In Case You Missed It! – August 2022

If you’ve missed any of the latest news we’ve shared through our social media this month, don’t worry! Get up to date with this edition of “In Case You Missed It!”

  • New medicine serdexmethylphenidate for IH set to enter clinical trials later this year
  • New medicine mazindol ER gets orphan drug designation for IH in Europe
  • U.S. Congress passes the Inflation Reduction Act
  • Healthcare professionals can earn CME credits with free online course about IH
  • Register today for a free camp for kids with narcolepsy and IH and their families
  • Project Sleep shares 4-part series about sleep and mental health

Don’t worry if you’ve missed anything. We’ve got you covered!


New medicine serdexmethylphenidate for IH set to enter clinical trials later this year 

KemPharm has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for serdexmethylphenidate (SDX) for the treatment of IH. A clinical trial in IH is planned to start later this year, and one for narcolepsy may start soon after.

SDX is in the same class of medicines as methylphenidate (such as Ritalin), but SDX is designed to give a smoother, more gradual release of medicine, which may help lessen side effects compared to other stimulant-based therapies. SDX is currently on the market in the U.S., as a component of AZSTARYS, which is approved for the treatment of ADHD. To learn more, read “New Idiopathic Hypersomnia Drug Candidate Set to Enter Clinical Trials Later This Year” from a recent online edition of Sleep Review magazine.

KemPharm’s clinical trial will hopefully provide stronger evidence for the use of methylphenidate-class medicines in people with IH, which could hopefully lead to easier insurance coverage. Currently, there is not much specific evidence for IH treatment, and the AASM treatment guidelines gives methylphenidate a conditional recommendation. (Learn more about the AASM treatment guideline HERE.)


New medicine mazindol ER gets orphan drug designation for IH in Europe

The European Medicines Agency (EMA) Committee for Orphan Medical Products (COMP) has granted Orphan Drug Designation (ODD) for mazindol ER for the treatment of IH. This means that when this medicine becomes approved in Europe, it will already have an orphan drug designation there for IH. Mazindol ER has also has been granted ODD in both Europe and the U.S. for the treatment of narcolepsy. This is great news, and hopefully mazindol ER will eventually become approved and available in the U.S., Europe, and elsewhere. (To learn more, read the press release HERE.)

Also, the Japanese Patent Office has granted a patent for mazindol ER, including for the treatment of excessive daytime sleepiness (EDS) in narcolepsy and IH. Key patents are now granted in major markets including the U.S., Europe, Japan, Canada & South Korea. (To learn more, read the press release HERE.)

NLS Pharmaceutics plans to report the final results from their Phase 2a narcolepsy trial for Quilience® in late September and also give an update from their open-label extension study in both narcolepsy and IH later this year. To learn more, check out news we shared earlier this year via social media and “SomnusNooze”:


U.S. Congress passes the Inflation Reduction Act 

Great News! The U.S. Congress recently passed the Inflation Reduction Act. The bill includes many “must haves” for the health and wellbeing of people and families in the U.S. with rare diseases. For example:

  • The extension of enhanced Advanced Premium Tax Credits for 3 years will help keep quality health insurance accessible to millions of Americans.
  • The creation of an annual out-of-pocket cap on prescription medicine costs for people with Medicare will help those who struggle to afford their medications.

Read NORD’s complete statement HERE.


Healthcare professionals can earn CME credits with free online course about IH

At SLEEP 2022, Haymarket and Hypersomnia Foundation presented “A New Dawn for Better Outcomes in Patients With Idiopathic Hypersomnia: New Data, New Guidance, New Treatments.” This CME program is now available free online to healthcare professionals, such as sleep specialists, psychiatrists, neurologists, and advanced-practice clinicians. For a link to this free course, visit the “My CME” website.

Recent updates in AASM Guidelines and the approval of the first medicines to treat IH have the potential to change the way IH is managed. Learn from experts, Russell Rosenberg, PhD, FAASM, Anne Marie Morse, DO, FAASM, Lynn Marie Trotti, MD, MSc, and a person with idiopathic hypersomnia, as they discuss how to overcome challenges in diagnosing IH, and focusing on personalized management to treat not only disease symptoms, but also quality of life and function in IH.


Register today for a free family camp for kids with narcolepsy and IH 

Register now for the Wake Up Narcolepsy and The Center for Courageous Kids (CCK)Narcolepsy Family Weekend”—a fun-filled, free weekend retreat for kids with narcolepsy and idiopathic hypersomnia with their families. The camp is located in Scottsville, Kentucky and is scheduled for September 30 through October 2, 2022.

Connect with other families and create new friendships through workshops and discussion groups while also enjoying fishing, swimming, dancing, climbing, and horseback riding. For affected kids and their families, some of their anxieties and concerns can be significantly helped by learning that this neurological disorder affects others just like them.

For details and registration information, visit the CCK website.


Project Sleep shares series about sleep and mental health

We are happy to share Project Sleep’s 4-part series about connections between sleep and mental health. Originally posted during Mental Health Awareness Month, we thank Project Sleep for this important information, providing helpful support to our families and communities. The series covers the following topics:

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