BIIB092 for progressive supranuclear palsy
BIIB092 is a product that is being investigated for the treatment of progressive supranuclear palsy (PSP). PSP is a rare condition that is a result of destruction of nerve cells in certain parts of the brain causing problems with balance, movement, vision, speech and swallowing. In patients with PSP, an abnormal form of a protein called tau accumulates in specific areas of the brain by spreading from brain cell to brain cell leading to their damage. Over time, PSP gets progressively worse, with people becoming severely disabled within three to five years of onset. Currently, there is no cure for PSP and no treatment to slow down the disease.
Selumetinib for neurofibromatosis type 1
Selumetinib is in clinical development for children with neurofibromatosis type 1 (NF1), also called von Recklinghausen’s disease. NF1 is a rare genetic disorder characterized by the development of multiple benign tumours of nerves and skin and areas of abnormal skin colour. NF1 is caused by mutation in a gene that regulates the production of a …
Edaravone for amyotrophic lateral sclerosis
Edaravone as an intravenous injection is in clinical development for people with amyotrophic lateral sclerosis (ALS). ALS is a neurological condition that affects nerve cells in the brain and
spinal cord. It results in gradual weakness and wasting of muscles of the body. Respiratory muscles are involved as the disease progresses, leading to shortness of breath and ultimately
death. Little is known about the cause of the disease, and there is currently no cure.
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.