Equillium Granted U.S. FDA Orphan Drug Designations for EQ001

Equillium (NASDAQ:EQ) has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designations for EQ001 for the prevention and treatment of acute graft-versus-host disease (aGVHD).

As quoted in the press release:

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 that affect fewer than 200,000 people in the United States. As a result of EQ001’s Orphan Drug designations, Equillium is eligible for an additional seven years of market exclusivity if the drug is approved, tax credits for qualified clinical research costs and a waiver of the Prescription Drug User Fee paid at the time of submission of a marketing application to FDA.

“Acute GVHD is a life-threatening condition faced by up to 50 percent 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,” said Stephen Connelly, Ph.D., chief scientific officer of Equillium. “Receiving both FDA Orphan Drug designations for EQ001 underscores the grave need for new treatment options. We believe the unique mechanism of 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.”

Equillium plans to initiate the Phase 1b/2 EQUATE clinical trial (NCT 03763318) in early 2019, which will evaluate EQ001 in combination with corticosteroids for the initial treatment of patients presenting with aGVHD.

About Graft-Versus-Host Disease
Both aGVHD and chronic graft-versus-host disease (cGVHD) are multisystem disorders that are common complications of allogeneic hematopoietic stem cell transplants (HSCT). Graft-versus-host disease (GVHD) is caused by the transplanted immune system recognizing and attacking the recipient’s body. Symptoms of GVHD include rash, itching, skin discoloration, nausea, vomiting, diarrhea, jaundice, difficulty breathing, as well as eye dryness and irritation.

Click here to read the full press release.