FDA Grants Rare Disease and Orphan Drug Status to Nobias for 22q11.2 Syndrome

Nobias Therapeutics, a clinical-stage company developing novel small-molecule therapeutics for rare diseases, announced that the United States (U.S.) Food and Drug Administration (FDA) has granted rare pediatric disease designation (RPDD) and orphan drug designation (ODD) to the company’s small-molecule metabotropic glutamate receptor (mGluR) modulator NB-001 for the treatment of neuropsychiatric symptoms associated with 22q11.2 deletion syndrome (22q11DS).

Nobias announced positive top line data from a multi-center, randomized, double blind, placebo-controlled Phase 2 clinical trial of NB-001, which was first presented at the 52nd Child Neurology Society (CNS) Annual Meeting, in late 2023. With an estimated prevalence of 1 in 2148 live births, approximately 1600 children are born with 22q11DS in the U.S. every year, affecting approximately 65,000 Americans and making it the most common human microdeletion.

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Receiving Rare Pediatric Disease and Orphan Drug designations from the FDA are important mile markers in our efforts to develop the first therapeutic indicated for the neuropsychiatric symptoms – which can often be quite severe – of this syndrome.

“Patients living with 22q11DS are in need of better treatment options,” said Patrick Dougherty, CEO at Nobias Therapeutics. “Receiving Rare Pediatric Disease and Orphan Drug designations from the FDA are important mile markers in our efforts to develop the first therapeutic indicated for the neuropsychiatric symptoms – which can often be quite severe – of this syndrome. We are now focused on Q4 discussions with the FDA to clarify the regulatory path for advancing NB-001 into a registrational study for 22q11DS.”

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Children living with 22q11DS, also known as DiGeorge syndrome, typically experience a distinctive mix of neuropsychiatric symptoms that include elements of attention deficit hyperactivity disorder (ADHD), anxiety, and autism. Currently, there are no population-specific approved therapies for these conditions, leaving many patients with inadequate treatment options and resulting in a significant burden for their parents and other caretakers. As an mGluR modulator, NB-001 is uniquely positioned to help address the disrupted neuronal signaling in 22q11DS – the primary driver of the neuropsychiatric symptoms experienced by these patients.

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The FDA’s RPDD signifies eligibility for a priority review voucher (PRV), a program meant to encourage development of novel therapies for rare pediatric diseases. As a result of the ODD, Nobias will qualify for various incentives, including tax credits for qualified clinical testing, waiving of application fees associated with the Prescription Drug User Fee Act, and seven years of market exclusivity following marketing approval.

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Source – prweb