The CheckRare Brief

CheckRare Media
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A weekly roundup of the latest news in rare disease. The CheckRare Brief delivers a concise roundup of the week's most important developments in rare diseases. Each episode covers FDA approvals, clinical trial updates, conference highlights, scientific publications, and industry news that matter to healthcare professionals, researchers, advocates, and the rare disease community. Produced by CheckRare, The CheckRare Brief helps you stay informed—in just a few minutes each week.

Episodes

  1. 2d ago

    FDA Approves Multiple Myeloma Drug;  FDA Denies Neuroendocrine Drug; Safety Concerns For Prader-Willi Drug

    On this week’s episode of The CheckRare Brief, we discuss FDA’s approval of Zenbexus (iberdomide) to treat patients with multiple myeloma, the complete response letter issued to ITM-11 to treat patients with neuroendocrine tumors, and safety concerns about VYKAT XR for Prader-Willi syndrome (PWS). The US Food and Drug Administration (FDA) granted accelerated approval to Bristol Myers Squibb’s iberdomide to treat patients with relapsed or refractory multiple myeloma on August 13, 2026. This treatment is a cereblon E3 ligase modulator (CELMoD), a new therapeutic class of drug. The approval is largely based on results from the EXCALIBER-RRMM clinical trial (NCT04975997), a two-stage, randomized, multicenter, open-label trial in adults with relapsed or refractory MM who had previously received one or two prior lines of therapy. Additionally, another new trend is the outcome measure of minimal residual disease, a departure from more traditional measures such as overall survival or progression free survival. In other regulatory news, the FDA gave ITM a complete response letter (CRL) for ITM-11, a radioisotope (177-lutetium) attached to a peptide (edotreotide) that emits beta radiation towards targeted tumors, for neuroendocrine tumors. A phase 3 clinical trial showed the treatment to be significantly better than the control group, with a progression free survival of 24 months in the ITM-11 group versus 14 months in the control group. It appears that the FDA's concern wasn't the drug efficacy but about the manufacturing process. While it is a setback, it does not necessarily mean the drug has been rejected permanently. The company can address the FDA's concerns and resubmit the application. Finally, the Foundation for Prader-Willi Research are voicing concerns about VYKAT XR (diazoxide choline), a treatment approved in 2025 for children with Prader-Willi syndrome to address dysphagia. The main concern has to do with excessive fluid retention occurring in some patients that can lead to other serious adverse events, including cardiac and breathing concerns. The drug was approved last year to treat children with Prader-Willi syndrome to better control their hunger. These concerns highlight the importance of making physicians aware of the drug’s safety and efficacy in a real-world setting following drug approval.  Sources FDA Grants Accelerated Approval For Multiple Myeloma Drug https://www.businesswire.com/news/home/20260811027471/en/U.S.-FDA-Grants-Accelerated-Approval-to-Bristol-Myers-Squibbs-First-CELMoD-Therapy-ZENBEXUS-in-Combination-with-Daratumumab-and-Hyaluronidase-fihj-and-Dexamethasone-ZDd-for-Patients-with-Multiple-Myeloma-as-Early-as-First-Relapse    ITM Receives Complete Response Letter for ¹⁷⁷Lu-edotreotide (ITM-11) https://www.itm-radiopharma.com/news/press-releases/press-releases-detail/itm-receives-complete-response-letter-for-177lu-edotreotide-itm-11-763/    Prader-Willi Researchers Raise Concerns About the Safety of Approved Drug https://www.fpwr.org/blog/vykat-xr-clinician-recommendations-for-real-world-use-and-monitoring-side-effects Produced by CheckRare. Explore additional CME activities, physician interviews, podcasts, and rare disease resources at CheckRare.com. Subscribe to the CheckRare Podcast Network for the latest rare disease education, expert interviews, and weekly news. Part of the CheckRare Podcast Network: Trusted conversations, news, education, and expert insights across the rare disease community.

    FDA Approves Multiple Myeloma Drug;  FDA Denies Neuroendocrine Drug; Safety Concerns For Prader-Willi Drug

  2. Aug 12

    FDA Approves New Narcolepsy Treatment

    On this week’s episode of The CheckRare Brief, we discuss FDA’s approval of Orzeyful (oveporexton) to treat patients with narcolepsy type 1, CAMP4’s first-in-human clinical trial for patients with SYNGAP1-related disorders, and BioMarin’s termination for their ENPP1 deficiency program following mixed results from their phase 3 clinical trial. The US Food and Drug Administration (FDA) approved Takeda’s Orzeyful (oveporexton) for narcolepsy type 1 on August 5, 2026. The approval came nearly two months ahead of its September 30th PDUFA date. Oveporexton is an orexin receptor 2 agonist designed to directly target the pathway involved and the underlying biology of narcolepsy. This novel mechanism of action could dramatically change how patients with narcolepsy are managed, giving patients more choices. Plans to dispense the drug via specialty pharmacies following the DEA’s review are underway. CAMP4 is advancing CMP-002 into a first-in-human phase 1/2 trial for SYNGAP1-related disorder, a genetic condition that causes debilitating autistic-like behaviors, seizures, gastrointestinal problems, and intellectual disabilities. There are currently no treatments approved for this rare condition. CMP-002 is an antisense oligonucleotide that binds to regulatory RNA to increase activity of the SYNGAP1 gene and restore SYNGAP1 protein to normal levels.  This clinical trial is a good example of how the rare disease landscape has changed. Advances in genetic testing and the work of patient advocacy groups helped identify these patients and give a once unknown condition a name. Identifying a patient population is crucial to understanding the natural history of the disease, developing clinical trials, and ultimately attracting investment in treatments. Finally, BioMarin has terminated the development of enzyme replacement therapy BMN 401 after their phase 3 trial in ENPP1 deficiency failed to meet one of its two co-primary endpoints. ENPP1 deficiency is a genetic disorder that results in a reduction of pyrophosphate, causing rickets or soft bones. While the trial showed the drug improving pyrophosphate levels, improvements in bone health, the clinically relevant measure required by the FDA, were not observed. This failed trial is very disheartening for the ENPP1 community, but will hopefully aid in providing the foundation for more robust clinical trials going forward. Sources FDA approves Orzeyful (oveporexton) https://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-treat-full-range-narcolepsy-type-1-symptoms SYNGAP 1 Clinical Trial To Begin https://www.globenewswire.com/news-release/2026/07/27/3333378/0/en/camp4-therapeutics-secures-australian-regulatory-clearance-to-initiate-first-in-human-clinical-trial-of-cmp-002-in-patients-with-syngap1-related-disorder.html EENP1 Clinical Program Terminated https://www.prnewswire.com/news-releases/biomarin-reports-second-quarter-2026-financial-and-operating-results-302845167.html 2026 Orphan Drugs: PDUFA Dates and FDA Approvals https://checkrare.com/2026-orphan-drugs-pdufa-dates-and-fda-approvals/ Produced by CheckRare. Explore additional CME activities, physician interviews, podcasts, and rare disease resources at CheckRare.com. Subscribe to the CheckRare Podcast Network for the latest rare disease education, expert interviews, and weekly news. Part of the CheckRare Podcast Network: Trusted conversations, news, education, and expert insights across the rare disease community.

    FDA Approves New Narcolepsy Treatment

  3. Aug 5

    Episode 1: FDA Advisory Board’s Concerns About New Duchenne Drug, Brain Fog in Immune Thrombocytopenia; August is SMA Awareness Month

    The CheckRare Brief delivers a concise summary of the week's most important developments in rare diseases. Each episode covers FDA approvals, clinical trial updates, conference highlights, scientific publications, and industry news that matter to healthcare professionals, researchers, advocates, and the rare disease community. Madaline Spencer, Podcast Producer; James Radke, PhD; Education Director; Peter Ciszewski,  Founder and CEO, CheckRare   References for this episode’s topics: FDA Advisory Report on Capricor Therapeutics’ Orphan Drug to Treat Cardiomyopathy in Duchenne Muscular DystrophyFDA Advisory Board Report: LinkCapricor’s response: Link Cognitive Decline in Immune ThrombocytopeniaInterview with Dr. David Kuter: Link August is SMA Awareness MonthSMA Awareness Page: Link The CheckRare Podcast Network is dedicated to delivering news, education, and expert insights across the rare disease community. From physician interviews and conference coverage to weekly news updates and patient stories, our family of podcasts connects healthcare professionals, researchers, advocates, industry leaders, and patients with the information that matters most. Produced by CheckRare, each series is designed to advance awareness, education, and clinical care in rare diseases. For more information, visit www.CheckRare.com Produced by CheckRare. Explore additional CME activities, physician interviews, podcasts, and rare disease resources at CheckRare.com. Subscribe to the CheckRare Podcast Network for the latest rare disease education, expert interviews, and weekly news. Part of the CheckRare Podcast Network: Trusted conversations, news, education, and expert insights across the rare disease community.

  • 3 Episodes

About

A weekly roundup of the latest news in rare disease. The CheckRare Brief delivers a concise roundup of the week's most important developments in rare diseases. Each episode covers FDA approvals, clinical trial updates, conference highlights, scientific publications, and industry news that matter to healthcare professionals, researchers, advocates, and the rare disease community. Produced by CheckRare, The CheckRare Brief helps you stay informed—in just a few minutes each week.

Information

  • Creator
    CheckRare Media
  • Years Active
    2026
  • Episodes
    3
  • Rating
    Clean
  • Copyright
    © Copyright CheckRare Media
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    The CheckRare Brief