Rare Disease Report

FDA Grants Rare Pediatric Disease Designation to TXA127

JANUARY 18, 2017
Andrew Black
Tarix Orphan’s orphan drug TXA127 received a Rare Pediatric Disease Designation from the FDA for the treatment of recessive dystrophic epidermolysis bullosa.
 
TXA127, is a formulation of the natural Angiotensin (1-7) peptide which is designed to interfere with the TGF beta pathway. 
 
It is also being tested as a treatment for Duchenne muscular dystrophy (DMD). A Phase 2 study in DMD patients is expected to begin in early 2017. 
 
At this time, it is not known when a clinical trial for epidermolysis bullosa will begin. 

Pediatric Designation Could Bring $$$

The rare pediatric disease designation means the FDA will give Tarix a pediatric priority review voucher if the drug is approved for the pediatric indication. That voucher can then be used by the company for another drug—any drug—to be given a priority review. A priority review means the FDA will review the drug within 6 months instead of the standard 10 months.
 
The priority review voucher is also transferable to other companies—the current rate a priority review voucher goes for is up to $400 million.

About Epidermolysis Bullosa

Epidermolysis bullosa is a group of genetic conditions that cause the skin to be very fragile and to blister easily. Blisters and skin erosions form in response to minor injury or friction, such as rubbing or scratching.
 
Dystrophic epidermolysis bullosa symptoms of vary widely among affected individuals. In mild cases, blistering may primarily affect the hands, feet, knees, and elbows. Severe cases of this condition involve widespread blistering that can lead to vision loss, disfigurement, and other serious medical problems.
 


Stay informed on the latest rare disease news and developments by signing up for our newsletter.
Copyright © RareDR 2013-2018 Rare Disease Communications. All Rights Reserved.