Accept Cookies?
Provided by OpenGlobal E-commerce

Please wait while your page loads ...

Retrotope Granted Rare Pediatric Disease Designation from FDA

Retrotope Granted Rare Pediatric Disease Designation from FDA for Lead Development Candidate, RT001, in Two Life-Threatening Neurodegenerative Indications

Company Currently Conducting Late-Stage Clinical Trials in Both Infantile Neuroaxonal Dystrophy (INAD) and Friedreich’s Ataxia (FA)

RT001 also Granted Fast Track Designation by FDA in FA; Orphan Drug Designation by European Medicines Agency in INAD

LOS ALTOS, CA – February 25, 2021 – Retrotope, a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class therapies for degenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted two rare pediatric disease designations to RT001, the company’s lead development candidate. The first rare pediatric disease designation is for the treatment of infantile neuroaxonal dystrophy (INAD), with the second covering the treatment of Friedreich’s ataxia (FA). In addition, RT001 has been granted Fast Track designation by the FDA for the treatment of FA and orphan drug designation by the European Medicines Agency (EMA) for the treatment of INAD. RT001 has previously been granted orphan drug designation in the U.S. for the treatment of multiple diseases, including FA, progressive supranuclear palsy (PSP) and PLA2G6-associated neurodegeneration, which includes INAD.   more...

Read the Full article here

SHARE

FacebookTwitterLinkedInYoutube
ra-philly-18.jpg

 

Archived in
  Scientific News


 

 

Tagged in
FARA Scientific News


Site Map     Privacy Policy      Service Terms      Contact      Charity Navigator