OV101 Gets FDA’s Rare Pediatric Disease Status for Treating Angelman

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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rare pediatric disease designation, OV101

Ovid Therapeutics OV101 (gaboxadol) has been grantedrare pediatric disease designation by the U.S. Food and Drug Administration (FDA) for the treatment of Angelman syndrome.

This designation is given to experimental therapies that have the potential to treat serious diseases that mainly affect children (ages 18 and younger) and that affect fewer than 200,000 people in the U.S.

“OV101 has the potential to become the first FDA-approved therapy for individuals living with Angelman syndrome,” Amit Rakhit, MD, Ovid president and chief medical officer, said in a press release, calling the designation “a significant milestone for this program and underscores the critical value of our work.”

Of note, this designation means that — if OV101 is eventually approved — then Ovid would be eligible to receive a priority review voucher from the FDA. This voucher can be used to get priority review on subsequent submissions to the FDA (related to OV101 or not), or it can be traded to other companies.

“Importantly, with this designation, we may be eligible to receive a priority review voucher from the FDA, providing significant value as we work diligently towards the completion of our ongoing pivotal Phase 3 NEPTUNE trial,” Rakhit said. “We are grateful to the FDA and Congress for having enacted this law which helps Ovid and all companies developing innovative drugs for rare pediatric conditions.”

OV101 is an experimental therapy that is currently being developed to treat Angelman syndrome and Fragile X syndrome. It has previously received orphan drug and fast track designations from the FDA for both conditions.

Both syndromes are associated with reduced tonic inhibition — a process that decreases excessive activity of neurons in the brain. OV101 works by activating specific receptors for the neurotransmitter GABA (gamma-aminobutyric acid). By activating these receptors, OV101 is thought to increase tonic inhibition and, consequently, ease symptoms of these syndromes. 

In a Phase 1 trial (NCT03109756), OV101 was found to be generally safe and well-tolerated in people with Angelman or Fragile X syndrome. Subsequently, results from the Phase 2 STARS trial (NCT02996305) indicated that OV101 improved sleep, motor function, and communication skills in people with Angelman syndrome. The results also indicated that OV101 can reduce anxiety.

OV101 is currently being evaluated in the Phase 3 NEPTUNE trial (NCT04106557) in up to 90 children (ages 2–12 years) with Angelman syndrome. Participants are being randomized to receive OV101 or a placebo (taken by mouth once per day) for 12 weeks.

Results from NEPTUNE are expected by the end of this year. The trial is still recruiting participants at multiple locations in the U.S., Australia, the Netherlands, Germany, and Israel. Additional information is available here.