Drug nets orphan designation for beta-thalassemia



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The US Food and Drug Administration (FDA) has granted orphan drug designation to PTG-300, a subcutaneous injectable hepcidin mimetic, for the treatment of beta-thalassemia.

Protagonist Therapeutics, the company developing PTG-300, recently completed a phase 1 trial of the drug.

In this study, healthy volunteers treated with PTG-300 achieved dose-related and sustained reductions in serum iron levels.

In addition, PTG-300 was considered well tolerated, producing no serious adverse events or dose-limiting toxicities.

Protagonist Therapeutics intends to initiate a global clinical trial of PTG-300 in patients with beta-thalassemia following upcoming meetings with the FDA and European Medicines Agency.

“Beta-thalassemia is a rare genetic blood disorder that is characterized by impaired red blood cell production that can result in life-threatening chronic anemia, usually requiring regular and life-long blood transfusions for survival,” said David Y. Liu, PhD, chief scientific officer and head of research and development at Protagonist Therapeutics.

“Over time, these transfusions can lead to excessive iron levels in the body, which can be toxic and consequently lead to end-stage damage to vital organs such as the liver and the heart. As a hepcidin mimetic, PTG-300 is designed to help reduce these excessive iron levels, and, thereby, it may lead to improvements in anemia and decreased need for blood transfusions and chelation therapy.”

About orphan designation

The FDA grants orphan designation to products intended to treat, diagnose, or prevent diseases/disorders that affect fewer than 200,000 people in the US.

The designation provides incentives for sponsors to develop products for rare diseases. This may include tax credits toward the cost of clinical trials, prescription drug user fee waivers, and 7 years of market exclusivity if the product is approved.

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