The European Medicines Agency has granted Orphan Drug designation to AveXis’ gene transfer candidate. The therapy is currently in clinical development in the United States for the treatment of type 1 Spinal Muscular Atrophy and already received Orphan Drug designation for this disease in the United States.
Spinal Muscular Atrophy is an autosomal-recessive genetic disorder characterized by lower motor neuron loss and progressive muscle weakness. It is caused by a genetic defect in the gene that codes SMN, a protein necessary for the survival of motor neurons. This atrophy is the most common genetic cause of infant mortality and is the second-most common autosomal recessive genetic disorder.
To this aim, AveXis has developed ChariSMA, a type of viral gene therapy meaning that a viral vector will carry a particular gene – the transgene – into the cells of the body. The drug is about to be tested in a fully-enrolled Phase I clinical trial.
There are between 25,000 and 50,000 Spinal Muscular Atrophy patients in the United States,