FDA grants RPDD to META's hyperoxaluria drug

The US Food and Drug Administration (FDA) has awarded rare pediatric disease designation (RPDD) to META Pharmaceuticals' investigational drug, META-001-PH, for the treatment of primary hyperoxaluria (PH). This condition is a rare genetic disorder that results in the formation of kidney stones and can lead to renal failure. In severe cases, it can be life-threatening.

The RPDD is a mechanism designed to speed up the development of treatments for serious and life-threatening rare pediatric diseases affecting fewer than 200,000 individuals in the US. This designation is particularly focused on diseases that primarily affect children under 18. The legal basis for the RPDD falls under section 529(a)(3) of the Federal Food, Drug, and Cosmetic Act.

META Pharmaceuticals developed META-001-PH in collaboration with XtalPi’s AI-driven drug discovery team. From initial scaffold screening to preclinical compound nomination, XtalPi's automated robotic chemical synthesis lab played a crucial role in the efficient synthesis of this molecule series. The preclinical experiments conducted on META-001-PH demonstrated that the drug could reduce urinary oxalate excretion by up to 80%. Current treatments for PH are limited, often requiring invasive procedures or lifelong medication. However, META-001-PH, when taken orally daily, shows promising potential for providing better long-term control of kidney stone formation in PH patients. The preclinical animal models also indicated good tolerability and safety.

The drug is now undergoing investigational new drug (IND)-enabling toxicology studies. The next step in its development will be a Phase I safety assessment in healthy subjects, which is scheduled for the first half of 2025 in Australia.

The RPDD forms part of the FDA’s broader strategy to address the urgent need for therapies targeting rare pediatric diseases. It is also linked to the priority review voucher (PRV) program. The PRV program aims to incentivize the development of treatments for serious or life-threatening conditions by offering a voucher for expedited review of a future application. Upon approval of a new drug application (NDA) or biologics license application (BLA), sponsors can receive a PRV, which can be either used to accelerate the review process for future drugs or sold. Recent transactions for these vouchers have seen sale prices exceeding $100 million.

To summarize, META Pharmaceuticals' innovative effort in developing META-001-PH, a promising treatment for primary hyperoxaluria, has earned the FDA’s rare pediatric disease designation. This crucial designation not only highlights the potential breakthrough of the drug but also aligns with the FDA’s mission to expedite treatments for rare and critical pediatric conditions. With ongoing studies and planned clinical trials, META-001-PH represents a significant advancement in the long-term management of this devastating condition.