PHAR - Pharming's rare immunodeficiency disorder drug gets FDA priority review
- The U.S. Food and Drug Administration ((FDA) granted priority review to Pharming ( NASDAQ: PHAR ) application seeking approval of oral drug leniolisib to treat the rare primary immunodeficiency activated phosphoinositide 3-kinase delta syndrome (APDS) in patients 12 years of age and older in the U.S.
- The FDA accepted the company's New Drug Application (NDA) and is expected to make a decision by March 29, 2023. Under priority review, the FDA's goal is to take action within six months, compared to 10 months under standard review.
- The NDA was backed by data from a phase 2/3 study of leniolisib, which met its co-primary goals of reduction in index lymph node size and correction of immunodeficiency, the Netherlands-based company said in a Sept. 28 press release.
- APDS is a rare immunodeficiency disease that affects 1 to 2 people per million and is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells, according to the company.
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Pharming's rare immunodeficiency disorder drug gets FDA priority review