FDA Favors Clinical Trial of Potential Juvenile Batten Treatment, PLX-200

Polaryx Therapeutics has received approval from the U.S. Food and Drug Administration (FDA) to open a clinical trial into PLX-200 as a potential treatment for juvenile Batten disease. Juvenile Batten, also known as Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) or CLN3 disease, is caused by mutations in…

FDA Grants PLX-100 Orphan Drug Designation to Treat Batten Disease

Polaryx Therapeutics’ investigative drug PLX-100 has been granted orphan drug designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of neuronal ceroid lipofuscinosis (NCL), also known as Batten disease. This follows the previous announcement of ODD status granted to PLX-200, another drug being tested…

Polaryx’s Treatment Candidate for LINCL Granted FDA Orphan Drug Status

The U.S. Food and Drug Administration (FDA) granted orphan drug status to Polaryx Therapeutics’ PLX-200 in response to a lack of treatments available for late infantile neuronal ceroid lipofuscinosis (LINCL), a type of Batten disease. PLX-200 is a repurposed drug (one that was developed for one indication…