FDA Approves Sanofi’s All-Oral Treatment for Sleeping Sickness

July 22, 2021 - FDA recently approved Sanofi’s Fexinidazole, the first all-oral treatment for both stages of the Trypanosoma brucei gambiense form of sleeping sickness.

The agency approved Fexinidazole for patients six years of age and older, weighing at least 44 pounds. Sanofi developed the medication as part of a partnership between the non-profit research and development organization Drugs for Neglected Disease initiative (DNDi). 

The organization conducted clinical trials for the drug in partnership with Sanofi, the National Sleeping Sickness Programs of the Democratic Republic of Congo (DRC), and the Central African Republic (CAR).

Trypanosoma brucei gambiense affects populations living in remote rural areas of sub-Saharan Africa. If left untreated, the disease is almost always fatal.

According to the World Health Organization (WHO), Sanofi’s collaboration with the organizations decreased Trypanosoma brucei gambiense cases by 97 percent between 2001 and 2020.

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“This FDA approval is a key milestone in Sanofi’s long-term commitment to fight sleeping sickness, started 20 years ago alongside the WHO through an ambitious partnership to combat Neglected Tropical Diseases” Luc Kuykens, senior vice president of Sanofi Global Health unit, said in the announcement. 

“Following the positive scientific opinion granted by the European Medicines Agency end 2018, the FDA approval is an important step to revitalize efforts to support the sustainable elimination of the disease,” Kuykens continued. 

A Tropical Disease Priority Review Voucher (PRV) has been awarded to DNDi as a result of the FDA approval.

The FDA Tropical Disease PRV Program was established in 2007 to support the development of new treatments for neglected tropical diseases. Sanofi and DNDi will share benefits from the PRV. 

FDA Approves Rezurock for Chronic Graft-Versus-Host Disease

FDA recently approved Rezurock to treat adult and pediatric patients 12 years of age and older with chronic graft-versus-host disease (cGVHD) after the failure of at least two prior lines of systemic therapy.

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This treatment is the first and only approved small molecule inhibitor of ROCK2, a signaling pathway that modulates inflammatory responses and fibrotic processes.

“REZUROCK represents a new treatment paradigm for thousands of cGVHD patients, including those with difficult-to-treat manifestations like fibrosis,” Corey Cutler, MD, MPH, FRCPC, associate professor of medicine at Harvard Medical School, said in the announcement. 

“REZUROCK has shown robust and durable responses across the spectrum of cGVHD and is safe and well tolerated, allowing patients to stay on therapy and achieve meaningful benefit from treatment,” continued Cutler, who is also the medical director of the adult stem cell transplantation program at the Dana-Farber Cancer Institute. 

FDA approved the Rezurock 200 milligrams once daily based on the safety and efficacy results from ROCKstar, a randomized, open-label multicenter pivotal trial of Rezurock in patients with cGVHD. 

In 65 patients treated with Rezurock, the median time from cGVHD diagnosis was 25.3 months, and 48 percent of patients had four or more organs involved. Patients had cycled through a median of three prior lines of systemic therapy, and 78 percent were refractory to their last therapy. 

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Rezurock 200 milligrams achieved an overall response rate of 75 percent through Cycle 7 Day 1 of treatment, with 6 percent achieving complete response and 69 percent achieving a partial response. 

About 62 percent of responders did not require new systemic therapy for at least 12 months following response. 

“Patients receiving REZUROCK reported significant improvements in cGVHD symptoms, showing that not only did treatment result in organ responses, but it also made people feel better. This is so important for a chronic disease with a high symptom burden,” said Stephanie Lee, MD, MPH, professor at the Fred Hutchinson Cancer Research Center and the University of Washington School of Medicine. 

Rezurock is expected to be available in the US by late August 2021. 

FDA Approves Kerendia Tablets for Complications of Chronic Kidney 

FDA recently approved Bayer’s Kerendia to reduce complications of chronic kidney disease associated with type 2 diabetes in adult and pediatric patients. 

Specifically, the agency approved the tablets to reduce the risk of kidney function decline, kidney failure, cardiovascular death, non-fatal heart attacks, and hospitalization for heart failure.

FDA based its approval on a placebo-controlled study of 5,674 patients with chronic kidney disease associated with type 2 diabetes. The patients randomly received Kerendia (2,8333) or placebo (2,841). 

About 504 of the patients who received Kerendia had at least one of the events in the composite endpoint, compared to 600 of the patients who received placebo. And 367 of the patients who received Kerendia had at least one of the events in the composite endpoint, compared to 420 of the patients who received placebo. 

Overall, the treatment elicited a reduction in the risk of cardiovascular death, non-fatal heart attack, and hospitalization for heart failure.