The US Food and Drug Administration (FDA) has granted rare paediatric disease designation (RPDD) for META Pharmaceuticals’ investigational new drug, META-001-PH, to treat primary hyperoxaluria (PH).

This rare genetic disorder leads to kidney stone formation and renal failure, with severe cases being life-threatening.

The RPDD aims to expedite the development of treatments for serious and life-threatening rare paediatric diseases affecting fewer than 200,000 people in the US, primarily those under 18 years of age.

It falls under section 529(a)(3) of the Federal Food, Drug and Cosmetic Act.

Developed by META, META-001-PH is a small molecule drug created in collaboration with XtalPi's AI drug discovery team, from initial scaffold screening to preclinical compound nomination.

XtalPi's automated robotic chemical synthesis lab was instrumental in synthesising the molecule series efficiently.

Preclinical experiments have shown that META-001-PH can reduce urinary oxalate excretion by up to 80%.

Unlike existing treatments, when taken orally daily, it has the potential for better long-term control of kidney stone formation in PH patients and also showed good tolerability and safety in preclinical animal models.

META-001-PH is currently undergoing IND [investigational new drug]-enabling toxicology studies, with a Phase I safety assessment in healthy subjects planned for the first half of 2025 in Australia.

The RPDD is part of the FDA's efforts to address the urgent need for therapies targeting rare paediatric diseases.

It also ties into the priority review voucher (PRV) programme, which incentivises the development of new treatments for serious or life-threatening conditions.

Upon approval of an NDA or biologics licence application (BLA), sponsors can receive a PRV, which can expedite the review process for future applications or be sold, with recent transfer prices exceeding $100m.