The US Food and Drug Administration (FDA) has granted orphan drug designation to Poolbeg’s preventative therapy for cancer immunotherapy-induced Cytokine Release Syndrome (CRS).
The treatment, named POLB 001, is an oral preventative therapy to treat inflammation in blood and tissues. CRS is a severe side-effect that occurs in more than 70 per cent of patients, leading to severe side-effects or death.
Cytokines can sweep throughout the body and cause tissue damage and shut down circulation and other essential organs.
Orphan status is granted by the FDA for to support the development of treatments for rare disorders that affect under 200,000 people in the US, and makes the development of the drug less risky for Poolbeg, with the potential for seven-year period of US market exclusivity following approval of the treatment, waiver exemption of some fees and tax credits for qualified clinical trials.
“POLB 001 is potentially a breakthrough, orally delivered, preventative therapy for cancer immunotherapy-induced CRS which could significantly impact patients’ lives,” said Poolbeg chief executive Jeremy Skillington.
“We were delighted to receive Orphan Drug Designation from the FDA, which is a significant development for Poolbeg and for POLB 001, one that we believe will enhance the commercial appeal for prospective partners and help bring POLB 001 to the market faster. If approved, we believe POLB 001 has the potential to improve quality of life for patients, reduce pressure on healthcare systems, and expand access to cancer immunotherapies.”
There are currently no approved preventative therapies for CRS. The first patients are expected to get the drug in the second half of the year, as part of Poolbeg’s Phase 2a trial.
“Orphan Drug Designation from the FDA underscores the urgency and importance of developing innovative therapies for this critical unmet medical need,” said Professor Brendan Buckley, Poolbeg non-executive director and a member of the scientific advisory board.
“We look forward to progressing POLB 001 in our upcoming Phase 2a clinical trial and working closely with prospective partners and regulatory agencies to bring this potential therapy to patients as quickly as possible.”
