Purespring Therapeutics granted European Medicines Agency (EMA) orphan drug designation

London, 28 April 2025 - Purespring Therapeutics, a pioneering company focused on transforming the treatment of kidney diseases, today announces that the European Medicines Agency (EMA) has granted orphan drug designation to PS-002, Purespring’s lead programme, in IgA nephropathy (IgAN).

Orphan drug designation in the European Union (EU) is granted by the European Commission based on a positive opinion adopted by the EMA Committee for Orphan Medicinal Products. Orphan designation provides incentives to developers of medicines for limited patient populations, including market exclusivity, protocol assistance (guidance on study design and scientific evaluation) and regulatory fee reductions.

IgAN is a common, chronic autoimmune kidney disease that primarily affects young adults. In patients with the disease, a protein called immunoglobulin A (IgA) becomes trapped in the filters of the kidney, known as glomeruli, causing inflammation, damage and scarring to the whole kidney. There are currently no approved therapeutic options to stop disease progression, nor a cure, and about one third of high-risk IgAN patients will go on to lose kidney function within five years and require a kidney transplant or dialysis.

Haseeb Ahmad, Purespring’s Chief Executive Officer, commented:

Orphan drug designation will significantly support Purespring’s goal of progressing PS-002 through clinical development and, more broadly, with bringing solutions for kidney disease indications to patients.

We’re delighted that the EMA has recognised the promise of this potential breakthrough medicine and we look forward to working with them on the development pathway.

By developing a novel approach to treat IgAN, we believe we can offer hope to patients across Europe and globally who currently have limited therapeutic options, furthering our mission to transform the lives of people suffering from kidney diseases.

Supported by an oversubscribed £80 million / $105 million Series B financing, which completed in October 2024, Purespring is progressing PS-002 towards a Phase I/II clinical trial for IgAN, with additional gene therapy programmes in development for the treatment of both rare and common kidney diseases.

Purespring is the first company to successfully treat kidney disease models by directly targeting the podocyte, a specialised cell implicated in approximately 60% of renal disease, through its proprietary adeno-associated viral (AAV) gene therapy platform. As detailed in a presentation at the American Society of Nephrology (ASN) Kidney Week 2024, Purespring has previously demonstrated the potential of PS-002 as an important novel modality to treat IgAN.