Novo Nordisk Submits FDA Application for 7.2 mg Dose of WEGOVY Injection
Novo Nordisk has announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for a higher, investigational 7.2 mg dose of its once-weekly semaglutide injection, marketed as WEGOVY, for chronic weight management in adults with obesity. This submission is notable because it is being reviewed under the FDA’s Commissioner’s National Priority Voucher (CNPV) pilot program, an expedited pathway designed to accelerate the review of products addressing major national health priorities, such as the growing public health issue of obesity.
The application is supported by robust data from the 72-week Phase 3 STEP UP trial, a randomized, double-blind, active- and placebo-controlled study involving 1,407 adults with obesity (excluding those with diabetes). The trial demonstrated that the 7.2 mg dose was superior to both the currently approved 2.4 mg dose and placebo. Specifically, participants on the 7.2 mg dose achieved an average body weight loss of 20.7% after 72 weeks, compared to 17.5% for the 2.4 mg dose. Furthermore, a significantly higher proportion of participants on the 7.2 mg dose (over 30%) achieved a 25% or greater weight loss.
While the higher dose showed greater efficacy, the safety data indicated that gastrointestinal adverse events were more common with the 7.2 mg dose compared to the 2.4 mg dose. Serious adverse events, however, were less frequent in the 7.2 mg group compared to the 2.4 mg group. If approved, the 7.2 mg injection would provide healthcare professionals and patients with a new option for greater weight-loss potential, further underscoring the therapeutic potential of the semaglutide molecule. Novo Nordisk anticipates a regulatory decision within a short timeframe due to the CNPV program.
Ascendis Receives FDA Extension on TransCon CNP Review for Pediatric Achondroplasia
Ascendis Pharma A/S announced that the U.S. Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) of TransCon CNP (navepegritide), its investigational therapy for children with achondroplasia. The Prescription Drug User Fee Act (PDUFA) goal date has been moved back by three months, from the original date to February 28, 2026.
The extension was prompted by the FDA’s notification that information submitted by Ascendis on November 5, 2025, related to the post-marketing requirement (PMR), constituted a major amendment to the NDA. This administrative classification necessitated the three-month extension to allow the agency sufficient time for a full review of the revised study protocol.
Ascendis leadership confirmed that the post-marketing requirements were the lone item for discussion at their late-cycle meeting with the FDA, and they have responded to all outstanding requests, including the revised protocol for the post-marketing study. Importantly, analysts and company management have indicated that the extension does not appear to be related to concerns about the core clinical data package or the chemistry, manufacturing, and controls (CMC) aspects of TransCon CNP.
The company is committed to working closely with the FDA to finalize all regulatory requirements and bring this innovative treatment, which uses Ascendis’ proprietary TransCon technology to deliver C-type natriuretic peptide (CNP) sustainably, to children with achondroplasia in the U.S. as quickly as possible. The PDUFA date extension temporarily delays the expected commercial launch but does not diminish confidence in the therapy’s potential approval.
Otsuka Wins FDA Accelerated Nod for VOYXACT in Primary IgAN Patients at Risk of Progression
Otsuka Pharmaceutical Co., Ltd. has received Accelerated Approval from the U.S. Food and Drug Administration (FDA) for VOYXACT (sibeprenlimab-szsi) to reduce proteinuria in adults with primary IgAN who are at risk for disease progression. This approval marks VOYXACT as the first and only therapy in the U.S. to specifically target and block A-PRoliferation-Inducing-Ligand (APRIL), a key factor implicated in the pathogenesis of IgAN by promoting the production of pathogenic galactose-deficient IgA1 (Gd-IgA1).
The accelerated approval for IgAN treatment was granted based on significant reductions in proteinuria from the nine-month interim analysis of the pivotal Phase 3 VISIONARY trial, one of the largest clinical trials ever conducted in IgAN. VOYXACT achieved a statistically significant, placebo-adjusted treatment effect of 51% reduction in proteinuria at nine months. The FDA recognizes proteinuria reduction as a surrogate endpoint reasonably likely to predict clinical benefit in delaying progression to kidney failure.
As a condition of the accelerated pathway, continued approval for VOYXACT is contingent upon verification of clinical benefit in a confirmatory trial. The ongoing VISIONARY study will provide the full 24-month results, including data on the slowing of kidney function decline, as measured by estimated glomerular filtration rate (eGFR), expected in early 2026 and intended to support traditional FDA approval. The therapy is administered as a subcutaneous injection once every four weeks. VOYXACT provides a novel, targeted treatment option for IgAN patients, a serious kidney disease that can often progress to kidney failure, offering renewed hope for managing this complex condition.
Sarepta Cleared to Begin ENDEAVOR Cohort 8, Studying Improved Immunosuppression for ELEVIDYS in Non-Ambulatory DMD
Sarepta Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved dosing in Cohort 8 of the Phase 1b ENDEAVOR (Study 9001-103) trial. This new cohort is designed to evaluate an enhanced immunosuppressive regimen for ELEVIDYS (delandistrogene moxeparvovec-rokl), the company’s gene therapy for Duchenne muscular dystrophy (DMD), in non-ambulatory individuals.
The primary objective of Cohort 8 is to mitigate the risk of acute liver injury (ALI) and acute liver failure (ALF), which are known risks associated with AAV gene therapy, particularly in the non-ambulatory population. The enhanced regimen will integrate the drug sirolimus into the existing immunosuppression protocol, with dosing starting 14 days prior to the ELEVIDYS infusion and continuing for 12 weeks post-administration.
The cohort is expected to enroll approximately 25 non-ambulatory participants in the U.S. Key primary endpoints will assess the incidence of ALI and the level of ELEVIDYS-dystrophin expression at 12 weeks. Sarepta intends to initiate enrollment before the end of the year and anticipates completing primary endpoint data collection in the second half of 2026. The data gathered from Cohort 8 will be crucial, informing future discussions with the FDA regarding the potential resumption of commercial dosing of ELEVIDYS for this underserved non-ambulatory patient population. This move reflects Sarepta’s commitment to refining the safety profile of its gene therapy while striving to expand access to all individuals living with this fatal, progressive rare disease.
IMFINZI Receives US Approval as the Only Immunotherapy Indicated for Perioperative Treatment in Early Gastric and Gastroesophageal Cancer
AstraZeneca announced that IMFINZI (durvalumab), in combination with standard-of-care FLOT chemotherapy, has received US FDA approval for the perioperative treatment of adult patients with resectable, early-stage, and locally advanced (Stages II, III, IVA) gastric and gastroesophageal junction (GEJ) cancers. This landmark decision establishes IMFINZI as the first and only immunotherapy approved for use in the perioperative setting for these specific early-stage upper GI cancers, where cure is the primary therapeutic goal.
The approved regimen is comprehensive, involving neoadjuvant IMFINZI plus chemotherapy before surgery, followed by adjuvant IMFINZI plus chemotherapy, and concluding with IMFINZI monotherapy. The approval was based on compelling event-free survival (EFS) and overall survival (OS) data from the pivotal Phase 3 MATTERHORN trial.
In the MATTERHORN trial, the IMFINZI-based perioperative regimen demonstrated a 29% reduction in the risk of disease progression, recurrence, or death (EFS) compared with chemotherapy alone. The final OS analysis showed that the IMFINZI-FLOT combination reduced the risk of death by 22% compared with the FLOT-only arm, with an estimated 69% of patients treated with the immunotherapy regimen alive at 3 years, compared with 62% in the control arm. Notably, the OS curves continued to separate over time, signaling a durable and increasing benefit, regardless of PD-L1 status. This approval, which was granted Priority Review, establishes a new clinical standard of care for patients aiming for curative treatment of early gastric and GEJ cancers.
