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Executive Summary

PMA RARE DISEASE COMMISSION SEEKING SPONSORS FOR FIVE ORPHAN DRUGS: ethambutol for Menkes' Disease, dextran sulfate for treatment of cystic fibrosis, 3,4-diamino pyridine for Lambert Eaton syndrome, sodium dichloroacetate for congenital lactic acidosis, and L-cycloserine for Gaucher's disease, the organization announced Aug. 1. Three of the drugs have previously been marketed for other indications and two are available for license from academic research facilities. Four of the indications for which the current products are being proposed are considered very rare: Menkes' Disease, a syndrome of impairments to neurological development and copper metabolism that leads to early childhood death; Lambert Eaton myasthenic syndrome, a neurological disorder that induces muscular debility; congenital lactic acidosis, a condition causing excessive lactic acid in the bloodstream; and Gaucher's disease, the genetic disorder of the spleen for which Genzyme's Ceredase was recently approved. Cystic fibrosis, the potentially fatal pancreatic disease, is a more common but still an orphan indication (patient population less than 200,000). The oral chemotherapeutic ethambutol, formerly marketed by Lederle for pulmonary tuberculosis, in addition to its Menkes orphan possibilities has recently been studied by CDC and FDA researchers as a potential weapon against mycobacterium avium in AIDS ("The Pink Sheet," April 4, 1990, T&G-2). L-cycloserine, an anti-tuberculosis therapy from the 1950s, is believed by researchers to work against Gaucher's disease by a different mechanism than Ceredase, a drug with an annual treatment pricetag that ranges from $ 20,000 to $ 60,000 per year. Dextran sulfate, also off-patent, has been popular among the AIDS underground of unapproved therapies, although initial FDA clinical studies failed to find promise for an AIDS indication ("The Pink Sheet," Feb. 27, 1989, T&G-4). The rights to 3,4-diamino pyridine are available for license from the Mayo Clinic, where researchers said earlier this year that the compound was exhibiting dramatic effects in patients with the Lambert Eaton disorder, according to PMA. The University of Florida-Gainesville holds the rights to sodium dichloracetate, which PMA notes also holds potential against homozygous familial hypercholesterolemia, a condition inducing elevated blood cholesterol levels. PMA's Commission on Drugs for Rare Diseases collects and reviews suggestions and data on products with potential for treating rare illnesses that lack backers for commercial development, then attempts to bring orphan researchers and products together with potential sponsors. The commission also attempts to ascertain that marketing rights are available for the compounds. Potential sponsors need not be PMA members in order to participate.

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