A treatment for Spinal Muscular Atrophy received Orphan Drug designation


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, Europe and Japan combined, which makes ChariSMA, AveXis’ candidate, a perfect match for the Orphan Drug designation. With this designation, the European Medicines Agency intends to accelerate ChariSMA’s way to patients.

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