Pharma Friday – April 24, 2026

An Endocrine News roundup of the week’s pharmaceutical news, breakthroughs, and general information. *

Sanofi’s Tzield Approved in the US to Delay Onset of Stage 3 Type 1 Diabetes in Young Children

The U.S. Food and Drug Administration (FDA) has approved the supplemental biologic license application for Tzield (teplizumab-mzwv), expanding the indication from eight years and older to as young as one year of age to delay the onset of stage 3 type 1 diabetes in patients diagnosed with stage 2 type 1 diabetes. The approval was granted under a priority review process and is supported by one-year data from the PETITE-T1D phase 4 study (clinical study identifier: NCT05757713), evaluating safety and pharmacokinetics in young children.

“This approval opens an important new chapter in diabetes care for young children with stage 2 type 1 diabetes and their families,” said Kimber Simmons, MD, MS, associate professor of pediatrics at the Barbara Davis Center, Aurora, Colo. “This is especially important because these children are often at the highest risk of progressing quickly and without warning. Delaying the onset of stage 3 type 1 diabetes during the years when management is often most difficult because of a child’s small size and dependence on caregivers could have a truly meaningful impact for families.”

“The autoimmune attack driving this disease often begins early in life, and the burden that autoimmune type 1 diabetes poses in this very young population and their families is significant,” said Christopher Corsico, global head of Development at Sanofi. “This approval underscores the importance of targeting the immune system early in autoimmune type 1 diabetes, aiming to impact its natural progression by delaying the loss of insulin production in the pancreas.”

Tzield is also being reviewed by the FDA for a potential indication to delay the progression of stage 3 type 1 diabetes in patients eight years of age and older recently diagnosed with stage 3 type 1 diabetes.

Tzield is approved in the EU (under the name Teizeild), in the UK, China, Canada, Israel, Saudi Arabia, the UAE, Kuwait, and Brazil to delay the onset of stage 3 type 1 diabetes in adults and pediatric patients eight years and older diagnosed with stage 2 type 1 diabetes. Other regulatory reviews are ongoing. Tzield was previously granted FDA breakthrough therapy designation and orphan drug designation, for medicines that treat rare diseases affecting fewer than 200,000 people in the US.

Novo Nordisk’s Oral Semaglutide Demonstrates Potential to Be the First Oral GLP-1 RA Therapy for Children and Adolescents with Type 2 DNeurocrine Biosciences Presents New Two-Year CRENESSITY® (crinecerfont) Data Showing Sustained Glucocorticoid Dose Reductions While Maintaining Androgen Control in Adults with Classic Congenital Adrenal Hyperplasiaiabetes

On April 23, Novo Nordisk announced positive topline results from PIONEER TEENS, a phase 3a trial evaluating oral semaglutide for type 2 diabetes in children and adolescents aged 10–17 years with type 2 diabetes.

Oral semaglutide demonstrated a superior reduction in HbA_1c (a measure of blood sugar control) over placebo in the trial and showed a well-tolerated safety profile consistent with previous Novo Nordisk semaglutide trials. Oral semaglutide is available today as Rybelsus^® in the EU and US and will be available in the US as Ozempic^® pill soon.

“Over the past two decades, the prevalence of type 2 diabetes among children and adolescents has increased substantially, yet treatment options for this population remain limited, underscoring a significant unmet need. Oral semaglutide has already demonstrated clinically meaningful glycemic efficacy and a well-established safety profile in adults with type 2 diabetes, alongside proven cardiovascular benefits unique to this molecule,” said Martin Holst Lange, chief scientific officer and executive vice president, Research & Development, at Novo Nordisk. “These results from the PIONEER TEENS trial confirm that oral semaglutide is an effective treatment option for children and adolescents with type 2 diabetes who require glycemic control beyond that provided by the current standard of care.”

Type 2 diabetes in children and adolescents is a severe and progressive condition that is strongly associated with increased risks of early mortality in adulthood. Current management for glycaemic control in youth-onset type 2 diabetes remains constrained, and there is an unmet need for more treatment options. In 2021, 14.6 million adolescents were living with type 2 diabetes globally. By 2030, this number is projected to increase to 20.9 million.

Current guidelines recommend metformin and insulin as first-line treatments ; however, metformin is associated with failure in glycaemic control in approximately half of adolescents¹³, and insulin is associated with hypoglycaemia and weight gain^4,5. This is the first clinical trial of an oral GLP-1 RA therapy in this age group, addressing a critical unmet need. Pending regulatory approvals, oral semaglutide has the potential to be the first and only oral GLP-1 RA to demonstrate superior glycemic efficacy versus placebo in children and adolescents with type 2 diabetes, while maintaining the well-established safety profile seen across the semaglutide portfolio.

Neurocrine Biosciences Presents New Two-Year CRENESSITY® (crinecerfont) Data Showing Sustained Glucocorticoid Dose Reductions While Maintaining Androgen Control in Adults with Classic Congenital Adrenal Hyperplasia

Neurocrine Biosciences, Inc. recently announced the first presentation of new two-year data from the Phase 3 CAHtalyst^® Adult study demonstrating sustained, substantial reductions in glucocorticoid (GC) doses in adults with classic congenital adrenal hyperplasia treated with CRENESSITY^® (crinecerfont), with approximately 70% of patients achieving GC doses within the physiologic range. These data build upon previously reported one-year results.

Chronic exposure to supraphysiologic GC doses is associated with cardiometabolic comorbidities, bone density reductions, mental health issues and other long-term health risks that contribute to the cumulative treatment burden faced by patients over their lifetimes. Reducing high-dose, long-term GC exposure represents one of the most important goals in the management of classic congenital adrenal hyperplasia (CAH).

“For decades, the management of classic congenital adrenal hyperplasia has relied exclusively on supraphysiologic glucocorticoid dosing to control adrenal androgen and adrenocorticotropic hormone excess, exposing patients to significant cumulative long‑term risks,” says Sanjay Keswani, MD, chief medical officer, Neurocrine Biosciences. “These two‑year findings demonstrated that CRENESSITY provided durable androgen control while enabling meaningful reductions in glucocorticoid exposure. Importantly, these reductions were sustained over time without new safety or tolerability concerns, supporting CRENESSITY as a long‑term treatment option that advances the standard of care for people living with this complex condition.”

At Month 24 of the study, 69% (103/149) of participants achieved a physiologic GC dose (≤11 mg/m²/day hydrocortisone equivalents), with many participants eliminating nonphysiologic GC types. Of participants originally taking dexamethasone (n=20), 75% switched to a dexamethasone-free regimen, while 62% (37/60) of patients taking more than two doses of hydrocortisone per day were able to eliminate a dose outright. GC dose reductions and regimen changes were achieved without worsening androstenedione levels relative to baseline, indicating that lowering the GC dose was not achieved at the expense of androgen control.

*HCe denotes hydrocortisone equivalents.

Long‑term treatment with CRENESSITY was generally well tolerated, with more than 80% study retention at two years and no new safety signals observed.

“Many years of supraphysiologic glucocorticoid exposure increase the risk for long-term health consequences, which include obesity, diabetes, reduced bone density and psychosocial struggles,” says Richard J. Auchus, MD, PhD, professor of Internal Medicine and Pharmacology, University of Michigan Medical School and Principal Investigator for the CAHtalyst Adult study. “These sequelae significantly impact quality of life and commonly develop with traditional CAH treatment regimens. The two-year findings provide important information on the durable benefit of treatment with CRENESSITY. Providers can confidently incorporate this additional knowledge to guide their management of adult patients with CAH into the future.”

Neurocrine will be sharing additional two-year data across clinical endpoints and outcomes at upcoming medical meetings.

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