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:6/26/2016)... June 27, 2016 Jazz Pharmaceuticals plc (Nasdaq: ... under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as ... Celator Pharmaceuticals, Inc. ("Celator"; Nasdaq: CPXX ) ... Daylight Time). As previously announced on May ... definitive merger agreement under which Jazz Pharmaceuticals has commenced ...
(Date:6/24/2016)... SAN FRANCISCO, Calif. , June 24, 2016 ... GBT ), a biopharmaceutical company developing novel therapeutics ... significant unmet needs, today announced the closing of ... shares of common stock, at the public offering ... shares in the offering were offered by GBT. ...
(Date:6/24/2016)... 2016 Dehaier Medical Systems Ltd. (NASDAQ: ... markets and sells medical devices and wearable sleep respiratory ... strategic cooperation agreement with Hongyuan Supply Chain Management Co., ... June 20, 2016, to develop Dehaier,s new Internet medical ... Dehaier will leverage Hongyuan Supply Chain,s sales platform to ...
Breaking Medicine Technology:
(Date:6/27/2016)... ... June 27, 2016 , ... A revolution ... the emergency ambulance transport experience for the millions of people who require these ... disrupted the taxi industry through the use of technology. Now, SmartEMS has put ...
(Date:6/26/2016)... ... ... Quality metrics are proliferating in cancer care, and are derived from many ... the beholder, according to experts who offered insights and commentary in the current issue ... the full issue, click here . , For the American Society of Clinical ...
(Date:6/26/2016)... ... June 26, 2016 , ... Pixel Film Studios Released ProSlice Levels, a ... can give their videos a whole new perspective by using the title layers ... Film Studios. , ProSlice Levels contains over 30 Different presets to choose from. ...
(Date:6/26/2016)... ... , ... Brent Kasmer, a legally blind and certified personal trainer is helping to develop a ... fitness app plans to fix the two major problems leading the fitness industry today:, ... type program , They don’t eliminate all the reasons people quit their exercise ...
(Date:6/25/2016)... ... June 25, 2016 , ... Experts from the American Institutes ... Research Meeting June 26-28, 2016, at the Hynes Convention Center in Boston. , ... advance care planning, healthcare costs and patient and family engagement. , AIR researchers ...
Breaking Medicine News(10 mins):