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"The FDA's grant of orphan drug designation to Terguride for PAHencourages and strengthens our development program by offering regulatory,clinical development and commercial benefits," said Dr Rudolf Reiter, CEO ofErgonex Pharma. "We believe that Terguride can provide a significanttherapeutic benefit in PAH by inhibiting excess serotonin signalling."
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The U.S. Orphan Drug Act is intended to assist and encourage companies todevelop safe and effective therapies for the treatment of rare diseases,which affect 200.000 persons or less in the United States. Under the OrphanDrug Act, upon marketing authorization, the FDA does not accept or approveother applications to market the same medicinal product for the sameindication for a seven-year period. In addition to potential marketexclusivity, orphan drug designation provides protocol assistance, advice onthe conduct of clinical trials, tax credits for clinical research expenses,grant funding for research of rare disease treatments and waiver of thePrescription Drug User Fee Act filing fee.
About Terguride
Terguride has strong anti-serotoninergic activity by acting as a potentantagonist on 5-HT2B and 5-HT2A receptors: It has anti-proliferative andanti-fibrotic activity and drives reverse remodelling processes. Serotonin isa signal molecule in the body with many functions. In the blood vessel wallsof the lung it stimulates proliferation of smooth muscle cells and narrowingof the blood vessels, which leads to PAH. Excessive proliferative effects ofserotonin on the heart contribute directly to progression towards heartfailure.
Terguride is clinically approved in Japan for hyperprolactinemia actingas partial dopamine agonist on the pituitary.
About Pulmonary arterial hypertension
Pulmonary arterial hypertension is a disorder of the blood vessels in thelung, in which the pressure in large blood vessel rises above normal. Wallsof the blood vessels are thickened and hardened, becoming less elastic anddecrease in lumen leading to increases the pressure. Patients with PAH sufferfrom extreme shortness of breath as the heart struggles to pump against thesehigh pressures causing such patients to ultimately die of heart failure. PAHcan occur with no known underlying cause, or it can occur secondary todiseases such as connective tissue disease, congenital heart defects,cirrhosis of the liver and HIV infection.
About Ergonex Pharma
Ergonex Pharma is a pharmaceutical company focussed on developing andcommercialising well-tolerated and effective products for novel and typicallyunderserved indications. This is being achieved by forging collaborationswith commercial and academic partners with expertise in the field of interestand through outsourcing activities to service providers. (For furtherinformation see http://www.ergonex.com)Contact: Dr. Rudolf Reiter Ergonex Pharma GmbH Ruetistr. 20 9050 Appenzell Switzerland Tel.: +41(0)71-788-40-65 E-Mail: [email protected]
SOURCE Ergonex Pharma GmbH
