Researchers at the Spanish National Research Council (CSIC) have found that the Bazedoxifene acetate, used to treat osteoporosis, is useful to treat a rare disease, the hereditary hemorrhagic telangiectasia, which causes bleedings and deteriorates the patient quality of life. The European Medicines Agency has granted an orphan designation to CSIC, which authorises to carry out clinical trials in order to commercialize it. An orphan drug treats low prevalence diseases (affecting 5 in 10.000 people) but has no investment from the pharmaceutical industry because it is not profitable.
The hereditary hemorrhagic telangiectasia (called HHT, or Osler-Weber-Rendu syndrome) is a rare disease affecting 2 in 10.000 people. "It is a genetic disease that causes nose bleedings, and red spots in the hands, face and mouth; and affects internal organs, with bleedings in the lungs, brain, liver and spinal cord", said the CSIC researcher María Luisa Botella, at the Biological Research Center (Centro de Investigaciones Biólogicas), who has led the research.
"It is not a fatal disease, but the symptoms lead to a reduction in a patient's quality of life. Due to the bleedings, the anemia and the necessity of blood transfusions are common", she adds.
Due to the low prevalence of the rare diseases and the special symptomatology, there are no therapeutical methods to treat them. Furthermore, in the pharmaceutical industry it is not profitable to invest in clinical trials for diseases of low prevalence. This is why the pharmaceutical industry called them "orphan".
There are two types of orphan drugs: the ones that are specifically developed to treat a rare disease, and those used in the treatment of a frequent disease that are also useful for a rare disease.
The designation is the result of the research led by the group of the Biological Research Center (Centro de Investigaciones Biológicas), at CSIC, and from the Hospital de Sierrallana (Cantabria). The Centre for Network Biomedical Research on Rare Diseases, the Asociación HHT España, and the Ministry of Economy and Competitiveness have contributed in the research.
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Researchers at the Spanish National Research Council (CSIC) have found that the Bazedoxifene acetate, used to treat osteoporosis, is useful to treat a rare disease, the hereditary hemorrhagic telangiectasia, which causes bleedings and deteriorates the patient quality of life. The European Medicines Agency has granted an orphan designation to CSIC, which authorises to carry out clinical trials in order to commercialize it. An orphan drug treats low prevalence diseases (affecting 5 in 10.000 people) but has no investment from the pharmaceutical industry because it is not profitable.
The hereditary hemorrhagic telangiectasia (called HHT, or Osler-Weber-Rendu syndrome) is a rare disease affecting 2 in 10.000 people. "It is a genetic disease that causes nose bleedings, and red spots in the hands, face and mouth; and affects internal organs, with bleedings in the lungs, brain, liver and spinal cord", said the CSIC researcher María Luisa Botella, at the Biological Research Center (Centro de Investigaciones Biólogicas), who has led the research.
"It is not a fatal disease, but the symptoms lead to a reduction in a patient's quality of life. Due to the bleedings, the anemia and the necessity of blood transfusions are common", she adds.
Due to the low prevalence of the rare diseases and the special symptomatology, there are no therapeutical methods to treat them. Furthermore, in the pharmaceutical industry it is not profitable to invest in clinical trials for diseases of low prevalence. This is why the pharmaceutical industry called them "orphan".
There are two types of orphan drugs: the ones that are specifically developed to treat a rare disease, and those used in the treatment of a frequent disease that are also useful for a rare disease.
The designation is the result of the research led by the group of the Biological Research Center (Centro de Investigaciones Biológicas), at CSIC, and from the Hospital de Sierrallana (Cantabria). The Centre for Network Biomedical Research on Rare Diseases, the Asociación HHT España, and the Ministry of Economy and Competitiveness have contributed in the research.
Explore further: Molecule for Fragile X Syndrome treatment receives orphan designation
Provided by Spanish National Research Council (CSIC)
Medical Xpress on facebook
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Molecule for Fragile X Syndrome treatment receives orphan designation
Nov 17, 2014
The European Medicines Agency (EMA) has granted "orphan designation" to BMS 204352, a molecule developed by the CNRS to treat Fragile X Syndrome, a rare genetic disease for which there exists no treatment. Protocol assistance ...
FDA drug approvals reached 18-year high in 2014
Jan 02, 2015
The Food and Drug Administration approved 41 first-of-a-kind drugs in 2014, including a record number of medicines for rare diseases, pushing the agency's annual tally of drug approvals to its highest level in 18 years.
Fast-tracking rare disease drug candidates' approval
Sep 03, 2014
Aspiring new drugs face long and stringent tests on safety and effectiveness before making it to market. And rightly so. But giving drugs special designations that bring with them the right incentives can help bring them ...
Drug for rare paroxysmal nocturnal hemoglobinuria receives FDA orphan status
Oct 28, 2014
A Penn Medicine-developed drug has received orphan status from the Food and Drug Administration (FDA) this month for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening disease that causes ...
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