The drug is the first patient-friendly LINCL treatment of its kind and helps regulate Tripeptidyl Peptidase 1.
The US Food and Drug Administration granted an Orphan Drug Designation to PLX-200, a treatment for Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL) and for some forms of Batten disease (NCL).
LINCL, which qualifies as a rare pediatric disease under the parameters set by the Food, Drug and Cosmetic Act, usually allows a life expectancy of 6 to the early teens. There are no patient-friendly treatments available currently, making Polaryx Therapeutics' PLX-200 a unique treatment option.
"Granting PLX-200 the Orphan Drug Designation is one of the company's significant development milestones," Hahn-Jun Lee, MD, M.Sc., Ph.D., president and CEO of Polaryx Therapeutics, Inc., said in a statement. "We will accelerate this program into clinics as early as possible to help LINCL patients. We will confirm the efficacy of PLX-200 in humans and further confirm that it can be applied to other types of NCL disorders."
Caused by mutation in the Cln2 gene, LINCL leads to the deficiency and/or function loss of Tripeptidyl Peptidase 1 (TPP1), which in turn causes a clustering of autofluorescent storage materials in neurons and other cells, and eventually leads to seizures, visual impairment, and other symptoms.
PLX-200 is designed to bind to the retinoid X receptor-α (RXRα), which in turn binds to PPARα, and regulates TPP1 expression in brain cells. Kalipada Pahan, MD, PhD, professor of neurological sciences, biochemistry, and pharmacology, and the Floyd A. Davis, MD, Endowed Chair in Neurology at Rush University Medical Center, added in a statement that the FDA’s designation “recognizes the significant value of our technology that led to the extension of survival of an LINCL animal disease model and delayed the decline in motor function.”
“As this is a patient-friendly new treatment option that is a safely-used oral small molecule, it will help a lot to patients in many aspects," Pahan said.
The Orphan Drug Designation will allow incentives for sponsors to aid in the development in PLX-200 and will give 7 years of market exclusivity if PLX-200 is granted FDA approval.
Earlier this year, the FDA granted approval for the first treatment for CLN2, given to enzyme replacement therapy treatment Brineura (BioMarin Pharmaceutical).