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news.LifeMax Laboratories, a private company focused on treating rare diseases with few or no therapeutic options, announced that the Food & Drug Administration (FDA) granted rare pediatric disease designation to LM-030, an investigational therapy licensed from Novartis and ready to enter into pivotal clinical trials for the treatment of Netherton Syndrome.
Image: The US FDA’s Center for Drug Evaluation and Research. Photo: courtesy of The U.S. Food and Drug Administration.
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“We are very pleased to have received the rare pediatric disease designation for LM-030 which has demonstrated safety and clinical efficacy in a Phase I/II study. Netherton Syndrome is a severe genetic disorder with neonate onset that can be life-threatening. 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, an industry veteran who previously founded and built Impax Laboratories into a publicly traded multi-billion dollar company.
Rare pediatric disease is defined by the FDA as a disease affecting less than 200,000 people in the US and the most severe and life-threatening manifestation of which is primarily in pediatric patients.
If a new drug application (NDA) for LM-030 is approved, LifeMax may be eligible to receive a rare pediatric disease priority review voucher, which can be redeemed to obtain priority review for any subsequent NDA or biological license application (BLA) and can be sold or transferred.
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 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.
Source: Company Press Release
