The COMP recommended that the orphan designation of the medicine be maintained 1.ġThe maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. The Committee looked at the seriousness and prevalence of the condition, and the existence of other methods of treatment. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. ![]() On 8 April 2016, the Committee for Orphan Medicinal Products (COMP) completed its review of the designation EU/3/05/313 for Strimvelis (autologous CD34+ cells transfected with retroviral vector containing adenosine deaminase gene) as an orphan medicinal product for the treatment of severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). Severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency is chronically debilitating and life-threatening. Because children with SCID experience multiple infections, they fail to grow and to gain weight as expected. The symptoms of this type of SCID include an increased susceptibility to a variety of infections, including ear infections, lung infections and diarrhea. This deficiency usually results in the onset of one or more serious infections within the first few months of life. As a consequence of the adenosine deaminase (ADA) deficiency, lymphocytes, which proliferate greatly during their maturation, are injured by these accumulation of toxic metabolites. This enzyme is important in every cell of the body but in particular in those cells which proliferate rapidly, like the lymphocytes. ![]() A form of SCID is caused by a lack of adenosine deaminase (ADA), an enzyme (a protein that speeds up the conversion of certain substances into other substances) which helps the cell to clear the waste products it generates during proliferation. Severe combined immunodeficiency, or SCID, is a group of inherited disorders characterized by little or no body's defence (immune) response due to the total or partial lack of those specilised white cells (lymphocytes) which are normally part of the body's defense system.
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