New Drug (Nintedanib) Can Slow Pulmonary Fibrosis Progression

We at the Second Wind Foundation for Pulmonary Fibrosis, work to raise awareness about this rare but deadly disease.  We are also raising money to help fund research projects similar to this one below.  Come join us at our annual charity night with the Manchester Monarchs.  This year it will be on Feb 7th for “Pink in the Rink.”

Below is a press release from Boehringer Ingelheim a european drug manufacturer who is working on a drug that is showing real progress in slowing the progression of IPF in trials.

Nintedanib* receives positive CHMP opinion in
European Union for the treatment of IPF

Boehringer Ingelheim today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion for nintedanib* (suggested brand name OFEV®) for the treatment of patients with idiopathic pulmonary fibrosis (IPF). Results from the Phase III INPULSIS® trials published in the New England Journal of Medicine in May showed that nintedanib* significantly slowed disease progression in a broad range of patients with IPF**.1 The opinion comes after the recent Food and Drug Administration (FDA) approval of nintedanib* for the treatment of IPF.

“Boehringer Ingelheim welcomes the decision by the CHMP. There has been a high unmet need for effective treatments that can slow disease progression in IPF. We look forward to making nintedanib available soon to patients with IPF in the EU” said Professor Klaus Dugi Chief Medical Officer Boehringer Ingelheim.

IPF is a debilitating and fatal lung disease with a median survival of 2–3 years after diagnosis.2 It causes progressive scarring of the lungs resulting in continual and irreversible deterioration in lung function and difficulty breathing.3

“This decision is very encouraging as patients with IPF currently have very limited treatment options” said INPULSIS® study investigator Professor Luca Richeldi Professor of Respiratory Medicine Chair of Interstitial Lung Disease at the University of Southampton United Kingdom. “For the first time we have a drug that has consistently met the primary endpoint in two large Phase III trials confirming the results of the Phase II trial.”

The CHMP’s positive opinion is based on pivotal data from the replicate Phase III INPULSIS® trials involving 1066 patients from 24 countries. INPULSIS® results showed that nintedanib* slowed disease progression by reducing the annual rate of decline in lung function by 50% in a broad range of IPF patient types: this included patients with early disease (forced vital capacity (FVC) >90% pred) limited radiographic fibrosis (no honeycombing) on high resolution computed tomography (HRCT) and those with emphysema.1 As well nintedanib* significantly reduced the risk of adjudicated acute exacerbations by 68%.1 This can be crucial given that approximately 50% of patients hospitalised for an acute IPF exacerbation die during hospitalisation.4

Nintedanib* in IPF has been granted accelerated assessment by the EMA. Nintedanib* one capsule twice a day is the first treatment for IPF that has consistently demonstrated a significant reduction in the decline in lung function and has a manageable side effect profile.1 More than 90% of eligible patients who participated in the INPULSIS® trials opted to continue with nintedanib* treatment as part of an open-label extension trial.5

*Nintedanib is currently being assessed by the European Medicines Agency (EMA) and other regulatory organisations worldwide

**INPULSIS® recruited a broad range of patient types – similar to those seen in clinical practice including patients with early disease (FVC > 90% pred) no honeycombing on HRCT and/or concomitant emphysema

‡Adjudicated exacerbations’ was a pre-specified sensitivity analysis in the pooled data set. Time to first Investigator-reported exacerbation was a secondary endpoint which was met in TOMORROW and INPULSIS®-2 but not in INPULSIS®-1


Please click on the link below for ‘Notes to Editors’ and ‘References’:


New Inhaled “Pirfenidone” Treatment For PF Begins Trials In Early 2015

InhalerGenoa 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