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US FDA approves Takeda's narcolepsy drug Orzeyful

Created at 5 Aug · 10:50 PM1 source↑ Market-relevant
IN SHORT

The U.S. Food and Drug Administration has approved Takeda Pharmaceuticals' oral drug Orzeyful for narcolepsy type 1. The twice-daily pill is the first treatment to target the loss of orexin signaling, which causes the rare sleep disorder.

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Key Numbers

120,000people affected by narcolepsy type 1 in the U.S.
90days for DEA ruling on controlled substance classification

Who's Involved

U.S. Food and Drug Administration
approved Takeda's narcolepsy drug
Takeda Pharmaceuticals
developer of the narcolepsy drug Orzeyful
Drug Enforcement Administration
reviewing Orzeyful's controlled substance classification

↳ Why This Matters

The approval of Orzeyful offers a new therapeutic option for individuals suffering from narcolepsy type 1, addressing the root cause of the disorder and potentially improving quality of life for affected patients.

Key facts

  • The U.S. FDA approved Takeda Pharmaceuticals' oral drug Orzeyful for narcolepsy type 1.
  • Orzeyful is the first treatment approved to target the loss of orexin signaling, a cause of narcolepsy type 1.
  • The drug is a twice-daily pill.
  • Clinical studies showed patients taking Orzeyful had improved daytime wakefulness compared to placebo.
  • The DEA is reviewing Orzeyful's controlled substance classification, with a decision expected in 90 days.

The U.S. Food and Drug Administration has approved Takeda Pharmaceuticals' oral drug, Orzeyful, for the treatment of narcolepsy type 1. This marks the first approved medication that directly targets the loss of orexin signaling, a chemical messenger responsible for regulating wakefulness, which is the underlying cause of the rare sleep disorder.

Narcolepsy type 1 affects approximately 120,000 people in the United States and is characterized by excessive daytime sleepiness, sudden muscle tone loss, cognitive issues, and disrupted nighttime sleep. Orzeyful, administered as a twice-daily pill, demonstrated improvements in patients' ability to stay awake during the day in clinical studies compared to those receiving a placebo.

Previously, no medication was approved to treat narcolepsy type 1 as a unified disorder, and none acted on the orexin system. The Drug Enforcement Administration is currently reviewing Orzeyful's classification as a controlled substance, with a decision anticipated within 90 days. Following this classification, Takeda plans to make the drug available through a specialty pharmacy for U.S. healthcare providers and patients.

Frequently asked questions

Narcolepsy type 1 is a rare sleep disorder caused by the loss of brain cells that produce orexin, a chemical messenger regulating wakefulness. It leads to excessive daytime sleepiness, sudden muscle tone loss, cognitive symptoms, and disrupted nighttime sleep.

Orzeyful is a twice-daily pill that works by directly targeting the loss of orexin signaling, which is the cause of narcolepsy type 1.

The FDA's approval was based on data from two studies showing that patients given Orzeyful experienced improvements in their ability to stay awake during the day compared to those given a placebo.

The drug will be made available through a specialty pharmacy after the Drug Enforcement Administration completes its review of Orzeyful's controlled substance classification, which is expected within 90 days.

What Happens Next

01The DEA is expected to rule on Orzeyful's controlled substance classification within 90 days.
02Takeda will make Orzeyful available through a specialty pharmacy after classification.

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Cadence

How It Developed

The U.S. Food and Drug Administration approved Takeda Pharmaceuticals' oral drug Orzeyful for narcolepsy.
Orzeyful is the first treatment approved for narcolepsy type 1 that targets the loss of orexin signaling.
The drug's approval was based on studies showing improved wakefulness in patients compared to placebo.
The Drug Enforcement Administration is currently reviewing Orzeyful's controlled substance classification, with a ruling expected within 90 days.
Following classification, the drug will be available through a specialty pharmacy in the U.S.

Sources

T1
US FDA approves Takeda's sleep disorder pillReuters

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