Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score an­oth­er ear­ly win at the FDA for Al­ny­lam.

The FDA put out word to­day that the agency has ap­proved its third drug, lumasir­an, for pri­ma­ry hy­per­ox­aluria type 1, bet­ter known as PH1. The news comes just 4 days af­ter the Eu­ro­pean Com­mis­sion took the lead in of­fer­ing a green light.

An ul­tra rare ge­net­ic con­di­tion, Al­ny­lam CEO John Maraganore says there are on­ly some 1,000 to 1,700 pa­tients in the US and Eu­rope at any par­tic­u­lar point. The pa­tients, most­ly kids, suf­fer from an over­pro­duc­tion of ox­alate in the liv­er that spurs the de­vel­op­ment of kid­ney stones, right through to end stage kid­ney dis­ease.

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