According to a story from kait8.com, the drug development company Asceneuron SA recently announced that its experimental molecule ASN120290 was recently granted Orphan Drug Designation by the FDA. ASN120290 is in development for the treatment of progressive supranuclear palsy. Asceneuron is focused on the development of medications for the treatment of neurodegenerative illnesses.
About Progressive Supranuclear Palsy
Progressive supranuclear palsy (PSP), which is also known as Steele-Richardson-Olszewski syndrome, is a late onset degenerative disease that causes certain areas of the brain to deteriorate and decrease in volume. The cause of the disease is still a mystery. The disease does not appear to be heritable, as less than one percent of patients have a family member with the illness. Some environmental factors may play a role. Symptoms include poor balance, difficulty walking, falling, backward head tilt from stiffened neck muscles, slurred speech, difficulty swallowing, dementia, and urinary incontinence. Difficulty with eye movement is a diagnostic symptom that can help doctors identify progressive supranuclear palsy easily. Many patients have trouble moving their eyes up and down. Treatment for the disease is mostly supportive, as there is no known cure and no medication specifically approved for it. Average survival time after diagnosis is seven years. To learn more about progressive supranuclear palsy, click here.
Orphan Drug Designation
Orphan Drug designation is reserved for those treatments that are designed for treating a rare disease, which is defined as any illness that affects 200,000 people or less in the US. In addition, the medication must satisfy a currently unmet medical need or demonstrate the potential to offer significant advantages over currently available treatments. The designation provides incentives for companies that develop rare disease drugs, such as the waiving of certain fees, tax breaks, and a period of seven year market exclusivity if the treatment gains approval.
ASN120290 has shown significant potential in preclinical studies. The molecule was able to break up concentrations of tau protein, which builds up into neurofibrillary tangles. These aggregates of tau play a major role in several neurodegenerative illnesses and dementia. This suggests that ASN120290 could also be a potential treatment for other illnesses beyond just progressive supranuclear palsy.