On 12 June 2009, orphan designation (EU/3/09/643) was granted by the European Commission to Targeon SAS, France, for desipramine chlorhydrate for the treatment of Rett syndrome.
Desipramine chlorhydrate has been used in the treatment of depression since the 1960s. In Rett syndrome, the medicine is expected to treat breathing problems.
Girls with Rett syndrome tend to have low levels of norepinephrine, a substance in the brain and spinal cord that helps maintain a normal breathing rhythm. Desipramine chlorhydrate is a ‘norepinephrine re-uptake inhibitor’. This means that it works by increasing the levels of norepinephrine in the spaces between nerve cells in the brain and spinal cord. This is expected to restore normal breathing rhythm in girls with Rett syndrome.
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