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Articles

Jonathan Rubin, MD, MBA, chief medical officer at Supernus Pharmaceuticals, talked about the recent approval of the company’s subcutaneous infusion therapy SPN-830 for Parkinson disease.

Insights on the Newly Approved Subcutaneous Infusion Therapy for Parkinson Disease

In a recently presented study at ISC 2025, poststroke patients reported no additional benefit from using transcranial direct current stimulation as a potential intervention for recovery.

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Chief medical officer and senior vice president of Research & Development at Supernus Pharmaceuticals.

Nearly 4 and a half years since its original submission, the FDA has approved Supernus Pharmaceuticals’ investigational agent SPN-830 (Onapgo) as the first and only subcutaneous apomorphine infusion device for the treatment of motor fluctuations in adults with advanced Parkinson disease (PD). The company also noted that it will make SPN-830 available in the second quarter of 2025 with a support team of experts, including a robust nurse education program, and access support at launch.

"People living with Parkinson's and their families are hungry for new treatment options, and the PMD Alliance community was thrilled to learn about SPN-830's approval. Every newly approved Parkinson's therapy is a win for our community," 

 in a recent interview. "The introduction of this therapy brings with it an increased need for education, and underscores the importance of clinician-patient communication. PMD Alliance produces resources and education to help patients understand their symptoms, for personal empowerment, but also to streamline clinic visits. Our annual healthcare provider conference, 

Advanced Therapeutics in Movement and Related Disorders (ATMRD)

, equips clinicians with the information to integrate new therapies into practice."

The therapy’s approval was based on data from the TOLEDO study (NCT02006121), a randomized, double-blind study in which treatment with the device was associated with a difference of –1.89 hours per day of OFF time for patients with PD in comparison with placebo. Published in the 

in 2018, the study randomly assigned 106 patients living with the disease to either 3-mg/hour to 8-mg/hour dose of apomorphine (n = 53) or placebo saline infusion (n = 53) during their wake hours for a 12-week period.

"Hope is strong after this approval, the third in less than a year. Combined with momentum from the 

 being signed into law, 2025 has the potential to be a banner year for people living with Parkinson's and the healthcare providers caring for them," Merriam said. 

In TOLEDO, the intervention group, with a mean final drug dose of 4.68 mg/hour, showed significant improvements, including a reduction in OFF time of –2.47 hours/day compared with –0.58 hours/day for placebo (

 = .0025), and an increase in ON time without troublesome dyskinesia of 2.77 hours/day versus 0.80 hours/day for placebo (

 = .0008). Additionally, 62% of the apomorphine group were responders compared with 29% in the placebo group (P = .0008).

"The approval of ONAPGO (SPN-830) is a significant milestone in Parkinson’s disease management. As advanced practice providers, we play a critical role in optimizing treatment plans and ensuring patients have access to the best available therapies," 

Association of Movement Disorder Advanced Practice Providers (AMDAPP)

 in an interview. "The long-awaited approval of ONAPGO opens another valuable tool in our clinical toolbox, giving us a new option to address motor fluctuations and improve “on” time for our patients. This therapy has the potential to be a game-changer, helping us provide more individualized and effective care to those living with Parkinson’s. Additionally, as more therapies like this are approved, it reinforces the need for continued innovation in drug development, particularly in delivering more consistent symptom control without relying solely on oral medications."

The timeline for SPN-830 dates back to September 2020, when Supernus sent its original new drug application (NDA) to the FDA.

 Shortly after, the FDA issued a refusal to file letter indicating that the NDA was not sufficiently complete to permit a substantive review. Nearly a year later, in December 2021, the company resubmitted its NDA; however, it was once again met with resistance, as the FDA 

In the CRL, the agency noted that it required additional information and analyses related to the device, including labeling, product quality and manufacturing, device performance, and risk analysis. At the time, no additional efficacy or safety clinical studies were required. Notably, the FDA mentioned in the letter that the application's approval would require inspections that could not be completed in a timely manner, primarily because of travel restrictions amid the COVID-19 pandemic.

 for SPN-830 in October 2023 which was subsequently accepted by the FDA less than a month later.

 In April 2024, the FDA issued another CRL for the device, citing 2 areas that required additional review or more information.

 The first was related to product quality while the second related to the master file for the infusion device, which is proprietary to the device manufacturer. Supernus went back to make adjustments and thus, towards the latter half of 2024, the company announced that the FDA accepted its resubmission of the NDA for SPN-830, giving it a PDUFA date of February 1, 2025.

"The approval of SPN-830 is incredibly exciting! Extensive data supports its effectiveness, as it has been used in other regions for decades—dating back 29 years in Europe. As more treatment options become available for people living with Parkinson’s disease, we, as clinicians, can better tailor treatment plans to each individual’s unique needs and best interests," Erin Zinn, MSN, APRN-CNP, ANP-BC, CMRD, director of Operations & Strategy at AMDAPP, told 

. "SPN-830 represents yet another groundbreaking option for managing PD. This is an exciting time in the movement disorders community, with new advancements offering hope for improved quality of life for those with Parkinson’s."

Updates in Therapeutic Development: Clinical Trial Readouts to Watch in the First Half of 2025

At the end of the treatment period in TOLEO, patients on SPN-830 experienced a mean reduction in OFF time of –2.47 hours per day (SD, 3.70) compared with –0.58 hours per day (SD, 2.80) for those on placebo (

 = .0025). Investigators suggested that this therapy could potentially reduce the need for higher doses of concomitant oral antiparkinsonian medications.

Additional data from the study showed that 62% of patients on SPN-830 achieved at least a 2-hour reduction in OFF time compared with 29% of those on placebo. Overall, 71% of the SPN-830 group reported improvements in their general health compared with 18% in the placebo group. Patients on the active treatment also experienced a 2.77-hour/day increase in ON time without troublesome dyskinesia and significantly improved Patient Global Impression of Change scores (

 < .001). While reductions in oral levodopa dose were not significant (

 = .0615), SPN-830 led to a significantly greater reduction in levodopa-equivalent dose than placebo (

"This therapy is particularly beneficial for patients with moderate to advanced Parkinson’s disease who struggle with unpredictable OFF periods despite optimized oral treatment, as demonstrated in the TOLEDO study. By bypassing the gastrointestinal system, ONAPGO ensures more reliable medication absorption, which is critical for those with delayed gastric emptying or erratic response to oral levodopa," Zinn explained. "As with any infusion therapy, proper patient selection, dose titration, and monitoring are essential to maximize benefits while minimizing side effects such as nausea, hypotension, and skin reactions at the infusion site. All of which we can prepare for and manage well with proper education."

Apomorphine was first approved in 2004 under the brand name Apokyn for the acute, intermittent treatment of OFF episodes in PD. Apokyn is different from SPN-830, as it is administered by subcutaneous injection with a multiple-dose pen injector that can be used up to 5 times a day, with doses no less than 2 hours apart. Apomorphine works as a dopamine agonist that binds with high affinity to the dopamine D4 receptor, and moderate affinity for the dopamine D2, D3, and D5, and adrenergic α1D, α2B, α2C receptors.

"As APPs, we play a pivotal role in educating patients, initiating therapy, titrating doses, and troubleshooting infusion management to ensure the best possible outcomes. Hands-on training and education will be key to equipping providers with the skills needed to implement this therapy effectively," Papesh said. "At AMDAPP, we are committed to supporting APPs with the education and resources needed to integrate ONAPGO into clinical practice. We will do all we can to bring hands-on training and education to the community to ensure providers feel confident using this new treatment option."

In TOLEDO, SPN-830 was generally well tolerated, with no unexpected safety concerns. Most adverse events (AEs) were mild or moderate, and no deaths occurred. TEAEs were reported in 93% of patients on SPN-830 compared with 57% on placebo, with common AEs including skin reactions, nausea, and somnolence. A higher proportion of patients in the apomorphine group experienced AEs requiring dose modification.

“The approval of a new device that delivers a drug for folks with Parkinson’s disease, using an under the skin technology, and not requiring a wire in the brain or a tube in the stomach, is a huge advantage for many folks looking to improve their Parkinson’s disease symptoms," 

 and director of the Norman Fixel Institute for Neurological Diseases, told 

 in a recent interview. “This therapy has been under development for many years and finally got over the hurdle for FDA approval. We know from studies that have been published in journals like the 

 that the number of hours of ‘good quality on medication time’ for folks with Parkinson will potentially increase about two hours. This is a meaningful change.”

Six patients in the SPN-830 group withdrew from the study due to AEs. Three of these withdrawals were due to serious events: severe hypotension, myocardial infarction, and abnormal hematology results indicating mild leucopenia and moderate anemia (non-hemolytic). The remaining three withdrawals were attributed to visual hallucinations, moderate gait disturbance, and mild infusion-site erythema. Except for myocardial infarction, all AEs leading to withdrawal were considered treatment-related and resolved after discontinuation of SPN-830.

“The pump seems to be very well tolerated, however it will likely translate in the real world to requiring some maintenance, and we therefore believe that having really great care partners, which is something important in Parkinson’s disease outcomes, will be critical for folks interested in this pump technology," Okun said in the interview with 

. “This is the second subcutaneous pump recently approved by the FDA for treatment Parkinson’s disease and is great news for many of the folks who are suffering what we refer to as motor fluctuations; where their symptoms are bouncing up and down from feeling completely off medication to experiencing, in some cases dance like movements called dyskinesia.”

 “Though we are excited to see another pump therapy, and especially one that is under the skin and less invasive than other technologies, we will crucially need to learn how to apply this pump in clinical practice. We will need to understand what persons with [Parkinson] disease will benefit the most from this therapy, and also when to pull the trigger for more aggressive therapy like deep brain stimulation or focus ultrasound," Okun explained. 

REFERENCES
1. Supernus Announces FDA Approval of ONAPGO™ (apomorphine hydrochloride) for Parkinson’s Disease. News Release. Supernus. Published Febrauary 4, 2025. Accessed February 4, 2025. https://ir.supernus.com/news-releases/news-release-details/supernus-announces-fda-approval-onapgotm-apomorphine
2. Katzenschlager R, Poewe W, Rascol O, et al. Apomorphine subcutaneous infusion in patients with Parkinson's disease with persistent motor fluctuations (TOLEDO): a multicentre, double-blind, randomized, placebo-controlled trial. Lancet Neurol. 2018;17(9):749-759. doi:10.1016/S1474-4422(18)30239-4
3. Supernus Submits NDA for SPN-830 for Continuous Treatment of ON-OFF Episodes in Adults with Parkinson’s Disease Who Have Failed Two Treatments. News release. Supernus Pharmaceuticals. September 14, 2020. Accessed February 1, 2025. https://ir.supernus.com/news-releases/news-release-details/supernus-submits-nda-spn-830-continuous-treatment-episodes
4. Supernus Resubmits NDA for SPN-830 Apomorphine Infusion Device. News release. December 8, 2021. Accessed February 1, 2025. https://ir.supernus.com/news-releases/news-release-details/supernus-resubmits-nda-spn-830-apomorphine-infusion-device
5. Supernus Provides Regulatory Update on SPN-830. News release. Supernus Pharmaceuticals. October 2022. Accessed February 1, 2025. https://ir.supernus.com/news-releases/news-release-details/supernus-provides-regulatory-update-spn-830
6. Supernus resubmits NDA for SPN-830 apomorphine infusion device. News release. Supernus Pharmaceuticals. October 9, 2023. Accessed February 1, 2025. https://ir.supernus.com/news-releases/news-release-details/supernus-resubmits-nda-spn-830-apomorphine-infusion-device-0
7. Supernus Provides Regulatory Update for SPN-830. News Release. Supernus Pharmaceuticals. Published April 8, 2024. Accessed February 1, 2025. https://ir.supernus.com/news-releases/news-release-details/supernus-provides-regulatory-update-spn-830-0
8. Supernus Announces SPN-830 Apomorphine Infusion Device NDA Accepted for Review by FDA. News release. Supernus Pharmaceuticals. August 19, 2024. Accessed February 1, 2025. https://ir.supernus.com/news-releases/news-release-details/supernus-announces-spn-830-apomorphine-infusion-device-nda-0

Supernus Pharmaceuticals' SPN-830 (Onapgo) has been approved by the FDA as the first subcutaneous apomorphine infusion device for advanced Parkinson's disease, reducing motor fluctuations. The approval was based on the TOLEDO study, which demonstrated significant reductions in OFF time and increases in ON time without dyskinesia. Despite initial regulatory challenges, the device will be available in 2025. SPN-830 offers a non-invasive alternative to manage motor symptoms, bypassing gastrointestinal issues associated with oral medications, and providing more predictable symptom control.

SPN-830 is a wearable subcutaneous infusion device designed to deliver continuous treatment throughout the waking day, ensuring more consistent control of OFF time.

Clinical

FDA Approves Apomorphine Infusion Device SPN-830 as New Parkinson Treatment

’ resubmission of its new drug application (NDA) in September 2024, the agency has approved AXS-07, an oral, rapidly absorbed, multi-mechanistic agent, as a new acute treatment of migraine with or without aura in adults. Marketed as Symbravo, the company noted that it anticipates AXS-07 to be commercially available in the United States in approximately 4 months.

AXS-07, a novel agent, is thought to act by inhibiting calcitonin gene-related peptide (CGRP) release, reversing CGRP-mediated vasodilation, and inhibiting neuroinflammation, pain signal transmission, and central sensitization. The agent, which consists of meloxicam and rizatriptan, is enabled by Axsome’s MoSEIC (Molecular Solubility Enhanced Inclusion Complex) technology, which results in faster absorption and longer plasma half-life.

"In terms of use in clinical practice, we want to treat our patients with the simplest, most effective acute treatments that we're able to offer them. So, you would not use a combination triptan and non-steroidal product in a patient whose treatment needs were fully met, either by a triptan alone or by a non-steroidal anti-inflammatory alone," 

, professor of neurology and director of the Montefiore Headache Center at 

 in a recent interview. "In fact, the design for individuals with unmet needs on single, monotherapy acute treatments was specifically a reflection of the fact that those are the patients who need combination therapy. I think that's something most prescribers would do and should do."

 for its NDA submission of AXS-07, citing issues related to the chemistry, manufacturing, and controls considerations. At the time, the agency did not request any new clinical trials to be conducted.

The original NDA, accepted for review in September 2021, was supported by data from 2 phase 3 randomized controlled clinical trials—the MOMENTUM trial (NCT03896009) and the INTERCEPT trial (NCT04163185). Overall, the studies revealed a statistically significant elimination of migraine pain with AXS-07 compared with placebo and active controls. 

, AXS-07 met its coprimary end points with statistical significance, first showing a greater percentage of patients achieving pain freedom (19.9%) compared with placebo (6.7%; 

 <.001) 2 hours after dosing. Second, the absence of the most bothersome symptom (36.9%) compared with placebo (24.4%) was significant (

In MOMENTUM, 1594 patients with migraine were randomly assigned 2:2:2:1 to AXS-07, 10-mg rizatriptan, MoSEIC 20-mg meloxicam, or placebo, to treat a single migraine attack of moderate or severe intensity. INTERCEPT included 302 patients with migraine who were randomly assigned to either AXS-07 (n = 152) or placebo (n = 150) at the earliest onset of migraine pain.

 “It's always good when a new treatment is approved for migraine. This particular combination tablet is something that I've been working on as an investigator for years, so I'm delighted that it got approved, and I'm looking forward to when it becomes available in the next several months for patients for the acute treatment of migraine," 

, professor of neurology at the Geisel School of Medicine at Dartmouth and vice president of the New England Institute for Neurology and Headache, told 

 in a recent interview. "The hope is that as we educate practitioners, the understanding of why combining an [nonsteroidal anti-inflammatory drugs] with a triptan should provide additional benefit in the acute treatment of migraine for patients will become more and more clear, and that is my hope for the future."

In April 2020, months after the initial readout of MOMENTUM, Axsome announced 

. All told, the coprimary end points were also identical to MOMENTUM, with a statistically significantly greater percentage of patients achieving pain freedom compared with placebo (32.6% vs 16.3%, respectively; 

 = .002) at 2 hours post dose, as well as freedom from the most bothersome symptom (43.9% vs 26.7%, respectively; 

 = .003). AXS-07 also showed efficacy in preventing progression of migraine pain beyond mild intensity and significantly reducing the use of rescue medication, with freedom of pain progression occurring in 73.5% of AXS-07-treated patients compared with 47.4% of placebo patients between 2 and 24 hours post dose (

“Today’s approval of SYMBRAVO marks an important milestone for the migraine community by providing a rationally designed novel acute treatment for this debilitating condition. Migraine attacks strike without warning, and disrupt the lives of estimated more than 39 million patients in the U.S. alone," Herriot Tabuteau, MD, chief executive officer at Axsome Therapeutics, said in a statement.

 "SYMBRAVO provides patients and clinicians an important new option which can quickly stop a migraine attack, keep it away, and allow patients to resume their normal activities, with just a single dose. SYMBRAVO demonstrates Axsome’s commitment to developing and delivering differentiated new treatments to improve the lives of patients living with difficult to treat central nervous system disorders.”

2024 American Academy of Neurology Annual Meeting

pooled analysis of the MOMENTUM and INTERCEPT trials

, with results showing that AXS-07 was effective at acutely treating migraine. In the pooled analysis, treatment with AXS-07 resulted in greater headache pain freedom (23% vs 11%; 

<.001) and absence of the most bothersome symptom (39% vs 25%; 

<.001) at 2 hours compared with placebo. Led by Stewart J. Tepper, MD, professor of neurology at the Geisel School of Medicine at Dartmouth, 43% of AXS-07-treated patients had reduced rescue medication use through 24 hours vs 21% of those on placebo (

<.001). Notably, a higher rate of patients on the investigational agent returned to normal functioning in comparison with placebo starting at 1-hour post-dose.

“Migraine is one of the most misunderstood and stigmatized neurological diseases, despite the fact that one in four households in the U.S. includes someone living with it. This widespread lack of understanding creates unnecessary barriers for individuals seeking proper diagnosis, care, and treatment," Susan Doughty, executive director of the Coalition for Headache and Migraine Patients (CHAMP), said in a statement.

 "CHAMP, alongside our 20 plus dedicated coalition organizations and patient advocates, is committed to empowering the migraine community by providing education, reducing stigma, and advocating for fair and equitable access to treatment options. The approval of SYMBRAVO as a new acute treatment for migraine is an important step forward, offering a new option for people seeking relief. We also see this moment as an opportunity to continue to shine a bright light on migraine, fostering greater awareness and helping to dismantle the stigma that so often surrounds this disease.”

REFERENCES
1. Axsome Therapeutics Announces FDA Approval of SYMBRAVO® (meloxicam and rizatriptan) for the Acute Treatment of Migraine with or without Aura in Adults. News Release. Axsome Therapeutics. Published January 30, 2025. Accessed January 30, 2025. https://axsometherapeuticsinc.gcs-web.com/news-releases/news-release-details/axsome-therapeutics-announces-fda-approval-symbravor-meloxicam
2. Axsome Therapeutics Receives FDA Complete Response Letter for New Drug Application for AXS-07 for the Acute Treatment of Migraine. News release. Axsome Therapeutics. May 2, 2022. Accessed January 30, 2025 https://www.prnewswire.com/news-releases/axsome-therapeutics-receives-fda-complete-response-letter-for-new-drug-application-for-axs-07-for-the-acute-treatment-of-migraine-301537093.html
3. Axsome Therapeutics Announces AXS-07 Achieves Co-Primary and Key Secondary Endpoints in MOMENTUM Phase 3 Migraine Trial in Patients with History of Inadequate Response. News release. Axsome Therapeutics. December 30, 2019. Accessed January 30, 2025. https://www.biospace.com/article/releases/axsome-therapeutics-announces-axs-07-achieves-co-primary-and-key-secondary-endpoints-in-momentum-phase-3-migraine-trial-in-patients-with-history-of-inadequate-response
4. Axsome Therapeutics announces AXS-07 achieves both co-primary endpoints and prevents migraine pain progression in the INTERCEPT phase 3 trial in the early treatment of migraine. news release. Axsome Therapeutics. April 6, 2020. Accessed January 30, 2025. https://www.finance.yahoo.com/news/axsome-therapeutics-announces-axs-07-100010869.html
5. Tepper S. Lipton R, Chhabra A, Streicher C, Parks G, Tabuteau H. Combined efficacy and safety of AXS-07 (MOSEICTM Meloxicam and Rizatriptan) in two phase 3 clinical trials. Presented at: 2024 AAN Annual Meeting; April 13-18; Denver, CO. Abstract 00519

The FDA has approved AXS-07 (Symbravo), a novel oral treatment for acute migraine in adults, developed by Axsome Therapeutics. AXS-07 combines meloxicam and rizatriptan, utilizing MoSEIC technology for rapid absorption and extended plasma half-life. Clinical trials, including MOMENTUM and INTERCEPT, demonstrated significant efficacy in achieving pain freedom and reducing migraine symptoms compared to placebo. The approval addresses unmet needs in migraine treatment, offering a new option for patients with inadequate response to existing therapies. AXS-07 is expected to be available in the U.S. within four months.

Findings from previous phase 3 trial showed that treatment with AXS-07 resulted in significant reductions in headache pain freedom, migraine symptoms, and rescue medication use.

FDA Approves Axsome Therapeutics’ AXS-07 for Migraine Treatment

A recently published international survey reported that a small portion of patients with narcolepsy or idiopathic hypersomnia felt supported when adjusting to life with their condition. Published in 

the results of the survey highlighted the need for clinicians to assess social support and guide patients living with these sleep disorders toward resources that can foster community connections.

The online survey, conducted by Project Sleep

, gathered responses from 1308 adults with narcolepsy type 1 (48.4%), narcolepsy type 2 (28.7%), or idiopathic hypersomnia (22.9%). Participants were predominantly women (87.7%), White (88.8%), and under 40 years of age (68.7%). Participants represented 38 countries, with the majority (65.5%) residing in the United States, followed by 15.4% in Europe and 11.8% in Australia/New Zealand.

Adjusting to life with a diagnosis was challenging for the majority of respondents, with 67.6% rating the process as difficult or very difficult. Individuals with idiopathic hypersomnia reported greater difficulty compared with those with narcolepsy. Overall, only 32.3% of respondents felt supported in adapting to life with their condition.

"Clinicians see multiple patients with narcolepsy or idiopathic hypersomnia, but those patients don’t see or connect with each other. For those who did meet others, there was a wide range of experiences—some found social support quickly, within a few months or years, while others took much longer," lead author Julie Flygare, JD, president and CEO at Project Sleep, told  

 in a recent interview. "Visually, I think of social connection as opening a door. Without it, you’re behind a closed door, alone. But when that door opens, you see a world of others who share your experience. That’s powerful."

Led by senior author sleep disorder expert 

, director of Child Neurology and Pediatric Sleep Medicine at Geisinger Janet Weis Children's Hospital, the online survey was conducted between March 28 and April 11, 2021. The survey, composed of 30 questions, asked respondents about their diagnosis, time from symptom onset to diagnosis, ease or difficulty in adjusting to their diagnosis, whether or not they knew someone with the same diagnosis, the impact of knowing another person with the same diagnosis, methods of communicating with other people with their diagnosis and topics discussed, and sources of support.

"We weren’t really sure how to develop the survey because I hadn’t seen this question asked so directly in other communities either. So, we asked people whether they knew someone living with the same condition at the time of their diagnosis. Very few did," Flygare said. "In some cases, there are familial groupings—sometimes a child gets diagnosed because they have a parent with narcolepsy, or families become more aware of sleep disorders and recognize them more easily in their relatives. But for most, they didn’t know anyone else."

Delays in diagnosis were frequently reported, with over one-third of respondents indicating it took 10 years or more after symptom onset to receive a diagnosis, and only one-quarter were diagnosed in 3 years. Diagnostic timelines varied by condition, for example, individuals with narcolepsy type 1 were more likely to be diagnosed in 3 years (29.2%) compared with those with narcolepsy type 2 (17.9%) or idiopathic hypersomnia (17.7%). Conversely, a higher proportion of respondents with idiopathic hypersomnia (44.7%) experienced delays of 10 years or more, compared with those with narcolepsy type 2 (38.9%) and narcolepsy type 1 (35.2%).

FDA Clears Phase 2 Trial of Lorundrostat for Obstructive Sleep Apnea and Hypertension

At the time of diagnosis, most respondents (91.1 %) did not know anyone else with the same condition. This was reported by nearly all individuals with idiopathic hypersomnia (96.0%), as well as 88.8% of those with narcolepsy type 1 and 91.2% of those with narcolepsy type 2. These rates were consistent across geographic regions, with 91.0% of U.S. respondents and 91.2% of respondents from other countries reporting similar experiences. Rates were also comparable across age groups: 89.8% for ages 18–30, 92.1% for ages 31–50, and 91.3% for those over 50.

Regression analysis revealed that knowing someone with the same diagnosis was strongly associated with a greater sense of support. Younger respondents (ages 18–30) and those who were married also reported higher levels of perceived support. However, individuals with narcolepsy type 2 or idiopathic hypersomnia reported feeling less supported than those with narcolepsy type 1. Living outside the U.S. was another factor linked to lower perceived support. Additionally, respondents who identified as nonbinary, transgender, or genderfluid tended to feel less supported compared with those who identified as women (

"People with narcolepsy are diverse, and not every patient connection results in finding a new best friend. But over time, when people engage with patient advocacy groups, participate in programs, and take part in advocacy, they meet different individuals. Eventually, they often find close connections with others who truly understand their journey. They also find role models—people further along in their experience—who they can look up to. That’s different from peer support but just as important," Flygare explained in the interview.

The current study had several limitations including that diagnoses were self-reported and not verified through medical records. Additionally, the survey did not collect data on symptoms or their severity, making it impossible to analyze the relationship between social support and symptomatology. Conducted in 2021 during the COVID-19 pandemic, the survey’s findings may have been influenced by the unique social dynamics of that time, though the extent of this impact is unclear. While duplicate submissions were removed, there remains a small chance that some individuals submitted multiple responses with variations. However, this risk was deemed minimal and acceptable by researchers to preserve participant anonymity.

“I think clinicians can start by simply asking their patients, 'Do you know someone else living with this condition?' It’s a great way to begin a conversation. Social support can take many forms—not everyone wants to go to a support group, and that’s okay. But letting patients know about available patient advocacy organizations is crucial. In the U.S., there are 4 major groups, plus others worldwide. These organizations are great starting points for patients to explore what’s available to them,” Flygare highlighted in the conversation. "We often hear from people who went years after their diagnosis without knowing that Project Sleep existed, and when they finally find our resources, they’re so excited. That just feels sad because they could have had that support much earlier. So, letting patients know about advocacy organizations is a good start."

REFERENCES
1. Flygare J, Oglesby L, Parthasarathy S, et al. Social support and isolation in narcolepsy and idiopathic hypersomnia: An international survey. Sleep Med. 2025;125:65-73. doi:10.1016/j.sleep.2024.11.013
2. Project Sleep. Homepage. Accessed January 23, 2024. https://project-sleep.com/

A recent international survey highlights the challenges faced by patients with narcolepsy and idiopathic hypersomnia in finding social support. Conducted by Project Sleep, the survey involved 1308 participants, revealing that only 32.3% felt supported in adapting to their condition. Delays in diagnosis were common, with many taking over a decade to receive a diagnosis. Knowing someone with the same condition was linked to increased perceived support. The study underscores the need for healthcare providers to facilitate connections and guide patients toward supportive resources, despite privacy concerns.

In a recent survey, key factors associated with perceived support included knowing someone who had the same diagnosis of their sleep disorder, younger age, and marital status. 

Survey Highlights Patients’ Need for Support in Adapting to Life in Narcolepsy or Idiopathic Hypersomnia

New topline results from the randomized, double-blind, placebo-controlled phase 2 trial (NCT05109091) of Alterity Therapeutics’ ATH434 revealed that treatment with the investigational agent was well tolerated and demonstrated clinical as well as biomarker-based benefits, particularly at the 50 mg dose, among patients with early-stage multiple system atrophy (MSA).These findings support ATH434’s potential as a disease-modifying therapy for MSA to slow disease progression and reduce iron accumulation in key brain regions.

The study enrolled 77 participants across 23 sites in 6 countries, who received either 50 mg or 75 mg of ATH434 or placebo twice daily for 12 months. Findings showed that ATH434 was well tolerated and effectively reduced iron accumulation in brain regions impacted by MSA. The 50 mg dose demonstrated the greatest benefit, slowing clinical progression by 48% at 52 weeks compared with the placebo (

 = 0.03). Additional analyses also suggested trends in improved motor function and preserved brain volume.

"I was impressed with the effects, that appear durable over 52 weeks in the 50mg dose. As a clinical signal, it supports progressing to a larger phase 3 trial. We want to see a therapy have a chance to change the way MSA progresses. This is a step in the right direction. We see a clear signal for disease modification which is truly exciting," coordinating investigator 

"We see a clear signal for disease modification which is truly exciting. This is a new concept for MSA. There is a clear antioxidant mechanism, an effect on labile iron, and indication that MSA is important for clinical development of this compound."

The primary end point of the trial assessed brain iron content via MRI. All told, ATH434 reduced or stabilized iron accumulation in affected brain regions, with the 50 mg dose showing significant reductions in the putamen at 26 weeks (

 = 0.025) and approaching significance in the globus pallidus at 52 weeks (

 = 0.08). Clinical efficacy was assessed using the Unified MSA Rating Scale Part I (UMSARS I), a common MSA assessment that measures disability and disease severity.

ATH434, an investigational agent by Alterity Therapeutics, showed promise in a phase 2 trial for early-stage multiple system atrophy (MSA). The trial demonstrated that ATH434 was well tolerated and reduced iron accumulation in key brain regions, with the 50 mg dose significantly slowing disease progression by 48% over 52 weeks. The study's primary endpoint was brain iron content, with secondary endpoints including motor performance improvements. These findings suggest ATH434's potential as a disease-modifying therapy for MSA, with implications for other neurodegenerative disorders.

Recent results from a phase 2 trial of ATH434 revealed its potential in reducing brain iron accumulation and preserving motor function in early-stage multiple system atrophy.

Jonathan Rubin, MD, MBA

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