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 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 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.
Though the exact prevalence of Netherton Syndrome is not well documented, it has a reported prevalence of 1-9 per 1m.
LifeMax Laboratories, Inc. a wholly-owned subsidiary of LifeMax Healthcare International Corp. is a clinical-stage biotechnology company focused on treating rare diseases with few or no therapeutic options.
UroGen's UGN-103 IND accepted by FDA for bladder cancer treatment
MaaT Pharma reveals positive 18-month data for MaaT013 in GI-aGvHD
Innate Pharma advances Sanofi-developed NK cell engager to Phase 2 for blood cancer patients
Soligenix receives orphan drug designation from FDA for active ingredient in SuVax
Candel Therapeutics granted FDA Orphan Drug Designation for CAN-2409 in pancreatic cancer treatment
Lipogems completes patient enrolment in ARISE I US FDA IDE study
Amylyx Pharmaceuticals' AMX0035 shows promising impact on Wolfram syndrome symptoms
Roche attains CE Mark for first companion diagnostic for HER2-low metastatic breast cancer
Cadrenal Therapeutics' tecarfarin receives US FDA Orphan Drug Designation
BioCity Biopharma's BC2027 Phase one study IND application receives US FDA approval
Seres Therapeutics completes patient enrollment for SER-155 Phase 1B trial