QBM-001 aims to treat toddlers with pediatric nonverbal autism who suffer from nonverbal or minimally verbal capabilities (PMVA). The company says there is no effective treatment available to treat an estimated 250,000 children born each year worldwide (20,000 in the US) with the affliction.
READ: Q BioMed to apply for Orphan Drug status with FDA, European regulators for its pediatric autism drug
Orphan Drug designation offers clinical development benefits including tax credits, accelerated evaluation of registration files and eligibility for seven years of market exclusivity in the US.
"We look forward to working with the US Food and Drug Administration on this application that will ultimately help us better serve these children and their families," said Q BioMed CEO Denis Corin in a statement. "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."
The company said Dr Pamela Ventola from the Child Study Center at Yale University, and a specialist in autism spectrum disorders, will be the primary contact for ongoing correspondence with the Office of Orphan Products Development at the FDA.
Seeking Orphan Drug status from EMA
Earlier this month, Q BioMed indicated that it will not only ask the FDA, but also apply to the European Medicines Agency for Orphan designation.
The company said its application to EMA will be based on its recent breakthrough discovery of two biomarkers that identifies PMVA. The company's study examined 1,953 autistic biomarkers.
In the meantime, the New York-based company’s second phase of QBM-001’s formulation is expected to be finalized in August 2019.
At that time, Q BioMed said it will request a pre-investigational new drug meeting with the FDA and similar discussions with Europe's EMA. Based on regulatory guidance, Q BioMed anticipates filing its new drug meeting submissions, including its current clinical trial design, in December.
And clinical trials may commence as early as the first quarter of 2020, the company said.
Shares in Q BioMed traded flat at $1.51.
—(Updates with details, plans for Europe)—
Contact Uttara Choudhury at [email protected]