Esketamine Hydrochloride

The FDA on Wednesday approved Soleno Therapeutics’ new drug designed to treat the worst symptom of a debilitating rare disease known as Prader-Willi syndrome. Regulators approved diazoxide choline (often shortened to DCCR) to treat insatiable hunger, or hyperphagia, felt by patients with Prader-Willi syndrome, a rare disease caused by spontaneous genetic mutations. The drug will be branded as Vykat XR and will cost an average of $466,200 per year, Soleno executives said late Wednesday. Soleno expects the drug to be available as soon as next month, according to an SEC document . Patients with Prader-Willi syndrome are typically diagnosed at birth because they are born “floppy,” with little muscle tone, CEO Anish Bhatnagar told Endpoints News ahead of the FDA’s decision. But their hyperphagia symptoms typically manifest when a patient is between 8 and 12 years old. Some may exhibit hyperphagia as early as four years. “When these babies are born, they are born with very low muscle tone, and so they’re called floppy babies,” Bhatnagar said. “You pick up the baby in the palm of your hand, and they would drape over on both sides. So it’s very obvious to the doctor at the time that something is wrong.” The disease is caused by random mutations of chromosome 15 and occurs in roughly one of every 15,000 live births globally. It’s not an inherited disease, so the prevalence is consistent across different geographies and ethnicities. In Western countries, “virtually all” of these babies end up in the NICU, Bhatnagar said. There are no other approved treatments for patients with Prader-Willi syndrome. Patients with Prader-Willi syndrome have a wide range of symptoms beyond hyperphagia, including delayed motor skills, not making enough growth hormone and speech and behavioral problems. But hyperphagia remains the most difficult for patients and their families. Divorce rates among parents of children with Prader-Willi syndrome are higher than normal, and unaffected siblings sometimes have PTSD from patients’ sometimes aggressive food-seeking behaviors. Families have to lock access to all food and trash cans in the house, Bhatnagar said. Patients may end up living in group homes for those with Prader-Willi syndrome if their families can afford it. Other times, they know their hyperphagia is a problem but can’t do anything about it. The behavioral problems often lead to mental health challenges and prescriptions for antidepressants, but those don’t tend to help. “As rare diseases go, this is one of those problems which is truly intractable,” the CEO said. Vykat XR doesn’t treat the underlying genetic cause of the disease. But by going after the intense desire to eat, Soleno hopes patients and families can alleviate the condition’s biggest burden. It works by targeting KATP, or the ATP-dependent potassium channel, and a hunger mechanism in the hypothalamus to reduce appetite. Vykat XR is a once-daily pill. Soleno intends to commercialize Vykat XR on its own in the US, though it may seek a partner to commercialize the drug elsewhere, Bhatnagar said. Soleno had $318 million in cash and cash equivalents at the end of 2024, and Bhatnagar said the company is “well-positioned” to launch Vykat XR. Some analysts think the drug could become a blockbuster. Stifel’s Paul Matteis wrote earlier this month that Vykat XR could reach at least $1.5 billion in annual peak sales despite the drug failing its primary endpoint in Phase 3 due to a pandemic-related high placebo response. But the FDA has opted to exercise more flexibility in rare disease approvals, Matteis added, and a randomized withdrawal study after the Phase 3 was highly statistically significant. Johnson & Johnson’s Spravato — a depression treatment — was approved in January using a similar trial framework. “We believe there’s potential upside here, as well,” Matteis wrote. Editor’s note: This article has been updated with Vykat XR’s average annual price.