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Former Novartis drug for ultra-rare disease gets speedy FDA review
Three years after paying a modest $20 million upfront to grab a late-stage rare disease drug from Novartis, Pharming has lined up a priority review at…
Three years after paying a modest $20 million upfront to grab a late-stage rare disease drug from Novartis, Pharming has lined up a priority review at the FDA.
Known at the time as CDZ173, leniolisib belongs to the PI3Kẟ (phosphoinositide 3-kinase delta) inhibitor class, which is best known for cancer applications. But Novartis took a different path, testing it for an immunodeficiency disease called activated phosphoinositide 3-kinase delta syndrome, or APDS.
According to Pharming, the condition affects around one to two people per million and can be extremely severe for those patients, leading to repeated sinopulmonary infections and a variety of symptoms that can end in organ damage.
Anurag Relan, Pharming’s CMO, noted that patients currently rely on supportive therapies such as antibiotics and immunoglobulin replacement therapy.
Pharming’s small molecule drug is designed to tamp down the hyperactivity of PI3Kẟ, thus reversing the dysfunctional immune system.
As part of their deal, Novartis was in charge of the Phase II/III registration study, which showed that leniolisib reduced index lymph node size and normalized immune function — as measured by an increased proportion of naïve B cells — in the target population. The pharma giant also conducted an open-label extension trial, data from which were also included in Pharming’s FDA package.
The PDUFA date is set for March 29, 2023.
The Dutch biotech noted that if approved, it will likely be commercializing leniolisib among the same group of immunologists and hematologists it already works with on its approved drug for hereditary angioedema.
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