APPENZELL, SWITZERLAND, May 15 /CNW/ - Ergonex Pharma today announced
that the United States Food and Drug Administration (FDA) has granted orphan
drug designation to Terguride for the treatment of pulmonary arterial
hypertension (PAH). Terguride is currently being evaluated for PAH in a
pivotal Phase II trial in Europe.
"The FDA's grant of orphan drug designation to Terguride for PAH
encourages and strengthens our development program by offering regulatory,
clinical development and commercial benefits," said Dr Rudolf Reiter, CEO of
Ergonex Pharma. "We believe that Terguride can provide a significant
therapeutic benefit in PAH by inhibiting excess serotonin signalling."
The U.S. Orphan Drug Act is intended to assist and encourage companies to
develop safe and effective therapies for the treatment of rare diseases, which
affect 200.000 persons or less in the United States. Under the Orphan Drug
Act, upon marketing authorization, the FDA does not accept or approve other
applications to market the same medicinal product for the same indication for
a seven-year period. In addition to potential market exclusivity, orphan drug
designation provides protocol assistance, advice on the conduct of clinical
trials, tax credits for clinical research expenses, grant funding for research
of rare disease treatments and waiver of the Prescription Drug User Fee Act
filing fee.
About Terguride
Terguride has strong anti-serotoninergic activity by acting as a potent
antagonist on 5-HT2B and 5-HT2A receptors: It has anti-proliferative and
anti-fibrotic activity and drives reverse remodelling processes.
Serotonin is a signal molecule in the body with many functions. In the
blood vessel walls of the lung it stimulates proliferation of smooth muscle
cells and narrowing of the blood vessels, which leads to PAH. Excessive
proliferative effects of serotonin on the heart contribute directly to
progression towards heart failure.
Terguride is clinically approved in Japan for hyperprolactinemia acting
as partial dopamine agonist on the pituitary.
About Pulmonary arterial hypertension
Pulmonary arterial hypertension is a disorder of the blood vessels in the
lung, in which the pressure in large blood vessel rises above normal. Walls of
the blood vessels are thickened and hardened, becoming less elastic and
decrease in lumen leading to increases of pressure. Patients with PAH suffer
from extreme shortness of breath as the heart struggles to pump against these
high pressures causing such patients to ultimately die of heart failure. PAH
can occur with no known underlying cause, or it can occur secondary to
diseases 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 and
commercialising well-tolerated and effective products for novel and typically
underserved indications. This is being achieved by forging collaborations with
commercial and academic partners with expertise in the field of interest and
through outsourcing activities to service providers. (For further information
see http://www.ergonex.com)
For further information: Dr. Rudolf Reiter, Ergonex Pharma GmbH,
Ruetistr. 20, 9050 Appenzell, Switzerland, Tel.: +41(0)71-788-40-65, E-Mail:
info@ergonex.com
