Sijmen de Vries, Pharming CEO

Up­dat­ed: FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US reg­u­la­tors cleared an ul­tra-rare drug from Pharm­ing Group, by way of No­var­tis, on Fri­day af­ter­noon.

The Dutch biotech said the FDA green­lit le­niolis­ib for an im­mun­od­e­fi­cien­cy dis­ease known as ac­ti­vat­ed phos­pho­inosi­tide 3-ki­nase delta (PI3Kδ) syn­drome, or APDS. Peo­ple 12 years and old­er can re­ceive the oral drug, to be mar­ket­ed as Joen­ja, be­gin­ning ear­ly next month, Pharm­ing said, five days ahead of the de­ci­sion dead­line set by the FDA as part of a pri­or­i­ty re­view.

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