Polaryx Therapeutics Announces FDA Grants Orphan Drug Designation for PLX-200 in GM2 Gangliosidoses

https://www.prnewswire.com/news-releases/polaryx-therapeutics-announces-fda-grants-orphan-drug-designation-for-plx-200-in-gm2-gangliosidoses-301364861.html

Polaryx Therapeutics, Inc. (“Polaryx”), a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that the U.S. Food and Drug Administration (“FDA”) has granted Orphan Drug Designation for PLX-200 to treat GM2 gangliosidoses. This designation is one of the important development milestones for the company, because it allows an expanded use of PLX-200 to one of the key lysosomal storage disorders which have clear unmet medical needs. Furthermore, this designation validates the rationale for clinical use of PLX-200 in GM2 gangliosidoses patients.