Investment in rare disease biotechs has increased following renewal of the FDA’s Priority Review Voucher (PRV) programme, according to SynaptixBio, but the United States remains dominant compared to the UK. In February 2026, the PRV programme was signed into law and will remain in place until reviewed again in September… Read More
Brian Malkin and Sadaf Deedar, Spencer Fane LLP, unpack the recent changes to the FDA’s Priority Review Voucher scheme and the upcoming review of its effectiveness in promoting drug development for rare paediatric diseases. Congress recently enacted the Consolidated Appropriations Act, 2026 (CAA)… Read More
The latest episode of the DDW Highlights Podcast is now available to listen to below. DDW’s Bruno Quinney narrates five key stories of the previous week to keep DDW subscribers up-to-date on the latest industry news. Last week, researchers made a key discovery into cancer survival. Elsewhere, a global research effort… Read More
News round-up for 9 – 13 February by Bruno Quinney, Content Team at DDW. This week in DDW, researchers have made a key discovery into cancer survival. Elsewhere, a global research effort into kidney disease is underway. The top stories: Researchers identify RNA linked to cancer… Read More
The US Senate has reauthorised the US Food and Drug Administration (FDA)’s Priority Review Voucher (PRV), helping biotech companies to develop rare disease drugs. The PRV programme, which incentivises rare disease drug development, was reauthorised on 30 January, with President Donald Trump signing the bill. … Read More
The renewal of the US priority review voucher (PRV) is vital for the biotech sector, SynaptixBio CEO Dan Williams has said. The US Food and Drug Administration (FDA)’s PRV scheme, designed to incentivise rare disease drug development, was reauthorised by the US House of Representatives on December 1. … Read More
Madrigal Pharmaceuticals has submitted an application for resmetirom, a drug for nonalcoholic steatohepatitis (NASH) with liver fibrosis, to the US Food and Drug Administration (FDA) and has requested a priority review. Resmetirom is a once daily, oral, thyroid hormone receptor (THR)-β selective agonist designed to target… Read More
The European Medicines Agency (EMA) has granted PRIME (Priority Medicines) status to the gene therapy, GNT-0003, currently in clinical trials for Crigler-Najjar syndrome, a rare liver disease. This status, granted only to drug candidates with major therapeutic potential, follows the successful completion of the first phases of… Read More
The US Food and Drug Administration (FDA) has designated the investigation of Endogena Therapeutics’ EA-2353 for the treatment of retinitis pigmentosa (RP) as a Fast Track development programme. Fast Track is a process designed to enable patients to benefit earlier from important new drugs for serious conditions. … Read More
The European Medicines Agency (EMA) has granted priority medicines (PRIME) designation to FBX-101, Forge Biologic’s adeno-associated virus (AAV) drug candidate for treating patients with Krabbe disease. PRIME is a regulatory designation by the EMA that provides early and proactive support to developers of promising medicines, to advance… Read More
The US Food and Drug Administration (FDA) has accepted Sarepta Therapeutics’ Biologics License Application (BLA) seeking accelerated approval of SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulant individuals with Duchenne muscular dystrophy. The therapy, which is being developed in partnership with Roche, has been granted Priority Review… Read More
