Polaryx Therapeutics has received Orphan Drug Designation From the European Medicines Agency (EMA) for PLX-200 for the treatment of Neuronal Ceroid Lipofuscinosis. 

Neuronal Ceroid Lipofuscinosis is a group of autosomal recessive neurodegenerative lysosomal storage disorders. This disorder is caused by cellular accumulation of abnormal auto-fluorescent lipoproteins, resulting in deterioration of neurons in both the brain and retina. Because many patients suffer from vision loss, severe seizures, and declining motor function, leading to premature death, treatment for these diseases is indeed an unmet medical need. 

“Granting PLX-200 the Orphan Drug Designation (ODD) for Neuronal Ceroid Lipofuscinosis from the European Medicines Agency indicates that the EMA highly regarded the significant value of the PLX-200 program, especially its safety and many potential benefits to patients. This EMA designation will clearly facilitate PLX-200 clinical development, which also was granted ODD for Neuronal Ceroid Lipofuscinoses from the FDA last year. Polaryx will file an IND for PLX-200 to the FDA this year, 2018, to assess its clinical efficacy and benefits to LINCL patients, first. We will initiate this clinical testing under the right settings and criteria as early as possible,” said Dr. Hahn-Jun Lee, M.Sc., Ph.D., president and chief executive officer of Polaryx.