AMT files for re-examination of Glybera
Dutch gene therapy specialist Amsterdam Molecular Therapeutics BV (AMT) has filed a re-examination request for its lead product Glybera. At the end of June, EMA's CHMP recommended against approval of the product to treat lipoprotein lipase deficiency. However, the biotech announced plans to generate additional data from existing patients to support the review process. The active substance in Glybera, alipogene tiparvovec, is a medicinal product using a virus that has been modified so it can carry a gene for lipoprotein lipase. It was expected to be injected into the patient’s muscles, where it would correct the deficiency of lipoprotein lipase by enabling the cells of the
muscle to produce the enzyme. AMT presented the results of three main studies in 27 patients with lipoprotein lipase deficiency on a low-fat diet. Some patients who received Glybera also received immunosuppressive treatment to reduce the reactions of the body’s immune system against medicine. The main measure of effectiveness was based on a reduction in blood fat levels, with patients being followed up for up to five years.
However, the CHMP considered that Glybera could be a promising treatment, the committee concluded that there was currently insufficient evidence of its benefits and safety to recommend approval at this stage. The CHMP fears the benefits of Glybera had not been sufficiently shown to outweigh the product's risks and recommended therefore that it be refused marketing authorisation. AMT will now file a data package containing new figures from a long term treatment. The Company expects that the re-examination of the dossier will be completed by the end of 2011.