FDA Approves Leuprolide Acetate for Central Precocious Puberty


The injection therapy has shown capability to stop or even reverse the progression of early puberty in patients with the rare disease.


The US Food and Drug Administration (FDA) has approved the New Drug Application (NDA) for leuprolide acetate (FENSOLVI) injectable suspension for the treatment of pediatric patients ≥2 years old with central precocious puberty (CPP).

CPP, a rare disease defined by puberty onset prior to age 8 in girls and age 9 in boys, drives patient risk of significantly short stature as adults as well as issues in social, psychological, and emotional settings.

The one in 5000-10,000 children who develop CPP are prone to lower self-esteem, stress, anxiety, and depression. The leuprolide acetate injection—among the most common therapies for CPP—is a 45 mg therapy dosed every 6 months.

The NDA granted to Tolmar Pharmaceuticals was based on submitted findings of a multicenter, open-label, single arm phase 3 trial assessing the efficacy, safety, and pharmacokinetics of the therapy for injectable suspension in 64 children with central (gonadotropin-dependent) precocious puberty.

The trial achieved its primary endpoint, with 87% of children achieving a serum luteinizing hormone concentration of <4 IU/L at 6 months post-injection. Treated patients also demonstrated suppressed sex hormones at pre-pubertal levels, as well as stopped or even reversed progression of clinical signs of puberty.

Mild to moderate treatment emergent adverse events included injection site pain (31%), nasopharyngitis (22%), and fever (17%).

Clinicians praised the therapy’s shown clinical benefits, as well as the approval of another option in treating CPP.

"Children with CPP require treatment for several years and missing treatment or stopping treatment too soon may lead to significant short stature and misalignment between chronological age and physical and emotional development," Karen Klein, MD, Associate Clinical Professor at the Rady Children's Hospital, University of California San Diego, said in a statement. "Fensolvi offers treating physicians and their patients with CPP a safe and effective treatment option that is administered twice a year with a small injection volume that has the potential to improve compliance."

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