Navigation Links
Genoa Pharmaceuticals Receives Orphan-Drug Designation for Pirfenidone in the Inhaled Treatment of Idiopathic Pulmonary Fibrosis (IPF)

SAN DIEGO, Aug. 5, 2014 /PRNewswire/ -- Genoa Pharmaceuticals, the leader in inhaled medicines for pulmonary fibrosis, today announced the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation to Genoa for the use of pirfenidone in their lead program - inhaled GP-101 for the treatment of IPF.

"Acquiring orphan status marks an important regulatory milestone in GP-101's life cycle to treat people with this devastating disease," said Mark Surber, Ph.D., Genoa's President and Chief Executive Officer. "We are pleased to continue the development of inhaled GP-101, with clinical trials beginning in early 2015."

Oral pirfenidone (Esbriet®) has shown promise to slow IPF disease progression. Unfortunately, a very large oral dose is required to achieve efficacious lung levels. Despite being established at the upper safety threshold (801 mg TID), the resulting oral-delivered lung dose is too low for optimal effect. Moreover, gastrointestinal exposure and large-associated blood levels remain poorly tolerated. For these reasons oral-dose escalation for optimal IPF efficacy is not possible. Complicating matters, dose-absorbing food, first-pass metabolism, and safety-driven dose-reduction and stoppage protocols further reduce lung dose and interrupt maintenance therapy. 

To address oral shortcomings and maximize IPF efficacy, Genoa has reformulated pirfenidone for aerosol formation and inhaled, direct-lung delivery (GP-101).  By this approach, ~160-fold less inhaled pirfenidone is predicted to deliver Esbriet-equivalent IPF efficacy (5 mg vs. 801 mg). With such a small inhaled dose, remaining safety and tolerability concerns may be eliminated, enabling improved patient compliance and an increased inhaled dose for superior IPF efficacy. In addition to serving as an improved-effect Esbriet replacement, a safe and well-tolerated inhaled product is expected to enable desired, but otherwise poorly-tolerated combination regimens (e.g., with Boehringer Ingelheim's Nintedanib).

About Orphan Drug Designation

Orphan drug designation is a status assigned to a medicine intended for use in rare diseases. In the U.S., the Orphan Drug Designation program provides orphan status to medicines intended for the safe and effective treatment or prevention of rare diseases that affect fewer than 200,000 people. In the E.U., a medicine must meet similar criteria, affecting up to five in 10,000 people. Orphan designation for inhaled GP-101 will be pursued in the E.U. with clinical data.  Orphan status provides sponsors with development and commercial incentives, including 7 and 10 years market exclusivity for these two regions, respectively.

About IPF

IPF is a fatal lung disease caused by both genetic and environmental factors resulting in progressive lung scarring and death due to respiratory failure and/or co-morbidities.  Characterized by a dry cough, shortness of breath and decreased exercise capacity, this disease exhibits a post-diagnosis survival period of ~2-5 years, annually killing more people than breast cancer. As fibrosis is at present irreversible, an efficacious product will provide a well-tolerated stand-alone or combination maintenance therapy that protects healthy lung tissue against invading fibrosis or meaningfully slows disease progression.

About Genoa Pharmaceuticals

Genoa Pharmaceuticals, Inc. is committed to developing improved therapies for the treatment of IPF. Based in San Diego, Genoa's lead program, GP-101 (aerosol pirfenidone) plans to enter clinical trials in early 2015. Learn more at

SOURCE Genoa Pharmaceuticals
Copyright©2014 PR Newswire.
All rights reserved

Related medicine technology :

1. Genoa Pharmaceuticals announces $1.2M Financing
2. Jazz Pharmaceuticals Announces Participation at Canaccord Genuity Conference on August 13
3. Lemelson Capital Management Further Increases Short Position in Ligand Pharmaceuticals (NASDAQ: LGND), Updates LGND Research Report with New Material Information
4. Rock Creek Pharmaceuticals to Present at the Annual BioPharm America International Partnering Conference
5. Pharmaceuticals Learning the Language of the New Healthcare Landscape
6. Valeant Pharmaceuticals And Pershing Square Respond To Frivolous Allergan Lawsuit
7. Isis Pharmaceuticals Initiates Phase 3 Study of ISIS-SMN Rx in Infants with Spinal Muscular Atrophy
8. Inovio Pharmaceuticals to Host Second Quarter 2014 Financial Results Call on August 11, 2014
9. Arena Pharmaceuticals Provides Corporate Update and Reports Second Quarter 2014 Financial Results
10. Auxilium Pharmaceuticals, Inc. To Announce Second Quarter 2014 Results On August 7, 2014
11. Rock Creek Pharmaceuticals Provides Update of its European Drug Development Regulatory Strategy and selects Quotient Clinical for its early development programs
Post Your Comments:
(Date:11/24/2015)... Nov. 24, 2015  Ascendant Solutions, Inc. (Pink Sheets: ... Board of Directors has declared a special 1 percent stock ... is payable December 14, 2015, to shareholders of record December ... of additional shares of common stock. ... is a strong endorsement of our confidence in Ascendant,s growth ...
(Date:11/24/2015)... st  Scientific Assembly and Annual Meeting of ... taking place in Chicago on Nov-29 th ... --> st  Scientific Assembly and Annual Meeting of the Radiological ... in Chicago on Nov-29 th through ... Molecular Dynamics will present its revolutionary whole body CZT digital SPECT/CT ...
(Date:11/24/2015)... November 24, 2015 --> ... Inhibitors, MTTP Inhibitors, ApoB Inhibitors and PPAR Agonists ... to grow at the fastest rates? This visiongain ... data, trends, opportunities and prospects there. ,  ,Our 199-page ... most lucrative areas in the industry and the future ...
Breaking Medicine Technology:
(Date:11/24/2015)... ... November 24, 2015 , ... All her life, Don Peck’s mother wondered if she was ... she might have been. After a 25-year search for information, Don and his aunt ... shared a last name. Turns out, it was Don’s father who was descended from ...
(Date:11/24/2015)... Pass, OR (PRWEB) , ... November 24, 2015 , ... ... Lose too much body water to perspiration in the hot sun, and heat stroke ... to Fresh water advocate and radio host Sharon Kleyne. Every cell, system and structure ...
(Date:11/24/2015)... ... 2015 , ... The OSHA Training Center at Chabot-Las ... in Northern California, is calling on retail employers to implement safety measures to ... volume of sales and shoppers rapidly expands, retailers are strongly encouraged to develop ...
(Date:11/24/2015)... Miami, Fl (PRWEB) , ... November 24, 2015 , ... ... Farms. Adding houseplants is an easy and affordable way to bring long-lasting style and ... “green heroes” also provide oxygen, clean the air and keep on giving all year ...
(Date:11/24/2015)... (PRWEB) , ... November 24, 2015 , ... Take the audience on a journey with ... 3D camera path through a cloud of images without ever setting a single keyframe in ... the Inspector Window. Then use the controls to change the angle and depth of field ...
Breaking Medicine News(10 mins):