FDA Grants ODD to Investigational Drug for Epidermolysis Bullosa

americanpharmaceuticalreviewDecember 09, 2019

Tag: FDA , ODD , APR , APR-TD011 , EB

APR Applied Pharma Research (APR) has announced the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for its investigational drug code-named APR-TD011 for treatment of Epidermolysis Bullosa (EB).

APR-TD011 is a hypotonic acid-oxidizing solution containing hypochlorous acid in a sprayable form for topical application, specifically developed for EB. APR-TD011 could be able to prevent or reduce infections as well as inflammation through modulation of the wound microenvironment, thus accelerating the physiological wound healing.

"The granting of Orphan Drug Designation in the US highlights the significant need for a drug that could improve the treatment of EB, a debilitating disease that affects the life of half a million people in the US. We believe that APR-TD011 holds great promises for EB patients and their families and the Orphan status will help APR to find expedited pathways to fruition," said Paolo Galfetti, CEO of APR. "This designation is also an important milestone and step forward in APR's evolution as we advance our pipeline targeting rare diseases."

EB market is estimated to range between $1.0 - 1.3 billion worldwide. EB imposes a major burden for global health care: the cost of treatment of a severely affected patients in the United States can exceed $300,000 per year, whereas wound care supplies for those with Dystrophic EB could require more than $10,000 per month.

The Orphan Drug Designation program provides incentives and support for the development of drugs for patients with rare diseases, specifically to drugs that are intended for the treatment of diseases or conditions that affect fewer than 200,000 people in the United States. Orphan Drug Designation provides certain benefits, including seven years of market exclusivity upon regulatory approval for the designated indication, exemption from FDA application user fees, FDA assistance in clinical trial design as well as tax credits (up to 25%) for qualified clinical trial expenses.

APR-TD011 is a hypotonic acidic oxidizing solution, containing hypochlorous acid, obtained through the company patented nanotechnology platform, Tehclo™.

The solution is featured by an exclusive combination of three physico-chemical properties - highly pure HClO, hypotonic low pH and high ORP, which are believed to support a faster physiological healing of EB wounds by creating a favourable wound microenvironment. In particular, hypochlorous acid is well known as a broad-spectrum, fast acting antimicrobial agent, which reinforced by low pH and high ORP contributes to prevent and treat skin infections. APR-TD011 could also reduce inflammation by inhibiting the NF-kB pro-inflammatory pathway and by inactivating the Matrix metalloproteases, known to play a key role in the inflammatory process of wounds. APR-TD011 could reasonably ameliorate the quality of life of EB patients by supporting faster wound healing and by reducing itching and pain, linked to infections and inflammation. Available in an easy-to-use spray formulation, the solution allows wounds to be treated while avoiding skin contact and cross-contamination risk.

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