An Endocrine News roundup of the week’s pharmaceutical news, breakthroughs, and general information. *
CABOMETYX® Receives FDA Approval to Treat Neuroendocrine Tumors
On March 26, Exelixis, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved CABOMETYX® (cabozantinib) for the treatment of 1) adult and pediatric patients 12 years of age and older with previously treated, unresectable, locally advanced or metastatic, well-differentiated pancreatic neuroendocrine tumors (pNET); and 2) adult and pediatric patients 12 years of age and older with previously treated, unresectable, locally advanced or metastatic, well-differentiated extra-pancreatic NET (epNET). NET are heterogeneous tumors that arise from the neuroendocrine cells of the digestive tract and other organs, such as the lung and pancreas. Most patients with advanced disease face a poor prognosis.1,2,3,4
“The characteristics of NET vary widely from patient to patient, and very few treatment options have demonstrated the ability to improve outcomes across such a heterogeneous population,” said Jennifer Chan, MD, MPH, study chair for the CABINET trial, clinical director of the Gastrointestinal Cancer Center and director of the Program in Carcinoid and Neuroendocrine Tumors at Dana-Farber Cancer Institute. “It was encouraging to see that cabozantinib resulted in significant delays in disease progression in the CABINET trial — regardless of primary tumor site and grade. This FDA approval marks a meaningful advancement, which may establish an important new treatment option for patients, without limitations based on somatostatin receptor expression and functional status.”
The FDA approval — adding to five previous approvals for CABOMETYX — is based on results from CABINET, a phase 3 pivotal trial evaluating CABOMETYX compared with placebo in two cohorts of patients with previously treated NET: advanced pNET and advanced epNET. Final progression-free survival results were presented at the 2024 European Society for Medical Oncology Congress and published in The New England Journal of Medicine.
In January 2025, the National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology for Neuroendocrine and Adrenal Tumors were updated to include cabozantinib as a category 1 preferred regimen for the majority of well-differentiated advanced NET following specific treatments, and as a category 2A preferred regimen for other forms of advanced NET, depending on tumor grade and different requirements for prior therapy.
“As a company committed to improving the standard of care for people living with advanced, difficult-to-treat cancers, we are proud to bring CABOMETYX to patients with previously treated advanced neuroendocrine tumors,” said Amy Peterson, MD, executive vice president, Product Development & Medical Affairs, and chief medical officer, Exelixis. “I would like to extend our sincere gratitude to the Alliance for Clinical Trials in Oncology for conducting the CABINET trial, to the FDA for their collaboration on the review of this application and to all the patients and physicians who participated in this important study. Looking forward, we are doubling down on our commitment to the NET community as we prepare to initiate our STELLAR-311 pivotal trial examining zanzalintinib versus everolimus in the first half of 2025.”
The safety profile of CABOMETYX observed in each CABINET cohort was consistent with its known safety profile. No new safety signals were identified; however, the incidence of hypertension, regardless of treatment arm, was higher in NET patients compared to other approved tumor types. A majority of patients treated with CABOMETYX required dose modifications or reductions to manage adverse events.
FDA Approves New Treatment for Hyperphagia in Prader-Willi Syndrome
On March 26, Soleno Therapeutics, Inc., announced that the U.S. Food and Drug Administration (FDA) has approved VYKAT XR (diazoxide choline) extended-release tablets, previously referred to as DCCR, for the treatment of hyperphagia in adults and children four years of age and older with Prader-Willi syndrome (PWS).
Soleno, a biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, expects VYKAT XR to be available in the U.S. beginning in April 2025.
“The approval of VYKAT XR is a significant milestone for Soleno and, most importantly, for the PWS community who have had no options to treat the most disruptive aspect of this disease,” said Anish Bhatnagar, MD, chief executive officer of Soleno. “We are deeply grateful to the many individuals with PWS, their caregivers and clinical sites who participated in our trials, the advocacy groups, including FPWR and PWSA | USA, the advocates who have tirelessly supported the approval of VYKAT XR, the FDA for a collaborative review process, and our employees who have been committed to delivering VYKAT XR to those with PWS.”
“The FDA approval of VYKAT XR is an incredible achievement for the entire PWS community,” said Endocrine Society member Jennifer Miller, MD, professor of pediatric endocrinology at the University of Florida, Gainesville, who specializes in treating children and adults with PWS and is a principal investigator in the VYKAT XR clinical development program. “I am excited to have VYKAT XR available to help treat hyperphagia, which is the most life-limiting aspect of PWS. Families of people with PWS have been prisoners in their own homes because of the need to provide constant, eyes-on supervision 24/7 with access to food being completely restricted.”
The FDA approval of VYKAT XR was based on an adequate and well-controlled study and safety data from the comprehensive clinical development program. Efficacy was established during the 16-week randomized withdrawal study period of Study 2-RWP (Study C602-RWP), a Phase 3 multi-center, randomized, double-blind, placebo-controlled trial. Individuals randomized to switch to placebo demonstrated a statistically significant worsening of hyperphagia compared with individuals who remained on VYKAT XR. Prior to participating in the randomized withdrawal period, all individuals received double-blind and/or open-label VYKAT XR for a mean duration of 3.3 years.
VYKAT XR has a well-established safety profile with over four years of data across four double-blind and/or open label studies. The primary safety analyses are based on Study 1 (Study C601) and the most common adverse reactions occurring in greater than or equal to 10% of individuals receiving VYKAT XR and at 2% greater than placebo included hypertrichosis, edema, hyperglycemia and rash.
“Today marks a historic day for the PWS community. The FDA’s approval of VYKAT XR represents a monumental step forward in addressing the longstanding unmet needs of individuals living with PWS and their families,” said Stacy Ward, chief executive officer of the Prader-Willi Syndrome Association | USA. “Our families experience the constant and disruptive challenges of hyperphagia, and VYKAT XR offers hope to so many.”
“This approval is a testament to the power of persistence, science, and advocacy,” said Susan Hedstrom, executive director of the Foundation for Prader-Willi Research. “For years, families and researchers have worked towards a treatment option that truly addresses the complexities of PWS. Today, we take a major step forward in changing the future for individuals navigating hyperphagia associated with PWS.”