Abigail Brooks is an assistant managing editor for 

She joined MJH Life Sciences in August 2023 shortly after graduating from Monmouth University where she earned her BA in Communication with a concentration in Public Relations/Journalism and later an MA in Interactive Digital Media. She enjoys traveling, running, and reading books. She can be reached at abrooks@mjhlifesciences.com.

March brought a series of notable developments in endocrinology, with regulatory milestones and clinical trial advances continuing to reshape the treatment landscape across diabetes, obesity, and rare endocrine disorders. From first-in-class therapies to innovations in drug delivery, the month underscored a growing emphasis on improving both clinical outcomes and patient experience.

Several key approvals highlighted progress in areas of unmet need, including the first treatment for cerebral folate deficiency and an expanded indication for setmelanotide in acquired hypothalamic obesity. At the same time, the US Food and Drug Administration (FDA) clearance of a smaller, smartphone-controlled insulin pump and the approval of once-weekly insulin icodec signal a shift toward more convenient, patient-centered diabetes management.

Meanwhile, emerging data from trials of next-generation incretin-based therapies and investigational approaches like islet transplantation continue to push the boundaries of what’s possible in metabolic disease care.

Here’s a look at the key endocrinology headlines from March 2026 you may have missed:

FDA Approves Leucovorin Calcium (Wellcovorin) Tablets as First Cerebral Folate Deficiency Treatment

On March 10, 2026, the FDA approved GSK’s leucovorin calcium (Wellcovorin) tablets for the treatment of cerebral folate deficiency in adult and pediatric patients who have a confirmed variant in the folate receptor 1 gene (CFD-FOLR1), making it the first treatment for the rare genetic condition of cerebral folate deficiency. The approval was based on a systematic review of the published literature on the topic, including published case reports with patient-level information, as well as mechanistic data.

Interim Results from ACCESS Program of Aleniglipron Shows Weight Loss Efficacy

On March 16, 2026, Structure Therapeutics reported positive topline results from the ACCESS clinical program, investigating aleniglipron for patients with obesity or overweight and ≥1 weight-related comorbidity. The ACCESS program includes 44-week data from the phase 2 ACCESS II study and interim data from an ongoing body composition study, as well as the ACCESS open-label extension study.

Diabetes Dialogue: GLP-1 RA News & Updates in March 2026

This episode features discussion around 3 recent announcements from Novo Nordisk, Eli Lilly, and Structure Therapeutics. 

Tegoprubart Enables Insulin Independence in Type 1 Diabetes Islet Transplant Study

On March 16, 2026, Eledon Pharmaceuticals announced updated results from an investigator-initiated trial of 12 adults with long-standing type 1 diabetes undergoing allogeneic islet transplantation who received tegoprubart as part of a calcineurin inhibitor-free immunosuppression regimen. The pilot study, conducted at the University of Chicago Medicine Transplant Institute, showed tegoprubart enabled insulin independence and strong glycemic control in adults with long-standing type 1 diabetes undergoing islet transplantation.

Tegoprubart Improves Glycemic Control Following Islet Transplantation for T1D, With Piotr Witkowski, MD, PhD

FDA Clears MiniMed Flex, A Smaller and Smartphone-Controlled Insulin Pump

On March 18, 2026, the FDA cleared MiniMed’s MiniMed Flex™, a next-generation discreet, smartphone-controlled insulin pump designed to fit seamlessly into everyday life. The product is about half the size of the MiniMed™ 780G pump and roughly the size of 2 stacked insulin vials. It is cleared for individuals ≥ 7 years of age with type 1 diabetes and for individuals ≥ 18 years of age with insulin-requiring type 2 diabetes.

Retatrutide Delivers A1C Reduction, Weight Loss in Phase 3 TRANSCEND-T2D-1 Trial

On March 19, 2026, Eli Lilly and Company announced positive topline results from the phase 3 TRANSCEND-T2D-1 trial assessing the efficacy and safety of retatrutide, an investigational first-in-class GIP, GLP-1 and glucagon triple hormone receptor agonist, as an adjunct to diet and exercise in people with type 2 diabetes and inadequate glycemic control. In the study, retatrutide met the primary and all key secondary endpoints, delivering superior A1C reduction and weight loss at 40 weeks compared to placebo, using both the efficacy and treatment-regimen estimands.

FDA Approves Setmelanotide (Imcivree) for Acquired Hypothalamic Obesity

On March 19, 2026, the FDA approved an expanded indication for setmelanotide (Imcivree) to treat acquired hypothalamic obesity in adults and pediatric patients aged 4 years and older. The approval makes setmelanotide the first and only FDA-approved therapy for this rare condition, which is characterized by accelerated and sustained weight gain following injury to or dysfunction of the hypothalamus.

FDA Approves Insulin Icodec (Awiqli) as First Once-Weekly Basal Insulin for Type 2 Diabetes

On March 26, 2026, the FDA approved insulin icodec-abae (Awiqli) injection 700 units/mL, the first and only once-weekly basal insulin available in the US, as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes. The decision makes insulin icodec the first new class of basal insulin to reach US patients in more than 2 decades.

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March 2026 endocrinology updates include key FDA approvals, advances in diabetes and obesity therapies, and innovations in insulin delivery and rare disease care.

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7 Endocrinology Headlines You Missed in March 2026

Clinical Presentation of Hypercortisolism

In this episode, ‘Clinical Presentation of Hypercortisolism,’ the expert faculty discuss the evolving understanding of hypercortisolism beyond its classic clinical presentation, emphasizing its variable manifestations and significant cardiometabolic impact. While traditional teaching has focused on recognizable Cushingoid features such as moon face, central obesity, and dorsocervical fat pads, the panelists highlight that many patients, particularly those with resistant hypertension or diabetes, do not exhibit these hallmark signs. This variability contributes to under recognition in clinical practice, especially among cardiologists who may not routinely consider hypercortisolism in patients without physical features.

The expert panel further explores how excess cortisol functions as a metabolic condition along a continuum, rather than a binary diagnosis. They discuss how varying levels of cortisol dysregulation can lead to a range of adverse outcomes, including hypertension, hyperglycemia, cardiovascular disease, atrial fibrillation, and bone loss. The faculty also note emerging evidence suggesting that cortisol contributes to insulin resistance, visceral fat accumulation, and muscle effects, reinforcing its broad systemic impact.

Importantly, the panelists underscore that individual responses to cortisol excess can differ widely, making hypercortisolism a highly heterogeneous condition. Overall, the discussion highlights the need for increased awareness of non-classic presentations and a greater appreciation of hypercortisolism as a clinically relevant contributor to cardiometabolic disease.

The next episode in this series, ‘Evolving Hypercortisolism Screening and Diagnosis,’ features the panelists discussing how emerging data are reshaping screening approaches for hypercortisolism, particularly in patients with resistant hypertension. The panel highlights a shift toward simpler diagnostic strategies and increased recognition of potentially reversible endocrine causes of cardiovascular disease.

This episode explores how hypercortisolism extends beyond classic Cushingoid features, highlighting its variable presentation and role as a continuum of cortisol-driven cardiometabolic disease that is often underrecognized in clinical practice.

Video Series

Neurological and Psychiatric Manifestations in Wilson Disease

In this episode, 'Neurological and Psychiatric Manifestations in Wilson Disease,' the panelists explore the complex and often nonspecific neurological and psychiatric landscape of Wilson disease. Dr. Peter Hedera describes the condition as a "great imitator" because it frequently mimics more common movement disorders such as Parkinson's disease, dystonia, and essential tremor. This clinical overlap often leads to significant diagnostic delays, sometimes lasting two years or longer. The experts stress that a high index of suspicion is required for any patient under the age of 40 or 50 presenting with unexplained movement symptoms.

The discussion also delves into the psychiatric side of the disease, noting that approximately 10% of patients may present with psychosis. This can create a diagnostic trap where neuroleptic treatments for psychiatric symptoms cause extrapyramidal side effects that further mask the underlying Wilson disease. In pediatric populations, the panelists shift the focus to neuropsychiatric indicators, such as sudden changes in school performance, cognitive shifts, or hyperkinetic movements like chorea.

Finally, the panel addresses the overlap between hepatic and neurological symptoms. While adults frequently show signs of both, pediatric patients under age 10 rarely exhibit neurological symptoms. However, that frequency increases to roughly 30% in adolescents. The experts conclude that while the coexistence of elevated liver enzymes and movement disorders is a major red flag, clinicians must rely on thorough laboratory confirmation to navigate these overlapping clinical presentations and ensure the best patient outcomes through early diagnosis.

The next episode in this series, 'Understanding Pathophysiology and Copper Transport in Wilson Disease,' features the panelists advancing their conversation on Wilson disease and focusing on the genetic and cellular mechanisms of the condition. Dr. Peter Hedera explains how mutations in the ATP7B gene disrupt copper homeostasis, leading to toxic accumulation in the liver and brain.

Panelists discuss the often misdiagnosed neurological and psychiatric manifestations of Wilson disease, highlighting its overlap with common movement and mental health disorders, the need for high clinical suspicion, and the importance of early diagnosis through careful evaluation of overlapping symptoms.

EPI Across Disease States: Identifying High-Risk Patients

This episode focuses on the full spectrum of clinical conditions that warrant high suspicion for EPI. Jennifer Geremia leads with the primary pancreatic risk factors: chronic pancreatitis (with or without confirmatory imaging), recurrent acute pancreatitis regardless of etiology, prior pancreatic surgeries such as the Whipple procedure, cystic fibrosis, and pancreatic cancer. She notes that patients with these primary conditions are frequently under-screened and undertreated, even when the risk is well established.

The discussion expands to non-primary pancreatic conditions. The panelists highlight that celiac disease, type 2 diabetes, and Crohn's disease should also raise EPI suspicion—not because of direct pancreatic damage, but due to the shared mechanisms of malabsorption and maldigestion. Lifestyle factors such as smoking and heavy alcohol use are identified as important aggravators in the right clinical context.

A particularly valuable clinical insight emerges around severe acute pancreatitis and disconnected duct syndrome. Sarah Enslin notes that patients labeled simply as "acute" may actually have significant parenchymal loss and thus EPI—yet this is often missed because the framing remains acute rather than chronic. Dr. Kaul clarifies that typical mild acute pancreatitis does not routinely lead to EPI; it is the severe cases with significant parenchymal destruction that carry the highest risk.

The episode closes with a discussion of surgical and procedural risk factors: bariatric surgery (especially gastric bypass), small bowel resection for any cause (including IBD), and all foregut surgeries. The panel also spotlights pancreatic cancer patients—both surgical and non-surgical—as a special and complex high-risk population in whom EPI can be easily obscured by oncologic therapies and their side effects.

EPI Unmet Needs: Barriers to Diagnosis and Long-Term Consequences

," the panel explores the significant gaps in EPI diagnosis and the downstream consequences of untreated disease on patient quality of life and health.

Panelists discuss the wide range of pancreatic and non-pancreatic conditions that increase suspicion for exocrine pancreatic insufficiency to improve identification of high-risk patients.

James Murrough, MD, PhD, from the Icahn School of Medicine at Mount Sinai.

Kv7 Channel Modulator Azetukalner May Improve Anhedonia, With James Murrough, MD, PhD

A phase 2 trial evaluating the Kv7 potassium channel opener azetukalner in individuals with 

suggests potential clinical benefit despite failing to meet its primary neuroimaging endpoint. The findings, presented at the Anxiety and Depression Association of America (ADAA) 2026 meeting in Chicago, highlight both the potential advantages and ongoing hurdles in biomarker-based antidepressant development.

The phase 2, randomized, double-blind, placebo-controlled trial enrolled 60 adults with MDD and anhedonia who were randomized to receive either azetukalner 20 mg daily (n = 29) or placebo (n = 31) for 8 weeks. The primary endpoint was the change in ventral striatum activity during a reward task from baseline to week 8. Secondary outcomes included changes in depression severity and anhedonia using the Montgomery-Åsberg Depression Rating Scale (MADRS) and Snaith-Hamilton Pleasure Scale (SHAPS), respectively.

 spoke with investigator James Murrough, MD, PhD, from the Icahn School of Medicine at Mount Sinai, on the rationale for the trial. He said the research stemmed from preclinical work identifying Kv7 potassium channels, also known as KCNQ channels, as regulators of stress resilience and reward circuitry. In animal models, upregulation of these channels appeared to protect against depression-like behaviors, prompting interest in pharmacologic modulation as a novel therapeutic strategy.

“We [were] focused on anhedonia… [because it’s] a core symptom of depression,” Murrough said. “Most people with depression have this to some degree…it's thought that there are specific brain systems and behavioral tendencies that underlie anhedonia, which might be particularly amenable to targeting with specific treatments.”

Although the trial did not demonstrate a significant difference in ventral striatal activation between azetukalner and placebo, clinical measures showed a consistent pattern favoring active treatment. Improvements in both depression severity and anhedonia scales numerically favored azetukalner, and exploratory outcomes followed a similar trend. Adverse event-related discontinuation rates were low and comparable between groups.

Murrough noted that the negative neuroimaging result should be interpreted cautiously, particularly given the evolving role of biological endpoints in psychiatric trials. The use of functional imaging as a primary outcome was intended to test whether objective brain-based measures could serve as sensitive indicators of treatment response, potentially enabling faster “go/no-go” decisions in drug development.

“This is what's called the fast-fail approach,” Murrough said. “We'd like a biologically informed endpoint to quickly tell researchers that this target, although it may have been a good idea… [is] really not viable in humans for whatever reason. That was why it was the primary [endpoint], because we wanted to really stick to that…at the end of the day, in psychiatry, our best bet is looking for the pattern of clinical change. We had really no effect on the brain, but…this pattern of clinical changes was encouraging. What does this mean? To me, the use of brain imaging hasn't evolved to the point in terms of clinical trial utility, that if there's a null finding, then that tells us that there's no point in pursuing this.”

Murrough also pointed to ongoing analyses examining more complex measures of brain connectivity rather than single-region activation. Prior work with another Kv7 channel opener demonstrated that changes in functional connectivity between the ventral striatum and large-scale networks, including default mode regions, correlated with symptom improvement. Preliminary analyses from the current dataset appear to show similar patterns, though these findings are not yet published.

“Our specific questions about how it acted in the brain turned out not to be correct,” Murrough said. “Again, it was a small study, and some of the key outcomes didn't meet our statistical endpoint, so it's still considered preliminary. But when you looked at the totality of changes and the magnitude of some of the changes, particularly on this anhedonia metric, we were very encouraged that we [might] be onto something in terms of medicines that target this KCNQ channel. The study we're presenting at the poster at the meeting may be a small step on that journey.”

A relevant disclosure for Murrough includes Janssen Scientific Affairs, LLC

Fremont R, Neukam P, Govindarajulu U, et al. A Randomized, Controlled Trial Of The Novel Kv7 Channel Opener Azetukalner In Individuals With Major Depressive Disorder And Anhedonia. Poster presented at the 2026 Anxiety and Depression Association of America (ADAA) 2026 meeting in Chicago from April 9 – 11.

Phase 2 data presented at ADAA 2026 suggest azetukalner may improve anhedonia in MDD, even though the trial did not meet its primary neuroimaging endpoint.

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Alex Hillenbrand is an assistant editor for 

. She earned her BA in English from Lafayette College in 2023 and went on to write for pop culture, entertainment, and sports media outlets before joining MJH Life Sciences in 2025. In her free time, Alex enjoys cooking, going on walks, and meeting new people. Contact her at mjhlifesciences.com.

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Michelle O'Donoghue, MD, is the McGillycuddy-Logue Endowed Chair in Cardiology at Brigham and Women's Hospital and associate professor at Harvard Medical School

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Victoria Johnson joined MJH Life Sciences in 2020 and has written for NeurologyLive, CGTLive, and is now an assistant managing editor for HCPLive and RheumatologyLive. She doesn’t have enough time for her numerous hobbies. You can reach her at vjohnson@mjhlifesciences.com.

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2-year results from phase 3 APPLAUSE-IgAN demonstrate slower eGFR decline, reduced composite kidney failure, and proteinuria in IgAN patients treated with iptacopan. 


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APPLAUSE-IgAN: Iptacopan (Fabhalta) Exhibits 49.3% Slower eGFR Decline in IgAN Patients Than Placebo

Panelists discuss the signs, symptoms, diagnostic delays, and classification of obstructive hypertrophic cardiomyopathy, including how it differs from other forms and its implications for clinical management.

Recognizing and Classifying Obstructive Hypertrophic Cardiomyopathy

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Diagnosing Hypertrophic Cardiomyopathy: Challenges and Gaps in Clinical Practice

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Intravenous Iron — When to Escalate, Formulation Choice, and Safety Considerations 

The Q1 recap for pulmonology spotlights regulatory and clinical advances across asthma and pulmonary fibrosis.

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March 2026 saw new data from pivotal clinical trials and new expert conversations on HCPLive.

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7 Endocrinology Headlines You Missed in March 2026

Clinical Presentation of Hypercortisolism

Neurological and Psychiatric Manifestations in Wilson Disease

EPI Across Disease States: Identifying High-Risk Patients

Kv7 Channel Modulator Azetukalner May Improve Anhedonia, With James Murrough, MD, PhD