* Potential fast-track status possible where clinical Phase 2 trials would not be required assuming PK Bridging Studies for GTX-104 and GTX-102 meet their endpoints
** Single Dose (SD) and Multiple Ascending Dose (MAD)
References:
- The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the treatment, prevention or diagnosis of a rare disease or condition, which is one that affects less than 200,000 persons in the United States or meets cost recovery provisions of the Orphan Drug Act. The status helps incentivize the development of therapies to treat unmet medical needs by providing a company with seven years of exclusivity rights once a drug reaches market.
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- National Organization for Rare Disorders, Ataxia-Telangiectasia (2015)
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- Grace GTX-101 Phase 1 Study Report