LG Chem said Thursday that the U.S. Food and Drug Administration has granted an additional Orphan Drug Designation (ODD) status to its LB54640, which treats genetic obesity due to proopiomelanocortin (POMC) deficiency.

In September 2020, the U.S. regulator also designated LB54640 as a rare drug to treat leptin receptor (LEPR) deficiency.

The FDA operates the ODD system to encourage and support the development of treatments for rare and intractable diseases with a prevalence of fewer than 200,000 people. For example, there are about 120,000 rare cases of hereditary obesity in the U.S.

With the recent designation, LG Chem will receive benefits, such as exclusive rights to sell in the U.S. market for seven years, support for clinical trial costs and tax cuts in the U.S., and pre-consultation support for the drugs’ development.

LB54640 is a treatment targeting the action route of the melanocortin-4 receptor (MC4R), a protein in satiety. Even if MC4R's upper path genes, such as LEPR and POMC, are defective, it eventually acts directly on the MC4R protein that transmits satiety signals to induce appetite suppression.

LG Chem plans to present differentiated treatment experiences with new oral drugs armed with improved convenience in the rare obesity drug market led by injection treatment.

LG Chem recently completed the first phase of clinical trials in the U.S. of LB54640 for healthy overweight adults without genetic defects and planned to announce specific clinical results within the year. Subsequently, the company plans to enter the global second and third phase for patients with hereditary obesity, including the LEPR or POMC genetic deficiencies starting next year.

The LB54640 drug shows great potential as global market research from Evaluate Pharma indicates that the U.S. market for genetic obesity treatments is expected to grow rapidly from 36 billion won ($28 million) in 2022 to 1 trillion won in 2028.