Experimental Netherton Syndrome Treatment Receives Rare Pediatric Disease Designation from FDA

According to a press release from LifeMax Laboratories, Inc., the Food and Drug Administration (FDA) has granted the Company’s experimental Netherton syndrome drug LM-030 (licensed from Novartis) Rare Pediatric Disease designation. The designation acts as a kind of redeemable voucher for the priority review of a different product.

About Netherton Syndrome

Netherton syndrome is a genetically inherited condition characterized by physiological abnormalities of the skin and hair. Affected individuals frequently have problems with their immune systems as well.

Newborns with Netherton syndrome may have red and scaly skin that can, in some cases, leak fluid. Others may be born with a plastic wrap-like membrane around them called a collodion membrane, which usually sheds completely within a few weeks. Generally, the health of affected individuals improves with age. Many children with Netherton syndrome fail to grow at projected rates (failure to thrive), and even fully grown may remain underweight and shorter than their peers.

In later childhood and adulthood, skin irritation may fluctuate in severity over time. Parts of the skin may remain red and scaly (especially in childhood). Bouts of redness and ichthyosis linearis circumflexa (a skin condition characterized by the outbreak of patches of circular lesions around the body) may continue to affect certain patients into adulthood. Other skin problems, such as chronic itchiness (which may lead to infection), eczema, poor thermal regulation, and excessive shedding are also common.

Mild immune system dysfunction, such as hay fever, food allergy, or athsma is also frequently reported in Netherton syndrome patients. Patients with Netherton syndrome sometimes have unusually fragile hairs that vary in non-uniform thickness. Trichorrhexis nodosa, or “bamboo hair,” may also affect the eyelashes or eyebrows.

About LM-030 and Rare Pediatric Disease Designation

Rare Pediatric Disease” designation is given to drugs that aim to treat rare “serious or life-threatening” diseases in people younger than 18. Diseases are classified as rare if they affect fewer than 200,000 people in the United States.

Although Fast Track, Breakthrough, and Priority Review programs exist to accelerate the research and development of the recipient drug, rare pediatric disease designation acts as a voucher that the receiving company can redeem in the future for the priority review of another product. In other words, it’s kind of a one-use priority review credit that the FDA has to honor.

A phase 1/2 clinical study of LM-030 found the drug to be tolerable in humans, while providing benefits that were unspecified in the press release. Future studies are likely to build on these findings as LM-030 works its way towards a new drug application (NDA).

Additionally, LM-030 received Orphan Drug Designation towards the end of June. The designation offers governmental R&D support, as well as marketing perks that kick into effect if and when LM-030 receives its final marketing approval from the FDA.

Do you or does anyone you know live with Netherton syndrome? What do you think of this exciting news? Share your story with Patient Worthy!

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