Navigation Links
FDA Grants Orphan Drug Designation to Bayer's Investigational Compound Regorafenib for the Treatment of Gastrointestinal Stromal Tumors
Date:2/4/2011

WAYNE, N.J., Feb. 4, 2011 /PRNewswire/ -- Bayer HealthCare Pharmaceuticals Inc. announced today that its investigational compound regorafenib (BAY 73-4506) has been granted orphan drug status for the treatment of patients with gastrointestinal stromal tumors (GIST) by the U.S. Food and Drug Administration (FDA).

Regorafenib is an investigational agent and is not approved by the FDA, the European Medicines Agency (EMA) or other Health Authorities.

"This is an important step in the overall development process for this investigational compound," said Kemal Malik, MD, Head of Global Development and member of the Bayer HealthCare Executive Committee.

In January, Bayer began enrolling patients in a randomized, double-blind, placebo-controlled Phase III study of regorafenib plus best supportive care versus placebo plus best supportive care for subjects with metastatic and/or unresectable gastrointestinal stromal tumors (GIST) whose disease has progressed despite prior treatment with at least imatinib and sunitinib (NCT01271712). The trial is estimated to enroll 170 patients, who will be randomized in a 2:1 ratio to receive either regorafenib or placebo. Subjects receiving placebo who experience disease progression may be offered open-label regorafenib treatment (cross-over option).

The primary endpoint of this trial is progression-free survival (PFS), and secondary endpoints include overall survival (OS), time to progression (TTP), disease control rate (DCR), tumor response rate (RR), duration of response (DOR), and safety. All patients will enter the Survival Follow-Up Period upon discontinuation of study treatment, during which assessment of survival status will be performed every three months.

For information about this study, please visit www.clinicaltrials.gov (NCT01271712).

Orphan Drug Designation in the United States

In the United States, the Orphan Drugs Act (ODA) provides for the orphan drug designation which aims to encourage the development of drugs involved in the diagnosis, prevention or treatment of a medical condition affecting fewer than 200,000 people in the country. The designation grants U.S. market exclusivity to a drug for a particular indication for a seven-year period if the sponsor complies with certain FDA specifications. Additional incentives for the sponsor include tax credits related to clinical trial expenses and a possible exemption from the FDA-user fee. The designation does not shorten the duration of the regulatory review and approval process.  

About GIST

GIST is the most common form of sarcoma involving the gastrointestinal tract. GISTs arise in the GI tract, with most primary tumors originating in the stomach or small intestine. GIST represents a potentially life-threatening malignancy if the disease has spread to other parts of the body (metastasized) or is unable to be surgically removed with curative intent. The incidence of GIST is estimated to be 11 to 20 patients per million per year. The international prevalence of GISTs is comparable to that reported in the United States. In the U.S., it is estimated that there are approximately 4,500-6,000 new cases of GISTs diagnosed each year, of which about 1,500 have already metastasized when they are initially found. GISTs are difficult to diagnose and are usually found incidentally when the doctor is looking for other problems.

About Bayer HealthCare Pharmaceuticals Inc.

Bayer HealthCare Pharmaceuticals Inc. is the U.S.-based pharmaceuticals business of Bayer HealthCare LLC, a subsidiary of Bayer AG. Bayer HealthCare is one of the world's leading, innovative companies in the healthcare and medical products industry, and combines the activities of the Animal Health, Consumer Care, Diabetes Care, and Pharmaceuticals divisions.  As a specialty pharmaceutical company, Bayer HealthCare Pharmaceuticals provides products for Diagnostic Imaging, General Medicine, Hematology, Neurology, Oncology and Women's Healthcare.  The company's aim is to discover and manufacture products that will improve human health worldwide by diagnosing, preventing and treating diseases.

Forward-Looking Statements

This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer Group or subgroup management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer's public reports which are available on the Bayer website at www.bayer.com.  The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.


'/>"/>
SOURCE Bayer HealthCare Pharmaceuticals Inc.
Copyright©2010 PR Newswire.
All rights reserved

Related medicine technology :

1. US FDA Grants Orphan Drug Status to Immtechs Pafuramidine for Treatment of African Sleeping Sickness
2. Chinese SFDA Grants Immtech Fast Track Status
3. Ethics Group Asks USAID Inspector General to Investigate Legality of Grants to Soros Groups
4. NTMir Announces Research Grants for Epidemiology and Household Water as Source of Infection
5. FDA Grants Approval to Extend U.S. Enrollment and Clinical Trial
6. FDA Grants Priority Review for Daiichi Sankyo, Lilly Drug, prasugrel
7. FDA Grants Priority Review for ACIPHEX(R) (rabeprazole sodium) sNDA for Short-Term Treatment of GERD in Adolescents
8. FDA Grants Priority Review for PROMACTA(R) (eltrombopag)
9. FDA Grants NeoVistas Request to Expand CABERNET Trial to 30 Sites in the U.S.
10. FDA Grants Market Clearance for the LipiScan(TM) Coronary Imaging System Developed by InfraReDx, Inc. - A Novel Spectroscopy System for the Identification of Lipid Core Containing Plaques of Interest in the Coronary Arteries
11. FDA Grants Pediatric Exclusivity for TOPAMAX(R), Extending the Marketing Exclusivity by Six Months
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:2/21/2017)... Feb. 21, 2017 Patient Monitoring ... are used to monitor physiological signs. These devices are ... monitors. Remote patient monitoring is one of the key ... equipment in the market. Hence, vendors are developing technologically ... applications, which can send test results on time and ...
(Date:2/21/2017)... The global infusion pumps market is estimated ... to 2021, to reach USD 10.84 billion by 2021 ... chronic diseases along with increasing geriatric population, increasing adoption ... increase in surgical procedural volumes, and rising applications of ... key factors driving the growth of this market. However, ...
(Date:2/21/2017)... , Feb. 21, 2017  Luminex Corporation (NASDAQ: ... of directors has authorized the initiation of a quarterly cash ... This initial cash dividend will be payable on April 14, ... business on March 24, 2017. The board ... quarterly cash dividend of $0.06 to holders of its common ...
Breaking Medicine Technology:
(Date:2/22/2017)... ... February 22, 2017 , ... Bellus ... the latest innovation in the delivery of Platelet Rich Plasma (PRP). PRP systems ... pain management, to accelerate tissue synthesis and provide a faster and more efficient ...
(Date:2/22/2017)... ... February 22, 2017 , ... Super-Sod will attend the Athens Home Show ... Georgia. , A shift from Super-Sod’s simple Athens Home Show booth of 2016, this ... grass seed plant manager Chris Roquemore constructed furniture from recycled pallet wood at the ...
(Date:2/22/2017)... (PRWEB) , ... February 22, 2017 , ... ... threats. Yet with so many different solutions on the market, it is easy ... choices, Texas Premier Locksmith offers a complimentary security consultation. , Home ...
(Date:2/21/2017)... ... 2017 , ... Author Michèle Wolff has a passion for using food ... know all about it for optimal health. Wanting to share her knowledge to the ... and Wellness Solutions ” (published by Balboa Press AU). , This book inspires ...
(Date:2/21/2017)... ... February 21, 2017 , ... As recently as 2015, rhinoplasty was one ... Society of Plastic Surgeons. Some patients want to make a change in the appearance ... improve their breathing ability. The team at usrhinoplasty.org is expanding its article ...
Breaking Medicine News(10 mins):