Study confirms metyrapone is effective, safe for treating endogenous Cushing’s Syndrome

HRA Pharma Rare Diseases has presented data from PROMPT, the first ever prospective study designed to confirm metyrapone efficacy and good tolerance in patients with endogenous Cushing’s Syndrome.

The first results confirm that metyrapone controlled 80% of the patients at week 12 with either normalisation or at least 50% decrease of urinary free cortisol.

Cushing’s Syndrome is a rare condition where patients have too much cortisol in their blood. Endogenous Cushing’s Syndrome is most often caused by hormone releasing tumours of the adrenal or the pituitary glands. To manage this condition, controlling high cortisol levels in patients is important.

Metyrapone is an inhibitor of the 11-beta-hydroxylase enzyme, which majorly contributes to cortisol synthesis and is approved in Europe for the treatment of endogenous Cushing’s syndrome based on observational retrospective studies published over more than 50 years.

As this prospective study took place over five years from April 2015 to April 2020, the longitudinal format reduced potential sources of bias and helped determine the risk factors of metyrapone when compared to the previous retrospective studies.

The first results of this study showed that at the end of the 12 weeks, metyrapone therapy is a rapid-onset, effective and safe medical treatment in Cushing’s Syndrome patients. mUFC control was achieved in 80% of patients with either normalisation or at least 50% decrease at week 12.

The next set of data on the 6 month optional extension is awaiting confirmation and the full study with the final results will be published next year.

“Cushing’s Syndrome is a chronic disease that can lead to deterioration in patients’ condition if not treated appropriately. We are thrilled to announce that this first prospective study verifies that metyrapone is both an effective and safe way to treat endogenous Cushing’s Syndrome,” said Frederique Welgryn, Managing Director of HRA Pharma Rare Diseases.

“This is a big step given the high unmet medical need for patients with endogenous Cushing’s Syndrome.”

David Wright, CEO of HRA Pharma, added: “PROMPT study is another piece of evidence demonstrating the commitment of HRA Pharma in making effective, safe and affordable treatments for patients with rare diseases.”

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