Ul­tragenyx lands first FDA OK, with an­oth­er on the hori­zon in shift to com­mer­cial ops

Ul­tragenyx CEO Emil Kakkis is now op­er­at­ing a com­mer­cial op­er­a­tion, just as he dreamed he would when he start­ed the com­pa­ny in 2010.

The FDA has ap­proved Mep­se­vii (ve­stron­idase al­fa-vjbk) for an ul­tra-rare ge­net­ic en­zyme dis­or­der called MPS VII, and the OK comes with a pri­or­i­ty re­view vouch­er that could eas­i­ly be worth $125 mil­lion.

The biotech $RARE has been sig­nal­ing this ad­vance for some time now, ea­ger­ly await­ing a green light along with a big ap­proval in line for buro­sum­ab, which has a much larg­er mar­ket in view. Mep­se­vii — a re­place­ment of the miss­ing be­ta-glu­curonidase en­zyme that caus­es skele­tal de­for­ma­tion as tox­ic ma­te­r­i­al builds up in cells — was test­ed in 23 pa­tients rang­ing from 5 months to 25 years, demon­strat­ing a sig­nif­i­cant gain in walk­ing dis­tance for the pa­tients in the drug arm.

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