FDA grants rare pediatric disease designation to META-001-PH for primary hyperoxaluria

The FDA has granted a rare pediatric disease designation (RPDD) to the investigational small molecule drug META-001-PH for the potential treatment of patients with primary hyperoxaluria, META Pharmaceuticals, the developer of the drug, announced in a news release.¹

A phase 1 study of META-001-PH is planned for the first half of 2025.

An RPDD is granted to drugs and biologics that are intended to treat diseases that affect fewer than 200,000 patients and pose a serious life-threatening risk to children under the age of 18. Primary hyperoxaluria has an estimated incidence of 1 in 58,000, affecting more than 10,000 people in the United States and the European Union and more than 20,000 people in China, according to the news release.¹

Under the rare pediatric disease designation, META-001-PH is eligible for priority review following an FDA acceptance of a new drug application for the therapy. Priority review designations allow for a 4-to-6-month reduction in the review time for investigational therapies.

According to META, in preclinical studies, META-001-PH demonstrated the ability to reduce urinary oxalate excretion by up to 80%. Findings also showed that oral administration of the drug once daily led to long-term maintenance of normal oxalate levels, which the company says holds potential for better long-term control of kidney stone formation. The drug also showed a favorable tolerability and safety profile in preclinical animal models.

META-001-PH is currently undergoing investigational new drug (IND)-enabling toxicology studies. A phase 1 study of META-001-PH is planned for the first half of 2025. The phase 1 trial plans to assess the safety of the drug in healthy participants in Australia.

In addition to META-001-PH, META Pharmaceuticals also has 2 other investigational molecules currently in development, including META-1i-AID for patients with autoimmune diseases and META-2i, which is currently in IND-enabling studies for patients with metabolic diseases.²

References

1. META Pharmaceuticals announces FDA grants rare pediatric disease designation to META-001-PH for the treatment of primary hyperoxaluria. News release. Meta Pharmaceuticals Inc. Published online and accessed August 5, 2024. https://www.prnewswire.com/news-releases/meta-pharmaceuticals-announces-fda-grants-rare-pediatric-disease-designation-to-meta-001-ph-for-the-treatment-of-primary-hyperoxaluria-302214393.html

2. Pipeline Milestones. META Pharmaceuticals Inc. Accessed August 5, 2024. http://en.metabiopharma.com/index.php/milestone/