SARASOTA, Fla. – Biom Therapeutics, a clinical stage biotech company, announced today it has received orphan drug designation status for BIO017—which is designed to treat Angelman syndrome—from the U.S. FDA’s Office of Orphan Product Development. Biom anticipates commencing clinical trials for BIO017 in Q4 of 2021, following submission of an Investigational New Drug (IND) application with the FDA in Q3 of this year.
Drugs that receive orphan drug designation benefit from incentives, which include the following: Seven years of market exclusivity following FDA approval, assistance and recommendations from the FDA on clinical studies, accelerated evaluation of registration files, and up to 50% tax credit on clinical trials conducted in the U.S. Orphan designation can be given to drugs that treat ailments which impact less than 200,000 patients. In the U.S. Angelman syndrome afflicts 33,071 patients which BIO017 should be able to treat if approved after clinical efficacy data.
“We are excited to receive the orphan status for BIO017, and grateful that FDA clearly identified the unmet medical need for a treatment for Angelman Syndrome patients. Angelman Syndrome is a very underserved subset of epilepsy and doesn’t have any approved treatments in US.” Stated Biom’s CEO Dr. Bobban Subhadra.
Paul Carney, M.D., FAES a pediatric neurologist, epileptologist, and cofounder at Biom added: “Our preclinical results were compelling that show the efficacy of the drug to reduce seizures as well other behavioral improvements in a mouse model of Angelman syndrome. These young patients really suffer, and this drug status will help to fast-track clinical development of BIO017 that will ultimately help us better serve these children and their families.”
About Angelman Syndrome
Angelman syndrome is a rare genetic neurological disorder characterized by global developmental delays, learning disabilities, and severe speech impairment. Other symptoms include an inability to coordinate voluntary movements, and tremulous, jerky movements of the arms and legs; a distinct behavioral pattern characterized by a happy disposition and unprovoked episodes of laughter and smiling, often at inappropriate times; seizures, sleep disorders and feeding difficulties. Some affected children may have distinctive facial features.
About Biom Therapeutics
Biom Therapeutics, a clinical stage biopharmaceutical company, was founded by a team of business leaders, scientists, and clinicians with a focus on developing therapeutics for hard-to-treat neurological diseases. Biom Therapeutics has positioned itself to be a world-class biotech firm that improves human health and quality of life by developing innovative drugs for treating and possibly reversing the effects of hard-to-treat diseases, including rare forms of epilepsy and autism. Biom’s immediate goal is to bring practical treatment into current medical practice as quickly as possible.
Please visit: https://biomtherapeutics.com/
John Allen, President
Biom Therapeutics LLC
Email : [email protected]