Metopirone Available to Treat Endogenous Cushing’s in Colombia

HRA Pharma reports bringing first treatment to patients in that country

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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Adults and children with endogenous Cushing’s syndrome in Colombia will have access to Metopirone (metyrapone), the first Cushing’s treatment to be approved in the country, HRA Pharma Rare Diseases, which developed and markets the treatment, announced.

With this registration, Colombia joins several other countries, including in Europe, the U.K., Israel, Switzerland and New Zealand, where Metopirone is similarly approved for Cushing’s patients.

In the U.S., the medication is approved for diagnostic purposes, a component of laboratory tests for adrenal insufficiency — a condition in which the adrenal glands, which sit stop the kidneys, are unable to produce enough of certain hormones, including cortisol.

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“Cushing’s Syndrome is a chronic condition that can be fatal if left untreated,” Evelina Paberžė, chief operating officer of HRA Pharma Rare Diseases, said in a press release emailed to Cushing’s Disease News.

“As a company which is dedicated to improving the lives of those with rare diseases, we felt it was crucial to support the registration of a medicine that can help healthcare professionals in the treatment of Cushing’s Syndrome. We are confident that Metopirone will help improve the lives of many,” Paberžė said.

Cushing’s syndrome comprises a group of disorders characterized by excessive cortisol, a stress hormone that’s released from the adrenal glands. The disease is considered endogenous when a person’s body is the source of this overproduction.

Endogenous Cushing’s syndrome is commonly caused by tumors in the brain’s pituitary gland, which release adrenocorticotropic hormone (ACTH) that signals the adrenal glands to make more cortisol. This specific form is called Cushing’s disease.

Metopirone belongs to a class of medications called steroidogenesis inhibitors. It essentially works by suppressing the last step of cortisol production in the adrenal glands, lowering blood cortisol levels and easing Cushing’s symptoms.

Most evidence for Metopirone’s effectiveness in Cushing’s come from observational and retrospective studies.

According to HRA Pharma Rare Diseases, data show that treatment leads to improvements in clinical features of the disease, including high blood pressure, impaired glucose (blood sugar) metabolism, psychiatric disturbances, and muscle weakness.

The prospective Phase 3/4 PROMPT study (NCT02297945) and its extension phase, which finished last year, evaluated the effects of Metopirone in 50 adults with endogenous Cushing’s.

Data from the trial showed that treatment with Metopirone lowered cortisol levels over nine months, eased symptoms and improved patients’ quality of life.

Colombian registration, HRA Pharma stated, is part of its decision to expand its global reach, “in order to ensure that no patient is left behind.”

With its exclusive local partner, Valentech Pharma, the company has been working since 2019 to achieve registration. Efforts were delayed due to the COVID-19 pandemic.

“This registration was the result of various teams working together to bring this vital medicine to the people who need it,” said Gabriel Muñoz Di Rocco, CEO of Valentech.