- In February 2025, YolTech Therapeutics announced promising results from an ongoing investigator-initiated trial of YOLT-203, an investigational in vivo gene-editing therapy for PH1. The data showed an almost 70% reduction of 24-hour urinary oxalate levels in patients who received the high dose, sustained through a 16-week observation period. The therapy was well-tolerated with no serious adverse events
- In February 2025, Alnylam Pharmaceuticals reported continued market momentum for Oxlumo® (lumasiran), the first FDA-approved RNAi therapy for PH1, with global net product revenues reaching approximately USD 167 million for the full year of 2024, reflecting a 29% increase compared to the previous year. This growth underscores the expanding global adoption of Oxlumo and reinforces Alnylam's leadership in RNAi-based treatments for hyperoxaluria
- In December 2024, The FDA accepted an Investigational New Drug (IND) application for ABO-101, a liver-targeting gene editing therapeutic developed by Arbor Biotechnologies, to initiate a Phase 1/2 trial (redePHine) in adult and pediatric patients with PH1. ABO-101 is designed as a one-time treatment to permanently deactivate the HAO1 gene, thereby reducing oxalate production
- In September 2023, Novo Nordisk received FDA approval for Rivfloza (nedosiran), a novel RNA interference (RNAi) therapy targeting lactate dehydrogenase A (LDHA) for the treatment of primary hyperoxaluria type 1 (PH1). This approval marks a significant advancement in the management of PH1, offering a new therapeutic option for patients aged nine years and older
- In April 2023, Chinook Therapeutics voluntarily paused dosing in its Phase 1 clinical trial of CHK-336, an investigational oral LDH inhibitor for PH1, following a serious adverse event of anaphylaxis in a participant. The company is conducting a thorough investigation to assess the safety profile of CHK-336 before proceeding further
