META Pharmaceuticals Secures FDA Rare Paediatric Disease Designation for META-001-PH
Tuesday, August 06, 2024
META Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has awarded Rare Paediatric Disease Designation (RPDD) to its investigational drug META-001-PH, aimed at treating primary hyperoxaluria (PH).
META-001-PH is a novel small molecule drug developed by META Pharmaceuticals, in collaboration with XtalPi's AI drug discovery team. Preclinical studies in animal models have demonstrated that META-001-PH can reduce urinary oxalate excretion by up to 80%.
Unlike existing treatments, which struggle to control urinary oxalate long-term, META-001-PH can maintain normal oxalate levels with daily oral administration, offering potential for better long-term management of kidney stone formation in PH patients. It has shown good tolerability and safety in preclinical models, with a Phase I clinical safety assessment planned for 2025 in Australia.
PH is a rare genetic disorder that leads to kidney stone formation and potentially fatal renal failure. The RPDD aims to promote the development of treatments for severe and life-threatening rare paediatric diseases affecting fewer than 200,000 individuals in the U.S., primarily those aged 18 and younger. This designation is in accordance with section 529(a)(3) of the Federal Food, Drug, and Cosmetic Act.
PH is an autosomal recessive metabolic disorder that causes the overproduction and deposition of oxalate due to enzyme defects in the liver and other organs. Patients typically suffer from kidney stones, nephrocalcinosis, renal failure, and oxalate deposition in other organs. Severe cases can lead to end-stage renal disease (ESRD), requiring dialysis or transplantation.
Symptoms generally appear between ages 0 to 3, and most patients develop ESRD during adolescence without intervention. PH affects over 10,000 people in the U.S. and EU, and more than 20,000 in China. Current treatments focus on supportive care, such as increased fluid intake and medications like pyridoxine (vitamin B6), but these do not effectively control disease progression.
The Rare Paediatric Disease Designation encourages the development of treatments for serious rare paediatric diseases. Sponsors of approved applications may receive a Priority Review Voucher, expediting the review process for subsequent applications or providing significant trade value.
Source: prnewswire.com
