TNF-alpha Inhibitor XTMAB-16 Granted FDA’s Orphan Drug Designation

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to XTMAB-16, Xentria’s investigational TNF-alpha inhibitor for the treatment of sarcoidosis.

The designation is given to candidate therapies with the potential to be safe and effective treatments for rare diseases, defined as those affecting fewer than 200,000 people in the U.S., with unmet medical needs.

It is meant to expedite XTMAB-16’s clinical development and review by providing regulatory support and financial benefits, as well as a seven-year marketing exclusivity period in the U.S. upon regulatory approval.

“We are pleased the FDA has granted Orphan Drug Designation for XTMAB-16 for the treatment of sarcoidosis, as it reaffirms the need for an effective therapy for patients with this potentially life-threatening inflammatory disease,” Thomas Shea, Xentria’s president, said in a press release.

“This is an important regulatory milestone for our company, and we look forward to advancing XTMAB-16 into the clinical phase,” Shea added.

According to the company’s website, a Phase 1 trial is expected to start enrollment in the first months of 2021.

Sarcoidosis is a highly variable inflammatory disease that can lead to the formation of small abnormal lumps or nodules — called granulomas — in virtually any organ in the body. Lungs are the most commonly affected, followed by lymph nodes (small immunological structures), skin, and eyes.

Many sarcoidosis patients are given off-label treatments, meaning that they are approved for conditions other than sarcoidosis. This underscores the unmet need for approved therapies for this rare inflammatory condition.

A previous study showed that about 10% of sarcoidosis patients in the U.S. are being treated with off-label TNF-alpha inhibitors. These work by suppressing the activity of TNF-alpha, a pro-inflammatory molecule that contributes to sarcoidosis’ chronic swelling and inflammation, and granuloma formation.

TNF-alpha inhibitors are a type of biologic therapy, also known as biologics, which are made either from living organisms or contain components of living organisms.

Given directly into the bloodstream, XTMAB-16 is a chimeric antibody designed to specifically block TNF-alpha. Chimeric antibodies are made up from different species, in XTMAB-16’s case it’s from human and mouse. The therapy is being developed as a potential treatment for people with sarcoidosis, whether in the lungs or other organs.

According to Xentria, extensive analyses have validated XTMAB-16 as a TNF-alpha inhibitor and preclinical studies are underway.