Wund(er)heilung mit Amnion – DGFG erhält deutschen Wundpreis 2021
Ausschreibung DGNI-Pflege- und Therapiepreis 2022
Ausschreibung: Otsuka Team Award Psychiatry+ 2021
BGW-Gesundheitspreis 2022: Gute Praxis aus der Altenpflege gesucht!
Aktionsbündnis Patientensicherheit vergibt Deutschen Preis für Patientensicherheit 2021 an herausragende…
8.-10. September 2021: Weimar Sepsis Update 2021 – Beyond the…
13.09. – 18.09.2021: Viszeralmedizin 2021
24.06. – 26.06.2021: 27. Jahrestagung der Deutschen Gesellschaft für Radioonkologie
17.06. – 19.06.2021: 47. Jahrestagung der Gesellschaft für Neonatologie und…
16. bis 18. Juni 2021: Deutscher Kongress für Psychosomatische Medizin…
Committee for Orphan Medicinal Products (COMP) May 2012 highlights
London, UK (May 22, 2012) – During its May 2012 meeting, the Committee for Orphan Medicinal Products (COMP) adopted a total of 16 recommendations for orphan designation. Orphan designation can be granted early in the development process to substances that are intended for diagnosis, prevention or treatment of life-threatening or very serious conditions that affect not more than 5 in 10,000 people in the European Union (EU) or that would not be developed without incentives.
One of these diseases for which no adequate treatment currently exists is infection-associated haemolytic uraemic syndrome (HUS). During its meeting, the COMP recommended granting an orphan designation for eculizumab used for the treatment of this severe, life-threatening complication of enteric infections with Shiga toxin-producing Escherichia coli bacteria, such as enterohaemorrhagic E. coli, also called EHEC.
Infection-associated HUS occurs predominantly in childhood. Although it is a very rare disease, estimated to occur in less than 0.07 per 10.000 patients a year, infection-associated HUS is recognised as the most common cause of acute kidney failure in infants and young children. It can also affect adults and the elderly, who often die as a result of the disease.
Eculizumab, a monoclonal antibody, is already authorised in the EU for the treatment of paroxysmal nocturnal haemoglobinuria, a rare, life-threatening genetic disease that causes the red blood cells to be broken down too quickly. Its mechanism of action is thought to be also effective in patients with infection-associated HUS.
The COMP also recommended granting of an orphan designation for recombinant human interleukin-7 for the treatment of progressive multifocal leukoencephalopathy (PML). PML is a severe demyelinating disease of the central nervous system caused by the JC virus. It is seen in many conditions that affect immune system function such as HIV-1/AIDS, certain cancers and organ transplant subject to immunosuppressive therapies. Over the last five to 10 years, it has been increasingly reported as a serious adverse event of therapeutic monoclonal antibodies that modulate the immune system.
PML is a very rare disease, affecting less than 4 out of a million patients. There are currently no medicines available to treat PML or to prevent JC virus infection. The granting of an orphan designation is intended to encourage the development of a medicine for the treatment of this disease, which often leads to death or serious disability.
For the full list of opinions adopted by the COMP during its May 2012 meeting, see the COMP monthly report (http://www.ema.europa.eu/ema/pages/includes/document/open_document.jsp?webContentId=WC500127595).
The COMP recommendations for these substances are sent to the European Commission for the adoption of a decision. Once an orphan designation has been granted, sponsors have access to certain incentives during the development of the medicine.
For each medicine that has been granted an orphan designation, the Agency publishes a summary of the opinion on its website.
European Medicines Agency (EMA), 22.05.2012 (tB).