Rett syndrome (RTT) is a genetic disease that is caused by abnormalities in a gene called MECP2 which is important for the normal functioning of nerve cells. It is a rare disease which almost always affects females. Females with RTT lose their ability to properly control their muscles, have feeding difficulties and learning disabilities. Other symptoms include difficulty breathing, irregular heartbeat, sleeping problems, constipation, repetitive hand movements and fits.
Sarizotan is an oral medicinal product that is being developed for the treatment of respiratory symptoms associated with Rett syndrome. Sarizotan works by binding to serotonin and dopamine receptors. By stimulating serotonin and dopamine receptors, sarizotan replaces the effect of some of the missing serotonin in the brain and spinal cord. This is expected to help restore normal breathing rhythm in patients with Rett syndrome. Currently, there is no cure for Rett syndrome and treatment focuses on managing the symptoms. If licensed, sarizotan could become the first therapy approved for treatment of Rett Syndrome patients.
Cenobamate is a medicinal product that is being developed as a therapy for patients with partial focal epilepsy that would be taken in addition to other anti-epileptic medicine (adjunctive therapy). It is given as capsules. Cenobamate is considered a new generation antiepileptic therapy and clinical trials have shown that it may be more effective and safer than existing drugs. If licensed, cenobamate will offer a new adjunctive treatment option for patients with partial focal epilepsy.