The US Food and Drug Administration (FDA) has granted Orphan Drug Designation for Araim Pharmaceuticals' lead product candidate, Innate Repair Receptor activator ARA 290, for the treatment of sarcoidosis.

ARA 290 has previously been granted EU Orphan Drug Designation for the treatment of sarcoidosis, and has received US Orphan Drug and Fast Track designations for the treatment of neuropathic pain 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", commented Dr Daniel Culver, Respiratory Institute, Cleveland Clinic. "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. Sarcoidosis has a substantial impact on people in the prime of their life because of the disease, and affects their quality of life and productivity over many decades. 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 an uncommon inflammatory disorder that affects multiple organ systems and results in varied clinical signs and symptoms, often severe and disabling. Small fiber neuropathy (SFN) is a serious, potentially devastating complication of sarcoidosis that is being recognized with increasing frequency, with debilitating pain, abnormal sensory function, and severe dysautonomia as prominent manifestations. The precise pathophysiology of sarcoidosis SFN is unknown. No approved therapies are available, and off-label treatments remain symptomatic, inconsistent and limited.

Extensive preclinical data suggest a potential benefit of ARA 290 in treatment of SFN, as ARA 290 has demonstrated efficacy in a number of animal models of neuropathy, neuroprotection, and neuro-inflammation. Because of the broad range of efficacy noted with ARA 290 in preclinical models of neuropathy, ARA 290 is currently being evaluated in the clinical developmental programme not only for its ability to relieve symptoms associated with sarcoidosis SFN, but also for its potential for disease modification and long-term functional improvement in this condition.

Sarcoidosis is a chronic systemic granulomatous disease of unknown etiology, most commonly affecting young adults. The disease is chronic and progressive in more than a third of cases, leading to clinically significant organ impairment. While variable for each patient, the symptoms can cause a major loss in quality of life and inability to participate in the economic work force.  Morbidity in sarcoidosis is significant and multifactorial. Mortality is infrequent, but may be increasing over the years.

Araim Pharmaceuticals, Inc. is a clinical stage drug development company with a novel platform technology designed to address devastating injuries and chronic diseases underserved by current therapies.