A drug named DMX-200 has shown promising results in treating focal segmental glomerulosclerosis (FSGS), a rare kidney disease for which there are currently no FDA-approved treatments. Learn more about DMX-200 and what it could mean for people living with FSGS.*

What is FSGS?

FSGS is a kidney disease that affects about seven in a million people, but is responsible for approximately 20% of all kidney failure experiences in the U.S. FSGS permanently scars, and hardens, the tiny filtering units called glomeruli in your kidneys that clean your blood of wastes. 

This tissue scarring is called sclerosis, and in FSGS it only affects selected glomeruli, leaving others healthy. The disease can have a variety of causes, however, and many factors will determine how quickly FSGS progresses to kidney failure.

About DMX-200 research

DMX-200 was developed by biopharmaceutical company Dimerix, utilizing technology that it co-invented with researchers at The University of Western Australia. It’s an oral drug given to people with FSGS who are also taking angiotensin II receptor blockers (ARBs), medications used to treat high blood pressure and even heart failure. 

An early analysis of the first 72 randomized participants with FSGS in the drug’s Phase 3 clinical trial indicated that DMX-200 outperformed the placebo in reducing proteinuria (elevated protein in urine), a common marker of kidney disease progression. 

The Phase 3 trial, which includes both adults and children, is ongoing, with several hundred patients across multiple sites in 11 countries. 

Orphan drug designation

DMX-200 has been granted orphan drug designation by both the U.S. Food and Drug Administration (FDA) and the European Medical Association (EMA). 

It’s a status awarded to certain drugs called orphan drugs that show promise in treating or preventing orphan diseases (conditions that affect fewer than 200,000 people, but which are often life-threatening or life-limiting). 

Due to the relatively small number of people with a given orphan disease, producing treatments would not be profitable without assistance from the government.

According to the FDA, orphan drug designation renders sponsors eligible for incentives such as: 

• Tax credits for qualified clinical trials, and
• Exemption from certain fees.

It also qualifies them for up to seven years of market exclusivity following official approval of the new drug or therapy.

*The University of Western Australia. (2024, March 12). New drug shows promise in treating chronic kidney disease. https://www.uwa.edu.au/news/article/2024/march/new-drug-shows-promise-in-treating-chronic-kidney-disease 

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