Spinocerebellar ataxias (SCAs) are a group of rare, progressive hereditary genetic disorders that affects the cerebellum, brain stem and spinal cord. Currently, more than 30 types of SCAs have been identified. SCAs are caused by genetic mutations, which lead to faulty proteins being produced. This causes the degeneration of different populations of neurons, depending on the type of SCA.
For more information about different SCAs, please visit: https://www.ataxia.org.uk/about-ataxia-uk/types-of-ataxia/ and https://scasource.net/what-is-ataxia/
VICO Therapeutics is a company focusing on the development of therapies for rare neurological disorders. VICO’s product, SCA therapy VO659, is designed to suppress faulty proteins that cause SCAs, thereby slowing or halting disease progression.
In February 2021, VICO Therapeutics announced that the European Commission (EC) had granted orphan drug designation for VO659.
Qualified for orphan drug designation are drugs intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that impact fewer than 5 in 10,000 patients in the European Union. Orphan drug designation gives developing companies certain benefits.
In June 2021, VICO Therapeutics announced that the Office of Orphan Products Development (OOPD) of the U.S. Food and Drug Administration (FDA) had granted orphan-drug designation for VO659.
FDA’s Office of Orphan Products Development grants Orphan Drug Designation to drugs that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. The designation allows VICO Therapeutics to qualify for incentives.
VICO Therapeutics are currently preparing for their first in human trials of VO659, expected to start in 2022.
Read the press releases here: https://vicotx.com/european-commission-grants-vico-therapeutics-orphan-drug-designation-for-vo659-an-investigational-therapy-for-spinocerebellar-ataxia/ and here: https://vicotx.com/us-fda-grants-vico-therapeutics-orphan-drug-designation-for-vo659-an-investigational-therapy-for-spinocerebellar-ataxia/
Date posted: 26/08/2021
