Polaryx Therapeutics Receives FDA Orphan Drug Designation for PLX-200 to Treat Krabbe Disease

https://www.globenewswire.com/news-release/2021/09/02/2290516/0/en/Polaryx-Therapeutics-Receives-FDA-Orphan-Drug-Designation-for-PLX-200-to-Treat-Krabbe-Disease.html

Polaryx Therapeutics announced today that the U.S. Food and Drug Administration (“FDA”) has granted Orphan Drug Designation for PLX-200 to treat Krabbe disease. Granting by the FDA of Orphan Drug Designation for PLX-200 in Krabbe disease supports the use of PLX-200 to treat key lysosomal storage disorders with unmet medical needs. Because supportive care is the only available treatment option for most cases of Krabbe disease, this designation validates the scientific rationale and strongly motivates us to expedite the clinical development of PLX-200 in Krabbe disease. They are moving forward with the necessary development steps to move into the clinical study as soon as possible.