FDA Grants Orphan Drug Designation to Potential Acute Graft-Versus-Host Disease Therapy


There are currently no FDA-approved therapies for the prevention or treatment of acute graft-versus-host disease.

Officials with the FDA have granted Orphan Drug designations for Equillium’s EQ001 for both the prevention and treatment of acute graft-versus-host disease (aGVHD), according to a press release.

GVHD, which can be acute or chronic, is a common complication of allogeneic hematopoietic stem cell transplants (HSCT). The disease is caused by the transplanted immune system recognizing and attacking the recipient’s body. Patients who develop GVHD typically experience rash, itching, skin discoloration, nausea, vomiting, diarrhea, jaundice, difficulty breathing, and eye dryness and irritation.

GVHD is the leading cause of non-relapse mortality among patients with cancer receiving allogeneic HSCT. There is a 53% 5-year survival rate for patients who respond to corticosteroid treatment and a mortality rate as high as 95% in those who do not respond to corticosteroids.

Equillium, the company developing EQ001, plans to initiate the phase 1b/2 EQUATE clinical trial in early 2019 to study EQ001 in combination with corticosteroids for the initial treatment of patients with aGVHD.

GVHD is predominantly driven by T effector cells (Teff). EQ001 (itolizumab), a first-in-class monoclonal antibody, selectively targets the novel immune checkpoint receptors CD6. CD6 plays a role in modulating the activation, proliferation, differentiation, and trafficking of Teff cells, according to Equillium.

“Acute GVHD is a life-threatening condition faced by up to 50% of those who have undergone a hematopoietic stem cell transplant, and there are currently no approved therapies for either the prevention or treatment of aGVHD,” Stephen Connelly, PhD, chief scientific officer of Equillium, said in a statement. “Receiving both FDA Orphan Drug designations for EQ001 underscores the grave need for new treatment options. We believe the unique mechanism action of EQ001 in modulating both the activity and trafficking of pathogenic T cells is ideally suited for both the prevention and treatment of aGVHD.”

The FDA’s Orphan Drug designation program provides incentives to companies developing therapeutics for the safe and effective treatment, diagnosis, or prevention of rare diseases and disorders.


Equillium Granted US FDA Orphan Drug Designations for EQ001 (Itolizumab) for Both the Prevention and Treatment of Acute Graft-Versus-Host Disease [news release]. Equillium. https://ir.equilliumbio.com/news-releases/news-release-details/equillium-granted-us-fda-orphan-drug-designations-eq001. Accessed February 7, 2018.

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