FDA/CDC

Novel oral testosterone replacement therapy headed to FDA


 

Marius Pharmaceuticals has submitted a new drug application (NDA) to the Food and Drug Administration for Kyzatrex, an oral testosterone replacement therapy (TRT).

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With this NDA, the company is seeking approval for Kyzatrex as a treatment for adult men with primary and secondary hypogonadism, also known as testosterone deficiency. Marius has requested a priority review that, if accepted, would result in an anticipated 6-month review period.

Current treatment options for hypogonadal men consist of therapies with safety concerns, such as cardiovascular and metabolic risks, that make patient adherence to treatment very low.

Kyzatrex is a novel oral formulation of testosterone undecanoate administered twice daily in a soft gelatin capsule.

“TRT remains a therapeutic challenge because there are worrisome and conflicting data related to increased cardiovascular disease risk, which has special relevance to high-risk diabetic populations,” Paul S. Jellinger, MD, professor of clinical medicine at the University of Miami, told this news organization. Furthermore, “injectable depot testosterone may be associated with peak supraphysiological levels and a substantial increase in hemoglobin. Topical testosterone offers more stable levels without a peak and trough, but in some men achieving physiologic levels may be difficult.”

The NDA is supported by results from a 6-month treatment extension of the pivotal phase 3 MRS-TU-2019 study (NCT04467697). Final results from this study have not been presented, but the company wrote in a press release that the results will be published some time in 2021.

They further reported that Kyzatrex was well tolerated by patients, with more than 96% of study participants completing 90 days of treatment in the pivotal phase 3 study. Study patients achieved average testosterone levels in the normal range.

Across the pooled phase 3 trials, the most frequent treatment-related treatment-emergent adverse event (TEAE) was hypertension, and no serious TEAEs were considered treatment related.

“We are extremely proud to have generated compelling efficacy and safety data in our phase 3 trials,” said Om Dhingra, PhD, cofounder and CEO of Marius. “We look forward to continuing to work collaboratively with the FDA on the review of our application, and if approved, Kyzatrex has the potential to become the standard of care for the treatment of primary and secondary hypogonadism globally.”

“An oral [testosterone] preparation with steady state physiologic levels would be a welcome addition to our choices for therapy assuming, of course, the absence of adverse effects,” explained Dr. Jellinger. “However, the greater challenge of testosterone therapy is the appropriate selection of those suited for testosterone replacement therapy.”

The company also plans to submit a marketing authorization application with the European Medicines Agency in the first half of 2022.

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