate in Oncology 3Health News:Cequent Files its First IND with FDA: CEQ508, a tkRNAi Drug Candidate in Oncology 4
... doubled Nd:YAG laser is mounted on Haag ... selected between 0.2 and 2.0 mJ. The ... and 700 microns. Knobs for laser energy ... on the laser head. The laser system ...
... Tango ophthalmic laser system provides two lasers ... and a photodisruptor for posterior capsulotomy and ... Nd:YAG producing pulsed 532 nm light. The ... nm. The appropriate laser can be easily ...
... Laser System features a patented technique for ... It selectively targets pigmented cells and increases ... II is equipped with a Q-switch that ... which induces the same cell replacement mechanism ...
Thin and smooth jaws, designed to enter trough 3 mm incision, cross action....
Medicine Products: