Oveporexton Treats Root Cause of Narcolepsy Type 1: Saema Tahir, MD

Oveporexton (Orzeyful; Takeda), an oral orexin receptor 2 (OX2R) agonist, treats the orexin deficiency behind narcolepsy type 1 rather than only its symptoms, according to Saema Tahir, MD, a board-certified pulmonary, critical care, and sleep medicine physician in New York City and assistant professor of medicine at New York Medical College.

Root Cause Treatment May Support First-Line Use

“Before we had options to treat symptoms, but now we probably have early options to treat the disease itself,” Tahir said in an interview with The American Journal of Managed Care^®.

The treatment was recently approved by the FDA for the treatment of narcolepsy type 1 (NT1, narcolepsy with cataplexy) in adults. Guidelines for narcolepsy type 1 have not been updated to include the therapy, but she believes it is possibly a first-line agent. She noted that on the maintenance of wakefulness test, results were about 4 times better than with previous therapies, and subjective sleepiness scores also improved. Tahir stressed that oveporexton is approved only for narcolepsy type 1, not narcolepsy type 2 or idiopathic hypersomnia.

Insomnia and urinary urgency and frequency were common in the supporting phase 3 FirstLight (TAK-861-3001; NCT06470828) and RadiantLight (TAK-861-3002; NCT06505031) trials. Tahir discusses every potential adverse effect before prescribing, then monitors patients after starting treatment. Mild insomnia may respond to behavioral changes, but persistent symptoms can create a second sleep disorder.

“Side effects when they develop in a patient should never be ignored,” she said, adding that dose reduction or a switch in therapy are options.

Access Barriers Are Expected

Tahir anticipates prior authorizations, appeals, and specialty pharmacy delays, particularly while the US Drug Enforcement Administration (DEA) scheduling is pending.

“It’s a huge barrier to an already difficult disorder,” she said, noting that patients often wait decades for a diagnosis.

Tahir said routine review of systems checklists lack a sleep category. Clinicians should ask detailed questions about dozing and sleep problems, she said, and consider referral to a sleep specialist while evaluating anemia or thyroid disease.

“There’s more harm for our patients when we dismiss, ignore, or delay,” she said.