Rare Disease Report

Batten Disease Treatment Gets Orphan Drug Designation

AUGUST 06, 2017
Mathew Shanley
Early this morning, Polaryx Therapeutics announced that its drug PLX-200 as a treatment for late infantile neuronal ceroid lipofuscinosis (LINCL) was granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA). Forms of NCL are commonly known as Batten disease.
In LINCL, or Jansky-Bielschowsky disease, a mutation in the TPP1 gene causes a deficiency in tripeptidyl peptidase I, resulting in the accumulation of lipopigments in the body.
The condition also qualifies as a rare pediatric disease under Section 529 of the Food, Drug, and Cosmetic Act, and is an autosomal recessive neurodegenerative disorder.
"Granting PLX-200 the Orphan Drug Designation is one of the company's significant development milestones," says Dr. Hahn-Jun Lee, M.Sc., Ph.D., president and CEO of Polaryx Therapeutics, Inc. in a press release. "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."
Typically, early signs of LINCL are loss of vision impairment, muscle coordination (ataxia) and seizures that are non-responsive to drugs. Life expectancy in patients with this condition ranges from 6 years to early teens.
PLX-200 is designed to enhance production of transcription factor EB (TFEB) in brain cells, and is capable of regulating lysosomes.
"The FDA's decision 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 aspect,” Dr. Kalipada Pahan, Ph.D., a professor of neurological sciences, biochemistry, and pharmacology, and Floyd A. Davis, M.D., Endowed Chair in Neurology at the Rush University Medical Center in Chicago said in a joint statement.
In April, the FDA approved Brineura (cerliponase alfa); the first therapy for the late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) iteration of Batten disease. There aren’t currently any patient-friendly treatment options available.
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