News & Events
02/01/2009
Inside your lungs are millions of very small blood vessels called capillaries...
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Please mail photos, text, audio cassette or video as applicable to
PPH Cure Foundation
1735 Connecticut Ave., NW
3rd. Floor
Washington, DC 20009
 

Orphan Drug Development

During the past year the Foundation has become aware of the existence of a number of drug compounds that hold significant promise for managing PPH but which are not being made available to patients. These compounds are not in clinical trials for PPH because the size of the PPH community is too small to financially justify, for some large pharmaceutical companies, the multi-million dollar costs associated with clinical trials and regulatory approval.

Some of the compounds are long-acting oral forms (Schering's Iloprost and Toray's Beraprost) of the prostacyclin approved by the FDA for continuous intravenous use (Glaxo's Flolan). There are also other existing drug compounds that may arrest PPH by blocking certain endothelin receptors, by modulating the potassium ion channel in the vascular smooth muscle, and by inhibiting the formation of obliterative plexiform lesions in the pulmonary arterioles. These compounds are being developed for non-PPH conditions because those conditions affect much larger patient populations and thus offer greater promise of an acceptable financial plan.

The PPH Cure Foundation decided to deal with the challenge of developing existing medical compounds into approved medicines for PPH by encouraging the principal scientists behind Flolan to form a new biotech company focused on PPH and backed by a long-term contract from the Foundation. This new company, Lung Rx, Inc., was formed in August 1996 and is headed by Dr. James Crow, PhD, formerly of Glaxo-Wellcome. The mission of Lung Rx is to license from major pharmaceutical companies compounds that appear to have promise for arresting or reversing PPH, to conduct scientifically rigorous clinical trials to prove or disprove the safety and efficacy of these compounds, and to obtain regulatory approval to market the compounds to the international PPH community. We are hopeful that this new orphan drug company will expedite the clinical availability of PPH treatments.