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The FDA designates “lentiviral vector containing human liver and erythroid pyruvate kinase (PKLR) gene” as new Orphan Drug for the PKD treatment

The orphan drugs designation by the FDA, together with the one achieved by the European Medicines Agency in 2014, will allow promoters to get benefits for the development of this lentiviral vector to facilitate the approval for its commercialization

May 9, 2016

The Food and Drug Administration (FDA), the govern agency of United States responsible for the drugs regulations, has designated “lentiviral vector containing human liver and erythroid pyruvate kinase (PKLR) gene” as new Orphan Drug for the PKD treatment, a rare anaemia caused by mutations in the gene that codifies the pyruvate kinase enzyme, which impairs directly the metabolic energy of red cells and results in the reduced lifespan and early breakdown of erythrocytes in the blood. This drug is based on a lentiviral vector that will deliver the wild type PKLR gene into immature bone marrow cells, leading to the genetic correction of hematopoietic stem cells through gene therapy. Once the genetic defect has been corrected, the cells will be reinfused into the patients, being able to produce cells with the enzyme. As the genetic correction is permanent, patients will be definitely cured.

The orphan drugs designation by the FDA, together with the one achieved by the European Medicines Agency in 2014, will allow promoters to get benefits for the development of this lentiviral vector to facilitate the approval for its commercialization. Similar strategies have been taken successfully with other genetic disorders such as some primary immunodeficiencies. Lentiviral vectors are safer and more efficient than the vectors used in initial trials of gene therapy.

The project is directed by Dr. José Carlos Segovia, researcher from U710 CIBERER that is lead by Dr. Juan Antonio Bueren, and funded by different institutions such as European Commission (7º Framework Programme), como la Comisión Europea (7º Programa Marco), the Ministry of Economy and Competitivity (National Research Programs), the Health Institute “Carlos III” (Tematic Networks in Cooperative Research and CIBERER) and the Botin Foundation and the Health Research Institute “Fundación Jiménez Díaz". Furthermore, this designation constitutes the first FDA Orphan Drug  obtained by the Division of Hematopoietic Innovative Therapies of the CIEMAT/CIBERER/IIS-FJD (Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas-Centro de Investigación Biomédica en Red de Enfermedades Raras and Instituo de Investigación Sanitaria-Fundación Jiménez Díaz), highlighting the huge contribution to translational progress of these institutions.

The successful Orphan Drug designations are a key step forward towards achieving a clinically efficient and safe gene therapy clinical trial opening new potential therapeutic options.
 

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