Pharma Friday – June 26, 2026

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

Sanofi’s Tzield Approved in the U.S. for Patients with Stage 3 Type 1 Diabetes

On June 13, Sanofi announced that the US Food and Drug Administration (FDA) has granted accelerated approval to Tzield (teplizumab-mzwv) to delay the decline in endogenous (own) insulin production in children aged eight to 17 years recently diagnosed with stage 3 type 1 diabetes. Tzield is not effective as a disease-modifying therapy in non-autoimmune dysglycemic conditions.

“We now have a novel therapy that targets the autoimmune and progressive nature of stage 3 type 1 diabetes,” said Aaron J. Kowalski, PhD, CEO of Breakthrough T1D. “Approximately 64,000 people are diagnosed with T1D every year. We are excited that the approval of Tzield in this indication provides a treatment option for certain patients diagnosed in stage 3 T1D, which is when many start experiencing common symptoms of the disease.”

The approval was supported by data from the PROTECT phase 3 study (clinical study identifier: NCT03875729), evaluating beta cell function as assessed by significantly slowing the decrease in mean C-peptide levels (area under the curve after a four-hour, mixed-meal tolerance test; difference in least-squares means 0.13 pmol/mL; 95% confidence interval: 0.09-0.17; p<0.001) at trial completion, compared to placebo, as well as data from the broader clinical development program that included over 900 patients who received Tzield. Adverse events observed in the PROTECT phase 3 study were consistent with previous studies.

The most common adverse reactions were lymphopenia, vomiting, rash, leukopenia, diarrhea, neutropenia, increased liver transaminase, and headache. Serious events such as cytokine release syndrome and life-threatening cases of viral reactivation have been reported with Tzield. Patients who are immunocompromised are at increased risk for viral reactivation.

This indication is granted under accelerated approval based on evidence of reduced C-peptide decline. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory study(ies).

Medicines that receive accelerated approval are intended to treat serious conditions that fill an unmet medical need, based on a surrogate endpoint reasonably likely to predict clinical benefit. In line with this, the confirmatory BETA-PRESERVE phase 3 study (clinical study identifier: NCT07088068) was initiated and is currently enrolling participants.

“We welcome this accelerated approval by the FDA, which recognizes the potential of Tzield to delay the progression of recently diagnosed stage 3 T1D in children aged eight to 17 years,” said Christopher Corsico, global head of development, Sanofi. “Tzield will now offer a new pathway in the treatment paradigm of stage 3 T1D, one that we hope will further enable healthcare providers in the US to take a more proactive approach to disrupt the underlying autoimmune attack against insulin-producing beta cells.”

Prior to this approval in recently diagnosed stage 3 T1D, in April 2026, the FDA expanded the indication to delay the onset of stage 3 T1D in adults and children eight years and older with stage 2 T1D, to include children aged one year and above. It is also approved to delay the onset of stage 3 T1D in adults and children eight years and older with stage 2 T1D in the UK, the EU (under the name Teizeild), China, Australia, Canada, Israel, Saudi Arabia, the UAE, Kuwait, Brazil and Switzerland. Regulatory reviews are ongoing in other jurisdictions around the world. Tzield was previously designated by the FDA as breakthrough therapy and was granted orphan drug designation, for investigational medicines that treat rare diseases affecting fewer than 200,000 people in the U.S.

TRYNGOLZA® (Olezarsen) Approved by the FDA as the First and Only Treatment to Reduce Triglycerides and the Risk of Acute Pancreatitis in Patients with Severe Hypertriglyceridemia (sHTG)

Ionis Pharmaceuticals, Inc. on June 24 announced that the U.S. Food and Drug Administration (FDA) has approved TRYNGOLZA® (olezarsen) as an adjunct to diet to reduce triglycerides (TG) and the risk of acute pancreatitis in adults with severe hypertriglyceridemia (sHTG: TG greater than or equal to 500 mg/dL). TRYNGOLZA is available in a 50 mg or 80 mg dose and is self-administered once monthly via an autoinjector. sHTG is characterized by an increased risk of acute pancreatitis, which causes debilitating abdominal pain that often leads to repeated and prolonged hospitalization, permanent organ damage and can be life-threatening.

“The approval of TRYNGOLZA marks an historic advance for people who have long struggled to control their dangerously high triglycerides, providing the only approved therapy for sHTG to dramatically lower triglyceride levels and significantly reduce acute pancreatitis events,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. “TRYNGOLZA reflects the strength of Ionis’ innovative science and our commitment to transforming patients’ lives. As our first independent launch in a prevalent disease, this milestone builds on our success in familial chylomicronemia syndrome, a rare form of sHTG, and marks a defining moment for Ionis as we bring our groundbreaking medicines to even more patients in need. We are deeply grateful to the clinical trial participants, investigators, the Ionis team and many others whose dedication made this achievement possible.”

“As a physician, I have seen firsthand how challenging it can be for patients with sHTG to lower their triglycerides below 500 mg/dL, despite background lipid-lowering therapies and lifestyle changes, which leaves them at risk of a devastating and potentially life-threatening acute pancreatitis attack,” said Archna Bajaj, M.D., assistant professor of clinical medicine, University of Pennsylvania. “TRYNGOLZA is a transformational new therapy that showed unprecedented, clinically meaningful outcomes for sHTG, with the potential to redefine the treatment paradigm.”

The FDA approval was based on positive results from the Phase 3 CORE and CORE2 studies, which were published in The New England Journal of Medicine.

In the CORE and CORE2 studies, TRYNGOLZA demonstrated rapid and consistent triglyceride control, lowering fasting triglyceride levels by up to 72% compared to placebo at six months and sustaining those reductions at 12 months. Additionally, TRYNGOLZA significantly reduced acute pancreatitis events by up to 91%. Among patients treated with TRYNGOLZA with baseline and 12-month data, 86% achieved triglyceride levels below 500 mg/dL, a critical threshold for reducing acute pancreatitis risk. The number needed to treat (NNT) over one year to prevent one episode of acute pancreatitis was 20 in the overall cohort and four in patients with triglycerides ≥880 mg/dL and a prior history of acute pancreatitis, indicating a strong clinical benefit across the spectrum of sHTG patients and an exceptional clinical benefit in the highest risk subgroup.¹

Across the clinical program, TRYNGOLZA demonstrated a favorable safety and tolerability profile. The most common adverse reactions in patients with sHTG (incidence ≥2% higher than placebo) were injection site reactions and liver enzyme increases.

“With limited options to lower triglycerides, people living with sHTG often face a constant and real fear that a debilitating acute pancreatitis attack could strike at any time without warning,” said Emily Draud, interim executive director, National Pancreas Foundation. “The availability of TRYNGOLZA for sHTG represents an important new option for this community, offering hope for people who have been waiting for a new treatment to reduce the risk of acute pancreatitis by significantly lowering their triglyceride levels. It also underscores the urgent need for continued innovation and improved care for patients living with this serious condition.”

Ionis is committed to helping people access the medicines they are prescribed and will offer a full suite of services for people prescribed TRYNGOLZA through Ionis Every Step. Ionis Every Step offers personal support, including nutrition information and injection training, insurance support and financial assistance programs. Visit TRYNGOLZA.com for more information.

TRYNGOLZA will be available for sHTG in the U.S. in July.

*Inclusion in Pharma Fridays does not suggest an endorsement by Endocrine News or the Endocrine Society.

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