META Pharmaceuticals announces FDA Grants Rare Pediatric Disease Designation to META-001-PH for the Treatment of Primary Hyperoxaluria

HONG KONG , Aug. 5, 2024 /PRNewswire/ -- META Pharmaceuticals Inc. announced that the U.

S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to its investigational new drug META-001-PH for the treatment of primary hyperoxaluria (PH). Primary hyperoxaluria is a rare genetic disorder that can lead to kidney stone formation, renal failure, and can be life-threatening in severe cases.

The RPDD is intended to facilitate the development of drugs and biologics for serious and life-threatening rare pediatric diseases that affect fewer than 200,000 people in the U.S. and predominantly occur in patients aged 18 years and younger.

This designation is pursuant to section 529(a)(3) of the Federal Food, Drug, and Cosmetic Act (FD&C Act) (21 U.S.C.

360ff(a)(3)). About Primary Hyperoxaluria (PH) Primary Hyperoxaluria (PH) is an autosomal recessive metabolic disorder in which oxalate is overproduced and deposited in the body due to defects in enzymes responsible for oxalate metabolism in the liver and other organs. Patients typically present with kidney stones, nephrocalcinosis, renal failure, and oxalate deposition in other organs.

Severe cases can lead to end-stage renal disease (ESRD) requiring dialysis, kidney transplantation, or combined liver-kidney transplantation. Symptoms of the disease usually appear at the age of 0 to 3. Without intervention, most patients will develop end-stage renal disease during adolescence, which severely threatens their .