Navigation Links
McGill's 'Orphan Drug' research offers hope
Date:6/1/2010

This release is available in French.

In addition to their suffering, rare disease patients often have to face the harsh reality that few pharmaceutical companies will ever be able to offer new treatments for their condition because the costs of new treatments will never be recovered from such a small market. But there are ways they can be helped. The U.S. Food and Drug Administration's "Orphan Drug Designation" offers a wide range of benefits that help organizations developing treatments for diseases and conditions affecting fewer than 200,000 patients in the United States. It was recently granted to McGill University for research conducted at the MUHC into the use of the drug fenretinide for the treatment of pulmonary infections caused by Pseudomonas aeruginosa in patients with Cystic Fibrosis (CF).

CF is a chronic, hereditary disease affecting the respiratory and digestive systems of approximately 70,000 people worldwide. Pseudomonas aeruginosa infects more than 80 per cent of CF patients and contributes to the decline in pulmonary function the ability of the body to pump blood between the heart and the lungs. The infection is a major cause of sickness and death and is difficult to treat since it is resistant to many drugs.

"We strongly believe that fenretinide has the potential to improve morbidity and prognosis in CF patients at different levels," said Dr. Danuta Radzioch, a professor of Experimental Medicine at the MUHC and McGill's Faculty of Medicine. "Our research has demonstrated that fenretinide reduced lung inflammation as well as the frequency and the severity of pulmonary infections." Fenretinide is a drug that was originally investigated for potential use in the treatment of cancer but has not been commercially available.

Orphan Drug Designation provides opportunities for grant funding toward clinical-trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the U.S. following drug approval by the FDA. "We felt extremely proud when McGill received the FDA's Orphan Drug status for fenretinide. This is a very important step in our development and commercialization objectives for this truly innovative approach for the management of pulmonary infections in CF patients" said Dr. Mounia Azzi, Investment Manager at MSBi Valorisation, a partner organization.

"This Orphan Drug Designation is a critical, value-added step towards the development of fenretidine treatment for the benefit of CF patients" said Dr. Costas Karatzas, Director of Business Development at the Research Institute of the MUHC. Dr. Michle Beaulieu at McGill's Office of Sponsored Research stated that the next step will be a clinical trial to assess safety, tolerability and efficacy of fenretinide in CF patients, to be undertaken in collaboration with the Montreal Chest Institute.


'/>"/>

Contact: William Raillant-Clark
william.raillant-clark@mcgill.ca
514-398-2189
McGill University
Source:Eurekalert

Related medicine news :

1. NextAid Presents BIDS BUILD HOPE Online Auction to Benefit Projects on the Ground in Africa on World AIDS Orphans Day
2. Celebrity Event to Help Haitian Orphans Devastated by Earthquake
3. Foster care associated with improved growth, intelligence compared to orphanage care
4. International Collaboration: FDA and European Medicines Agency Agree to Accept a Single Orphan Drug Designation Annual Report
5. Aton Pharma Announces Michael H. Richardson to Head Orphan Drug Division
6. International Breast Milk Project Delivers Donor Breast Milk to Orphaned and Sick Babies in Haiti
7. Orphans International Worldwide Haiti Near Jacmel to Grow After Earthquake
8. Haitian Orphanage Receives BPA-Free Baby Bottle Donation from Glendale Company
9. Israel Flying Aid, Orange Israel Create Haiti Orphanage, Provide Humanitarian Aid
10. Haitian Orphanage Takes Quick Action to Fill Void after Devastating Earthquakes
11. BHR Pharma Files Investigational New Drug Application, Receives Orphan Drug Designation, for Traumatic Brain Injury Treatment
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:3/30/2017)... ... March 30, 2017 , ... AudioEducator, ... conference “ Preventing Hospital Readmissions Through Discharge Planning ” with noted expert Sue ... ET. This conference discusses strategies to prevent readmissions in light of the most ...
(Date:3/29/2017)... ... 29, 2017 , ... During the last week of March, Chad Kawa, MD ... individuals in the local community. , Colon cancer is the second leading cause ... cancer while it is small, confined and easier to treat. If you are 50 ...
(Date:3/29/2017)... San Diego, CA (PRWEB) , ... March 29, 2017 , ... ... meningiomas(1,2), and up to 300,000 people each year develop other types of metastatic brain ... spread quickly throughout the brain(3). As efforts focus on finding more effective treatment options, ...
(Date:3/29/2017)... ... March 29, 2017 , ... Full Contact K9, an Atlanta-based ... Pet Protect Law that assists dog owners in creating legally-enforceable pet trusts for ... taking the natural next step to protect their new companion. Says Evan Dunbar, CEO ...
(Date:3/29/2017)... ... March 30, 2017 , ... CHARM CITY RUN WELCOMES MERCY MEDICAL ... that Mercy Medical Center will serve as the official title sponsor of the Baltimore ... June 25, 2017, thousands of women will walk or run the course around the ...
Breaking Medicine News(10 mins):
(Date:3/30/2017)... March 30, 2017  Akcea Therapeutics, a subsidiary of ... of a Phase 2b dose-ranging study of AKCEA-APO(a)-L Rx ... The goal of the study is to determine the ... in a planned Phase 3 cardiovascular outcome study. ... Akcea,s strategic collaboration with Novartis to develop and commercialize ...
(Date:3/30/2017)... March 30, 2017  Purcell Julie & Lefkowitz LLP, a ... investigating a potential breach of fiduciary duty claim involving the ... SGYP ). If you are a shareholder ... regarding this investigation, free of charge, please visit us at: ... ...
(Date:3/29/2017)... 2017 Wound care devices and products are ... wound. The industry mainly consists of establishments engaged in ... of wounds caused by mechanical, chemical, thermal, and radiogenic ... as diabetes, skin related diseases, immunological diseases, and other ... was the largest region in the wound care devices ...
Breaking Medicine Technology: