Q BioMed Files for Orphan Drug Designation with U.S. FDA for Autism Spectrum Disorder Subtype
- Currently no effective treatment exists for the 20,000 children in the U.S. and 250,000 born worldwide each year with pediatric minimally verbal autism (PMVA)
New York, New York--(Newsfile Corp. - June 28, 2019) - Q BioMed Inc. (OTCQB: QBIO), a commercial stage biotech company, announced today it has filed an application for Orphan Drug designation with the U.S. FDA's Office of Orphan Product Development for QBM-001, which is designed to treat pediatric minimally verbal autism (PVMA). Q BioMed anticipates commencing clinical trials for QBM-001 in the first half of 2020, following submission of an Investigational New Drug (IND) application with the FDA later this year.
Drugs that receive Orphan Drug designation benefit from incentives including 7 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 indications impacting less than 200,000 patients per year in the U.S. Of the estimated 67,000 children diagnosed with autism spectrum disorder each year in the U.S., 20,000 become minimally-verbal; QBM-001 should be able to treat 15,000 of these children.
"We look forward to working with FDA on this application that will ultimately help us better serve these children and their families," stated Q BioMed CEO Denis Corin. "Pediatric minimally verbal autism is a very underserved subset of autism and we are grateful to all the clinical psychologists, neurologists and doctors who have contributed to this filing and the differential diagnosis for this subset."
Dr. Pamela Ventola from the Child Study Center at Yale University, and a specialist in autism spectrum disorders, has agreed to be the primary contact for ongoing correspondence with the Office of Orphan Products Development at the FDA.
About Pediatric Minimally Verbal Autism (PMVA)
There are approximately 18,000-20,000 new cases of pediatric minimally verbal autism in the U.S. each year and a similar amount in Europe. The majority of these children are diagnosed between the ages of 4 and 7 and fall within the autism spectrum. Individually, the economic costs for toddlers who become non- or minimally verbal is $4.5 million dollars on average per child over a lifetime. Collectively, an estimated $100 billion is spent yearly on individuals who are minimally verbal in the U.S. Not all individuals who are minimally verbal will benefit from QBM-001. However, with validated biomarkers, testing from trained specialists and genetic testing, children who fall in this targeted population can be identified, and will have a higher likelihood of responding to an approved treatment.
About Q BioMed Inc.
Q BioMed Inc. is a biotech acceleration and commercial stage company. We are focused on licensing and acquiring undervalued biomedical assets in the healthcare sector. Q BioMed is dedicated to providing these target assets; strategic resources, developmental support, and expansion capital to ensure they meet their developmental potential, enabling them to provide products to patients in need.
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