The Food and Drug Administration (FDA) has granted Orphan Drug designation to LM-030 (LifeMax), an investigational treatment for Netherton syndrome (NS), an autosomal recessive monogenic disease caused by mutations in the SPINK5 gene that encodes a serine peptide inhibitor. The disease is characterized by congenital erythroderma, “bamboo hair” and immune system abnormalities.

LM-030 is a topical treatment that LifeMax licensed from Novartis in 2018. A phase 1 trial assessing the safety of the ointment was previously conducted by Novartis in 12 patients with either NS or atopic dermatitis.

“Netherton syndrome is a severe genetic disorder with neonate onset that can be life-threatening,” said Larry Hsu, LifeMax’s Co-founder and CEO. “We are very pleased to have received the Orphan Drug designation for LM-030 which has demonstrated safety and clinical efficacy in a phase 1/2 study.”

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There are currently no approved treatments for NS. If approved, Orphan Drug designation guarantees the Company 7 years of market exclusivity. 

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