EMA rec­om­mends he­mo­phil­ia B gene ther­a­py for ap­proval, de­nies Y-mAbs neu­rob­las­toma drug

The Eu­ro­pean Med­i­cines Agency on Fri­day rec­om­mend­ed a con­di­tion­al mar­ket­ing au­tho­riza­tion for CSL’s Hem­genix (etranaco­gene deza­parvovec), the FDA-ap­proved, first gene ther­a­py for the treat­ment of he­mo­phil­ia B, which is an in­her­it­ed dis­ease that can in­crease bleed­ing.

He­mo­phil­ia B pa­tients cur­rent­ly re­quire life­long in­tra­venous in­fu­sions of fac­tor IX re­place­ment prod­ucts to main­tain suf­fi­cient fac­tor IX lev­els, and this is what Hem­genix is aim­ing to re­duce. While the long-term dura­bil­i­ty of the ther­a­py is un­known, EMA’s rec­om­men­da­tion is based on the re­sults of two open-la­bel, sin­gle-dose, sin­gle-arm stud­ies, in which 55/57 adult male pa­tients with mod­er­ate­ly se­vere or se­vere he­mo­phil­ia B sus­tained pos­i­tive treat­ment ef­fects for at least two years.

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