FDA Grants LifeMax Rare Pediatric Disease Designation for Netherton Syndrome Therapy

The U.S. Food and Drug Administration granted LifeMax Laboratories Rare Pediatric Disease designation to LM-030, an experimental therapy in development to treat Netherton syndrome, a rare and potentially life-threatening condition.

Photo: Larry Hsu, CEO of LifeMax

Netherton Syndrome is a severe autosomal recessive disease characterized by congenital erythroderma, “bamboo hair,” and abnormality in the immune system. It can be life-threatening in pediatrics due to an impaired skin barrier that leads to severe dehydration, hypernatremia, hypothermia, gross weight loss, and sepsis. Failure to thrive is common in childhood as a result of chronic erythroderma, persistent cutaneous infection, malnutrition, and metabolic disorders. The severity of the skin abnormality in older patients can fluctuate over time. Most Netherton syndrome patients are also inflicted with immune system-related disorders such as food allergies and asthma.

LifeMax licensed LM-030 from Novartis and said it is has demonstrated safety and clinical efficacy in a phase 1/2 study and is ready to enter into pivotal clinical trials.  

“Receiving the rare pediatric disease designation on the heel of receiving the orphan drug designation represents another significant step in the development of LM-030 for this devastating disease,” said Larry Hsu, LifeMax’s co-founder and CEO.

Rare pediatric disease is defined by the FDA as a disease affecting less than 200,000 people in the United States. If a new drug application for LM-030 is approved, LifeMax may be eligible to receive a rare pediatric disease priority review voucher.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, GW Pharma sold its priority review voucher for $80.6 million.

Author: Rare Daily Staff