1st Potential Therapy for Sarcoidosis, ARA 290, Named Orphan Drug by FDA to Speed Its Testing

Daniela Semedo, PhD avatar

by Daniela Semedo, PhD |

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FDA orphan drug designation

Araim Pharmaceuticals recently announced that its lead candidate, Innate Repair Receptor (IRR) activator ARA 290, has been designed an orphan drug for the treatment of sarcoidosis by the U.S. Food and Drug Administration (FDA).

ARA 290, a synthetic 11-amino acid peptide IRR agonist, is currently in Phase 2 clinical trials as a potential treatment of patients with sarcoidosis-related small fiber neuropathy. ARA 290 has also been granted orphan drug status in the European Union, as well as Fast Track designation by the FDA for the treatment of painful small fiber neuropathy in patients with sarcoidosis.

“The granting of Orphan Drug Designation for the treatment of sarcoidosis in both the US and Europe highlights the significant need for a drug that could transform the treatment of sarcoidosis,” Dr. Daniel Culver, with the Respiratory Institute at the Cleveland Clinic, said in a recent press release. “It is important to drive therapeutic research forward in this area. Patients with sarcoidosis have many unmet needs, as the condition is underappreciated and undertreated. … ARA 290 has the potential to become an important disease modifying therapy for sarcoidosis patients as well as other diseases characterized by chronic inflammation and persistent tissue injury.”

Sarcoidosis is a multisystem inflammatory disease of unknown etiology that manifests as granulomas, predominantly in the lungs and intrathoracic lymph nodes. Small fiber neuropathy, a serious complication of sarcoidosis, is being recognized with increasing frequency, being linked to debilitating pain, abnormal sensory function, and severe dysautonomia (conditions characterized by malfunction of the autonomic nervous system).

No treatments are currently approved to treat sarcoidosis and, according to the release, off-label treatments are inconsistent and limited.

The age-adjusted incidence of sarcoidosis in the U.S. is 11 cases per 100,000 population in whites, and 34 cases per 100,000 population in African-Americans.