In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat rare or chronic illnesses. For the purpose of this designation, “rare illnesses” are defined as those affecting under 200,000 individuals. Once a drug is granted this status, the drug developer is given incentives such as increased regulatory assistance, tax credits, fee waivers, and 7 years of market exclusivity upon approval. As a result, Orphan Drug status serves to improve drug development for those with rare conditions. According to a recent news release from biotechnology company Polaryx Therapeutics, Inc. (“Polaryx”), its therapeutic option PLX-200 received Orphan Drug designation for Krabbe disease.
So what exactly is PLX-200? According to Polaryx, PLX-200 is a repurposed:
PPARα agonist that boosts lysosome biogenesis via TFEB upregulation. It has therapeutic and/or prophylactic potential for Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL or CLN2) and for other NCLs, such as Juvenile Infantile Neuronal Ceroid Lipofuscinosis (JNCL or CLN3).
In the past, PLX-200 received Fast Track designation from the FDA, as well as Orphan Drug designation for all forms of neuronal ceroid lipofuscinosis. Thus, the drug’s Orphan Drug designation for Krabbe disease is its second. PLX-200, in trials, has been shown to be neuroprotective and anti-inflammatory.
Moving forward, Polaryx hopes to hold clinical trials to fill unmet treatment needs for patients with Krabbe disease and other lysosomal storage disorders.
Also known as globoid cell leukodystrophy, Krabbe disease is a rare inherited disorder caused by gene mutations on chromosome 14. Due to these mutations, patients with Krabbe Disease do not produce enough galactocerebrosidase (GALC), an enzyme which helps break down lysosomes. Because of this, galactolipids, a type of fat, accumulates in the body. This toxicity eventually causes the death of myelin, or the protective nerve cell coating. For children with the infantile form of Krabbe disease, most symptoms appear before the child is 1 year old; in many cases, this disorder is also fatal before age 2. However, in rarer cases, patients have late-onset Krabbe disease, at which point survival rates vary. Symptoms of infantile Krabbe Disease include:
- Difficulty feeding or swallowing
- Excessive irritability and/or unexplained crying
- Developmental delays
- Decline in alertness
- Muscle spasms or rigidity
- Loss of head control
- Easy startle response
In late-onset Krabbe disease, symptoms include:
- Progressive vision loss
- Muscle weakness
- Difficulty walking
- Loss of manual dexterity
Learn more about Krabbe disease.