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news.The US Food and Drug Administration (FDA) has designated orphan drug status to Amsterdam Molecular Therapeutics' (AMT) AMT-080, a gene therapy for Duchenne muscular dystrophy (DMD).
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AMT-080 has shown efficacy in studies of a preclinical model of DMD. The proof of concept studies demonstrated that AMT’s technology resulted in functional dystrophin synthesis in both the heart and skeletal muscles, leading to the prevention of muscular dystrophy.
The data are strengthened by a study in which this gene therapy approach was shown to successfully restore dystrophin activity in diseased human muscle cells obtained from biopsies of DMD patients. A Phase I/II clinical trial is scheduled to start by the end of 2012.
AMT has received an Innovation Credit of up to €4m from the Dutch government to support the development of AMT’s gene therapy treatment for Duchenne Muscular Dystrophy (DMD). The credit is granted by SenterNovem, an agency of the Dutch Ministry of Economic affairs.
AMT CEO JA rn Aldag said that AMT has successfully conducted three clinical trials with its lead product Glybera that employs this technology, confirming that AAV-based delivery technology is safe and efficacious.