The following article appeared on PharmaLive.com. I thought those of us with Hermansky-Pudlak Syndrome might be interested. I'm excited about it. To me, it just bolsters the case that Pirfenidone could be useful to us. And, hopefully, the research in Japan will only help us here as well.
Shionogi: Results of Phase III Clinical Trials of the Idiopathic Pulmonary Fibrosis Treatment S-7701
OSAKA, December 22, 2006 — Shionogi & Co., Ltd. (Head Office: Osaka; President: Motozo Shiono) today announced that it has achieved the primary objectives of Phase III clinical trials for the idiopathic pulmonary fibrosis treatment S-7701 (generic name: pirfenidone), which the Company is developing in Japan under a license from U.S.-based Marnac, Inc. and KDL, Inc., Tokyo.
Idiopathic pulmonary fibrosis (IPF) is a medical condition of unknown etiology with poor prognosis in which progressive fibrosis of the alveolar walls produces irreversible “honeycomb lung”*. In general, restrictive impairment (reduction of vital capacity (VC) and total lung capacity (TLC)) is evident. As the symptom (fibrosis of the alveolar walls) progresses, gas exchange in the lungs (exchange of oxygen and carbon dioxide) becomes difficult. In some cases, oxygen therapy becomes necessary. Because of its severity, IPF is designated as a “specified disease” (in other words, an intractable disorder).
Under development as a treatment for IPF, S-7701 has been designated as an orphan drug (a drug used to treat a rare disease) by the Pharmaceuticals and Medical Devices Agency.
Honeycomb lung: A high-resolution CT scan of the lung yields a honeycomb pattern. In the Phase III clinical trials for this drug with VC change (from before commencement of treatment to 52 weeks after commencing treatment) as the primary endpoint, both high and low doses of the drug (600mgl per day, three times a day and 400mgl per day, three times a day, respectively), significantly inhibited worsening of the condition compared with a placebo.
While continuing to conduct further analysis and study, Shionogi plans to expedite the application process based on these clinical results, with the intention of submitting a new drug application (NDA) within the current fiscal year.
In the process of developing this drug, Shionogi has received many earnest inquiries from patients with IPF and their families regarding the extent of progress of development, and urgent requests for the medication. With this situation in mind, Shionogi intends to do its best to expedite submission and approval for S-7701 in order to offer the quickest possible relief to patients suffering from this disease.
For further information, contact:Public Relations UnitShionogi & Co., Ltd.(Osaka)Tel: +81-6-6209-7885 Fax: +81-6-6229-9596(Tokyo)Tel: +81-3-3406-8164 Fax: +81-3-3406-8099.
Shionogi: Results of Phase III Clinical Trials of the Idiopathic Pulmonary Fibrosis Treatment S-7701
OSAKA, December 22, 2006 — Shionogi & Co., Ltd. (Head Office: Osaka; President: Motozo Shiono) today announced that it has achieved the primary objectives of Phase III clinical trials for the idiopathic pulmonary fibrosis treatment S-7701 (generic name: pirfenidone), which the Company is developing in Japan under a license from U.S.-based Marnac, Inc. and KDL, Inc., Tokyo.
Idiopathic pulmonary fibrosis (IPF) is a medical condition of unknown etiology with poor prognosis in which progressive fibrosis of the alveolar walls produces irreversible “honeycomb lung”*. In general, restrictive impairment (reduction of vital capacity (VC) and total lung capacity (TLC)) is evident. As the symptom (fibrosis of the alveolar walls) progresses, gas exchange in the lungs (exchange of oxygen and carbon dioxide) becomes difficult. In some cases, oxygen therapy becomes necessary. Because of its severity, IPF is designated as a “specified disease” (in other words, an intractable disorder).
Under development as a treatment for IPF, S-7701 has been designated as an orphan drug (a drug used to treat a rare disease) by the Pharmaceuticals and Medical Devices Agency.
Honeycomb lung: A high-resolution CT scan of the lung yields a honeycomb pattern. In the Phase III clinical trials for this drug with VC change (from before commencement of treatment to 52 weeks after commencing treatment) as the primary endpoint, both high and low doses of the drug (600mgl per day, three times a day and 400mgl per day, three times a day, respectively), significantly inhibited worsening of the condition compared with a placebo.
While continuing to conduct further analysis and study, Shionogi plans to expedite the application process based on these clinical results, with the intention of submitting a new drug application (NDA) within the current fiscal year.
In the process of developing this drug, Shionogi has received many earnest inquiries from patients with IPF and their families regarding the extent of progress of development, and urgent requests for the medication. With this situation in mind, Shionogi intends to do its best to expedite submission and approval for S-7701 in order to offer the quickest possible relief to patients suffering from this disease.
For further information, contact:Public Relations UnitShionogi & Co., Ltd.(Osaka)Tel: +81-6-6209-7885 Fax: +81-6-6229-9596(Tokyo)Tel: +81-3-3406-8164 Fax: +81-3-3406-8099.
Comments
Thanks for posting this. I found it very interesting. Yesterday, I got a booklet from my insurance company entitled How to Manage Your COPD. I realized that my doctor must be putting COPD as my diagnosis in the forms they submit to the insurance company. Since HPS is so rare they do this to simplify things, as COPD is more common. Should I do anything about this? Is COPD a good generalization?
I've got a friend who is the same age as I am and has COPD because of lupus. Actually, she has much more severe breathing problems than I do and is caring for three children under the age of seven. Makes me shiver!
Anyway, although our lung problems are not caused by the same thing, we find we have a lot of the same practical problems and can benefit from some of the same coping strategies. So, hey, read up on COPD and let me know if you find anything helpful!
But, COPD and pulmonary fibrosis, as I understood, are not the same thing. If you think your doc. is writing COPD in your chart and you think your problems are really fibrosis, might be worth a conversation to find out why. Yes, HPS probably isn't on their list of codes, sort of speak. But pulmonary fibrosis should be.
When I was at ATS (American Thoracic Society) I went to the symposium on COPD. The causes weren't the same, but I still could very much relate to some of the common symptoms they talked about.
COPD patients should not do hard work Anyway, although our lung problems are not caused by the same thing.What is the prevention of this desease.
George Kevin
Connecticut Drug Treatment
Can I get Pirfenidone from you here in the STATES
Name: Dr. M. Rashed.
e. mail: moustafa.rashed@yahoo.com
Phone # (U.S.A.): (954-392-8392