Inebilizumab for Neuromyelitis optica
Inebilizumab is a humanised monoclonal antibody that is in clinical development for reducing the risk of an attack in patients with Neuromyelitis Optica Spectrum Disorders (NMOSD). NMOSD, previously known as NMO and as Devic’s disease, is a rare, disabling autoimmune disease of the central nervous system. It predominantly affects the optic nerve and spinal cord, …
Sarizotan for Respiratory Symptoms Associated Rett Syndrome
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 for Partial Focal Epilepsy – Adjunctive Therapy
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.
AVXS-101 for spinal muscular atrophy
AVXS-101 is a gene replacement therapy, made of a virus that has been modified to contain the primary gene for the SMN protein, which is lacking (or mutated) in patients with SMA. When injected into the patient, the virus is expected to carry the gene into the nerve cells, enabling them to start producing sufficient amounts of SMN. This is expected to improve the survival and function of the motor neurons, and so preserve muscle function. AVXS-101 is thought to address the root cause of SMA and therefore, if licensed, may offer an additional treatment option for patients with spinal muscular atrophy.
Pentetrazol for Idiopathic Hypersomnia and Narcolepsy Type 2
Pentetrazol is a medicinal product that is being developed for the treatment of IH and narcolepsy type 2. Pentetrazol is administered orally and it acts by blocking the effect of a chemical substance called Gamma-Aminobutyric Acid (GABA). GABA is thought to play a role in promoting sleeping and is believed to be elevated in people with IH. If licensed, Pentetrazol will offer a new treatment option for patients with IH or narcolepsy type 2.
Perampanel (Fycompa) for Paediatric Epilepsy
Epilepsy is a condition in which the brain is affected by abnormal discharge of electrical activity, causing seizures (fits). There are several different types of epileptic seizures, depending on what part of the brain they start in and which part they affect. Epilepsy can start in any age, but is most common in either childhood …
PXT3003 for Charcot-Marie-Tooth Disease Type 1A
Charcot-Marie-Tooth disease type 1A (CMT1A) is an inherited genetic disease in which the motor and/or sensory peripheral nerves are affected, resulting in muscle weakness and wasting, as well as sensory loss.
Ronopterin for Moderate to Severe Traumatic Brain Injury
Ronopterin is being developed as a continuous infusion that can be used individually or in combination with the current standard treatments for traumatic brain injury. The current medications available are only used to treat the symptoms of traumatic brain injury.