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news.The European Medicines Agency (EMA) has granted orphan drug designation to UK-based Purespring Therapeutics’ lead candidate, PS-002, aimed at treating IgA nephropathy (IgAN).
IgAN is characterised by the accumulation of immunoglobulin A in the kidney's glomeruli. Credit: Robina Weermeijer on Unsplash.
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This status is conferred by the European Commission following a positive opinion from the EMA Committee for orphan medicinal products and offers several benefits, including market exclusivity and regulatory fee minimisations.
IgAN is said to be a chronic autoimmune kidney condition, predominantly affecting the young adult population.
It is characterised by the accumulation of immunoglobulin A in the kidney’s glomeruli, leading to inflammation and potential kidney failure.
At present, no approved treatments can halt the progression of IgAN, leaving individuals at risk of requiring dialysis or kidney transplants within five years.
With the completion of £80m in the Series B funding round last October, the company is advancing PS-002 towards a Phase I/II trial.
Leveraging its adeno-associated viral (AAV) gene therapy platform, the company claims to treat models of kidney disease by targeting directly at a specialised cell, the podocyte, which is associated with nearly more than half of the renal conditions.
The company showed the therapy’s potential earlier as a key modality for treating IgAN.
Purespring Therapeutics CEO Haseeb Ahmad said: “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.”
