28 June 2019 - - The Food and Drug Administration granted orphan drug designation to LM-030, an investigational therapy licensed from Swiss drugmaker Novartis (NYSE: NVS) and ready to enter into pivotal clinical trials for the treatment of Netherton Syndrome, US-based rare disease specialist LifeMax Laboratories, Inc. said.
Orphan drug designation is granted by the FDA Office of Orphan Products Development to promote the development of investigational therapies for diseases affecting less than 200,000 people in the US.
Benefits that come with an orphan drug designation include, but not limited to, tax credits for clinical development cost and seven-year market exclusivity.
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.
Though the exact prevalence of Netherton Syndrome is not well documented, it has a reported prevalence of 1-9 per 1,000,000.
LifeMax Laboratories, Inc., a wholly-owned subsidiary of LifeMax Healthcare International Corp., is a clinical-stage biotech company focused on treating rare diseases with few or no therapeutic options.
LifeMax is applying its expertise and in-depth experience with drug development and commercialization to advance its well-balanced portfolio of products addressing critical unmet medical needs.