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.

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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.

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Byron Wade, a patient with IgA nephropathy (IgAN), discusses his personal diagnostic journey and a recent study looking at other patients’ experiences.

IgAN is characterized by the accumulation of IgA antibodies in the kidneys. In the early stages, patients with IgAN have no symptoms. The first sign of this condition may be blood in the urine. End-stage kidney disease may develop if the disease is not treated properly. In most instances, the cause of this condition is unknown; however, certain disorders have been linked with IgAN, such as cirrhosis of the liver, celiac disease, and HIV infection. The condition can also often be triggered by a viral illness.

Mr. Wade explains how at the time of his diagnosis in 2020, the most difficult part was the unknown. In 2020, IgAN was an even lesser known condition than it remains today and no approved treatments existed. While management with steroids and blood pressure medications were the initial treatment plan, Mr. Wade eventually required a kidney transplant in 2023. His hope for patients with IgAN moving forward, with greater awareness of the condition and several treatment options approved or in late stage development, is that other patients are able to avoid his struggles.

A study presented at GlomCon highlighted the experiences of patients with IgAN throughout different stages of their disease, of which Mr. Wade was a co-author.

Key findings showed that a vast majority of patients recalled experiencing symptoms prior to diagnosis, but waited about 9 months before seeking. Patients reported that typically abnormal lab results were usually what led to referral to a nephrologist, but these wait times could be up to 12 months. Additionally, many patients leave their appointments feeling confused and wanting clearer guidance on their disease and treatment options. Patients also indicated large gaps between what they value and how current treatments perform.

CHAPTERS
Introduction 00:00
Most Difficult Part of Diagnosis 00:36
Diagnostic Journey 1:48
Advice for Newly Diagnosed Patients 4:02
Effect on Daily Life 4:37
What Physicians Should Know 5:18
Patient Experiences With IgAN 6:03
Take Home Message 7:08

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Gaia Coppock, MD, Nephrologist and Associate Professor of Clinical Medicine at the University of Pennsylvania, discusses patient experiences with IgA nephropathy (IgAN).

IgAN is characterized by the accumulation of IgA antibodies in the kidneys. In the early stages, patients with IgAN have no symptoms. The first sign of this condition may be blood in the urine. End-stage kidney disease may develop if the disease is not treated properly. In most instances, the cause of this condition is unknown; however, certain disorders have been linked with IgAN, such as cirrhosis of the liver, celiac disease, and HIV infection. The condition can also often be triggered by a viral illness.

A study presented at GlomCon highlights the experiences and emotions of patients with IgAN throughout different stages of their disease, identifying unmet needs and opportunities to improve patient care and outcomes.

A total of 126 patients with biopsy-confirmed IgAN in the US were included in the study. Key findings showed that 88% of patients recalled experiencing symptoms, such as fatigue, hematuria, hypertension, edema, and back/stomach pain before their IgAN diagnosis, but waited an average of 9 months before seeking care with their primary care physician. Patients reported that typically abnormal lab results, rather than symptoms, were the trigger for a referral to a nephrologist, though referral wait times could be up to 12 months. Dr. Coppock explains how the vagueness of symptoms and lack of rare disease education is often the cause of these diagnostic delays.

Additionally, many patients leave their appointments feeling confused and wanting clearer guidance and 57% would prefer to receive information about IgAN from their nephrologist.
Patients also indicated large gaps between what they value and how current treatments perform, with less than half feeling their current therapies effectively stop symptoms, slow loss of kidney function, delay time to dialysis, and reduce proteinuria.

CHAPTERS
Introduction 00:00
Long Delays in Diagnosis 00:31
Improving Patient-Physician Communication 1:43
What Physicians Should Know 2:46
What Patients Should Know 3:40
Gaps in Patient Expectations and Treatments 4:26
Improving Patient Experiences 5:20
Take Home Message 5:37

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Rare Endocrine Exchange features interviews with leading endocrinologists, researchers, and patient advocates discussing the latest advances in rare endocrine disorders. Topics include emerging therapies, clinical research, conference coverage, and practical insights to help improve patient care.

Hosted by Dr. Margarita Ochoa-Maya and produced by CheckRare, Rare Endocrine Exchange connects the endocrine community with the latest science and expert perspectives that can improve clinical care.

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Rare endocrine disorders can be difficult to recognize, challenging to diagnose, and even harder for patients to navigate. For many patients, the journey to an accurate diagnosis can take years.

Rare Endocrine Exchange, a new podcast from CheckRare, aims to change that.
Hosted by clinical endocrinologist Margarita Ochoa-Maya, MD, the series brings together physicians, researchers, patients, advocates, and other leaders across the rare endocrine community to explore the science, challenges, and real-world experiences behind these disorders.

In this inaugural episode, Dr. Ochoa-Maya introduces the Rare Endocrine Exchange and discusses why greater awareness and collaboration are needed across rare endocrine diseases. Future episodes will explore some of the most important issues facing the community, including:

• Why are rare endocrine disorders diagnosed so slowly?
• Can AI help (or hurt) rare endocrine care?
• What do new guidelines mean for patients with rare endocrine diseases?
• The latest research and clinical advances
• Patient and caregiver experiences
• Advocacy and access to care
• Emerging treatments and clinical trialsThe goal is simple: to create a place where the rare endocrine community can learn from one another.

Whether the conversation is with a leading researcher, a practicing clinician, a patient, or an advocate working to change the system, Dr. Ochoa-Maya will explore the issues that matter most to the people living with and caring for rare endocrine disorders.

This is the Rare Endocrine Exchange.

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Matt Trudeau, President of ITF Therapeutics, discusses the importance of patient listening in the development of Duvyzat (givinostat) for Duchenne muscular dystrophy (DMD).

DMD is a rare genetic, neuromuscular condition characterized by progressive muscle wasting. Symptoms include progressive weakness and atrophy of both skeletal and heart muscle. Early signs may include delayed ability to sit, stand, or walk and difficulties learning to speak. DMD is caused by genetic changes in the DMD gene.

Just ahead of the second anniversary of its commercial launch, ITF Therapeutics’ givinostat was prescribed to its 2,000th patient in the US. Givinostat is a histone deacetylase (HDAC) inhibitor approved for patients ages six and older with DMD.

Mr. Trudeau highlights the importance of earning trust in the rare disease community, something he explains starts with listening to patients and patient advocates. From attending patient conferences to hiring patient advocacy roles and meeting with advocacy organizations, ITF Therapeutics strives to keep patients at the forefront of their treatment development process.

Improving communication with patients, developing effective educational tools, and removing non-clinical barriers while improving accessibility are other initiatives that Mr. Trudeau deems crucial.

While there is still work to be done in DMD, other indications currently in the pipeline for givinostat include Becker muscular dystrophy and polycythemia vera.

CHAPTERS
Introduction 00:00
DMD Overview 00:34
Development of Givinostat 1:33
Lessons Learned Since Launch 3:19
Other Indications in the Pipeline 7:03
Take Home Message 8:42

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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/

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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.

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