Bio­Mar­in's pre­vi­ous CEO want­ed to of­fer mon­ey-back guar­an­tees to in­sur­ers for cov­er­ing its he­mo­phil­ia gene ther­a­py Roc­ta­vian. In­vestors in the com­pa­ny may wish that such a promise on per­for­mance could have al­so in­clud­ed them.

For all its block­buster hopes and high-pro­file sta­tus in the gene ther­a­py field, Roc­ta­vian's com­mer­cial saga has end­ed. Af­ter a few months of seek­ing to di­vest Roc­ta­vian, Bio­Marin said Mon­day it has failed to find "a qual­i­fied buy­er" for the gene ther­a­py and will vol­un­tar­i­ly with­draw it from the mar­ket.

The FDA has placed a par­tial clin­i­cal hold on Macro­Gen­ics’ Phase 2 study in gy­ne­co­log­ic can­cers af­ter one pa­tient died and three oth­ers ex­pe­ri­enced life-threat­en­ing side ef­fects, the com­pa­ny dis­closed Tues­day.

The pa­tient who died had “grade 4 neu­trope­nia and con­cur­rent sep­tic shock," the com­pa­ny said in a press re­lease. The oth­er three grade 4 in­ci­dents in­clud­ed two cas­es of throm­bo­cy­tope­nia and one case of my­ocardi­tis, a type of heart in­flam­ma­tion.

Neu­trope­nia is an ab­nor­mal­ly low lev­el of a spe­cif­ic kind of white blood cells, ham­per­ing the body’s abil­i­ty to fight in­fec­tions. Throm­bo­cy­tope­nia is a low lev­el of platelets in the blood, lead­ing to high­er risks of bleed­ing and bruis­ing.

Macro­Gen­ics said 41 pa­tients have tak­en loriger­limab so far, and those in the study can con­tin­ue to take the drug.

Swedish biotech Im­med­ica won an ac­cel­er­at­ed ap­proval from the FDA, res­ur­rect­ing the first treat­ment to ad­dress per­sis­tent­ly el­e­vat­ed lev­els of plas­ma argi­nine, which is the pri­ma­ry dri­ver of the rare dis­ease ARG1-D.

Mon­day's ap­proval of Loar­gys, an argi­nine-spe­cif­ic en­zyme, fol­lows a com­plete re­sponse let­ter in Au­gust and Im­med­ica's ac­qui­si­tion of the com­pound in 2022 af­ter the bank­rupt­cy of Ae­glea Bio­Ther­a­peu­tics. Ae­glea in 2022 re­ceived a refuse-to-file let­ter from the FDA, which sought more “ev­i­dence show­ing that plas­ma argi­nine and metabo­lite re­duc­tion pre­dicts clin­i­cal ben­e­fit in pa­tients with ARG1-D."

Im­med­ica will win a pri­or­i­ty re­view vouch­er as part of the ap­proval, which could pro­vide the KKR-backed biotech with $100 mil­lion to $200 mil­lion when it's sold.