FDA Approves New Treatments for Hormonal Disorders in 2025

The US Food and Drug Administration (FDA) has made significant strides in the treatment of hormonal disorders throughout 2025. While the year lacked groundbreaking research breakthroughs, the agency has approved several new therapies and revised existing ones, enhancing treatment options for conditions ranging from growth hormone deficiency to adrenal insufficiency.

Key Approvals and Updates

On July 28, 2025, the FDA granted approval for lonapegsomatropin-tcgd, a new treatment for adult growth hormone deficiency (GHD). This approval stemmed from the phase 3 foresiGHt clinical trial, which demonstrated the drug’s superiority in reducing trunk fat and increasing lean body mass compared to a placebo. Notably, the treatment was found to be safe and well-tolerated throughout the trial, with no participants discontinuing use due to adverse effects.

In a significant step for patients with acromegaly, Crinetics Pharmaceuticals announced on September 25, 2025, that the FDA approved paltusotine as the first once-daily oral treatment for adults who have not responded adequately to surgery or cannot undergo surgical intervention. This approval was based on findings from the phase 3 PATHFNDR-1 and PATHFNDR-2 trials, which highlighted the treatment’s rapid onset and consistent efficacy.

Further expanding treatment options, the FDA approved an oral hydrocortisone solution on May 28, 2025, specifically for pediatric patients aged five and older with adrenocortical insufficiency. This formulation addresses challenges in administration and dosing accuracy, catering to patients who struggle with traditional pill intake. Adrenal insufficiency is a rare, yet serious condition that can lead to significant health complications.

Revisions and New Guidelines

On November 10, 2025, the FDA and the US Department of Health and Human Services announced plans to revise safety labeling for menopausal hormone therapy (MHT). Following a reevaluation of data from the original Women’s Health Initiative (WHI) trial conducted in the 1990s, officials recognized that the demographic represented did not accurately reflect the average age of menopause onset in the US. Consequently, the FDA proposed removing certain warnings related to cardiovascular disease, breast cancer, and dementia, while incorporating WHI data for women aged 50-59 years.

Significant changes in testosterone product labeling were also initiated on March 3, 2025, based on findings from the phase 4 TRAVERSE trial, which prompted labeling adjustments regarding increased blood pressure risks associated with these products. The FDA suggested retaining the “limitation of use” language for age-related hypogonadism and removing certain warnings previously linked to cardiovascular risks.

Dr. Mohit Kera engaged with Urology Times in December 2025 to discuss the implications of the FDA panel on testosterone replacement therapy (TRT) for men. This panel marked a pivotal moment, prompting a reassessment of long-standing views on testosterone use, including discussions on outdated labeling language and the drug’s classification as a Schedule III controlled substance.

The 2025 American Urological Association Annual Meeting featured the release of new guidelines addressing the Genitourinary Syndrome of Menopause. These guidelines, produced in collaboration with the Society of Urodynamics, Female Pelvic Medicine & Urogenital Reconstruction and the American Urogynecologic Society, highlighted the symptoms and physical changes associated with declining hormone levels during menopause.

In a related development, the phase 4 CATALYST trial revealed that mifepristone significantly improved glycemic control and reduced body weight in patients suffering from hypercortisolism and difficult-to-control type 2 diabetes. During the trial, which included a total of 136 patients, those receiving mifepristone showed considerable improvements compared to the placebo group. Despite its efficacy, nearly 49% of patients discontinued therapy, compared to just 18% in the placebo cohort.

As 2025 concludes, the ongoing advancements and regulatory updates in hormonal disorder treatments signal a progressive shift in how these conditions are managed, promising improved outcomes for patients worldwide.