Duchenne muscular dystrophy gene therapy AMT-080 gets oraphan drug status in Europe: Amsterdam Molecular Therapeutics

AMT-080, an experimental gene therapy to treat Duchenne muscular dystrophy from Dutch biotech firm Amsterdam Molecular Therapeutics has been granted orphan drug designation by the European Medicines Agency (EMEA).

Orphan Drug Designation for Duchenne muscular dystrophy (DMD) entitles Amsterdam Molecular Therapeutics to ten year market exclusivity in Europe following marketing approval for AMT-080 if this product candidate is the first new drug with a major medical benefit receiving marketing approval for the European Union.

Orphan Drug Designation also provides for special benefits, including research support, eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees during development or at the time of application for marketing approval.

“This designation (for muscular dystrophy) is an important next step in the development of a treatment for this progressive and devastating disease,” AMT Chief Executive Jorn Aldag stated in a press release.

Duchenne muscular dystrophy (DMD) is a severe disease characterized by progressive muscle degeneration.

Duchenne muscular dystrophy (DMD) affects young children, almost exclusively boys, and leads to paralysis and death in young adulthood.

Duchenne muscular dystrophy (DMD) disease is caused by mutations in the dystrophin gene, thereby blocking the production of functional dystrophin protein, an important structural component within muscle tissue.

Currently there is no treatment to prevent the fatal outcome of Duchenne muscular dystrophy (DMD) disease.

Duchenne muscular dystrophy (DMD) affects one in 3,500 males, making it the most prevalent of muscular dystrophies.

Amsterdam Molecular Therapeutics is developing a gene therapy product for Duchenne muscluar dystrophy based on technology that results in “skipping” of the defective portion of the dystrophin gene that leads to the formation of functional protein.

Positive long-term health effects of this gene therapy approach have been demonstrated in animals.

Amsterdam Molecular Therapeutics has a gene therapy platform. Using adeno-associated viral (AAV) vectors as the delivery vehicle of choice for therapeutic genes, Amsterdam Molecular Therapeutics has been able to design and validate what is probably the first stable and scalable AAV production platform.

It is now possible to accomplish long-term expression of therapeutic genes in various organs (muscle, liver, eye, brain) using AAV-based delivery systems. AMT has demonstrated excellent efficacy of replacement of the lipoprotein lipase gene (LPL) in preclinical models. LPL deficiency in humans is a severe and debilitating disease that is associated with extremely high serum triglyceride concentrations, a high morbidity and increased mortality. LPL gene therapy entered its first clinical trial in Q4 2005. Other products are in late preclinical stages.

Amsterdam Molecular Therapeutics’ proprietary platform holds promise for rare (orphan), especially diseases that are caused by one faulty gene.

Currently, AMT has a product pipeline with nine products at different stages of development including Glybera for lipoprotein lipase deficiency hyperlipoproteinemia type V    and non-alcoholic steatotic hepatitis (NASH); AMT-060 for hemophilia B; AMT-090   to treat Parkinson’s disease; AMT-021 for acute intermittent porphyria; AMT-030 for primary hyperoxaluria;  AMT-050   for ApoA-1 deficiency and AMT-070 for liver cirrhosis apart from AMT-080    Duchenne muscular dystrophy.