US FDA Pom Zoo C-hom Natriuretic Peptide Analogue Voxzogo (vosoritide) --- 1/2

BioMarin Pharmaceuticals tsis ntev los no tshaj tawm tias US Food and Drug Administration (FDA) tau nrawm qhov kev pom zoo ntawm Voxzogo (vosoritide), kev txhaj tshuaj ib hnub ntawm C-type natriuretic peptide (CNP) analogs rau kev kho mob ntawm 5 xyoos thiab hauv cov neeg mob me. nrog achondroplasia (achondroplasia) saum toj no thiab cov phaj loj hlob yog qhib, linear kev loj hlob yog nce. Achondroplasia yog qhov feem ntau tsis sib haum xeeb luv stature hauv tib neeg.

Thaum pom zoo Voxzogo, FDA kuj tau tshaj tawm daim ntawv pov thawj ntawm cov kab mob me me (PRV) rau BioMarin kom muab nqi zog rau lub tuam txhab rau nws cov txiaj ntsig zoo rau kev tsim cov tshuaj tshiab rau cov kab mob tsawg. Qhov PRV no tuaj yeem hloov pauv rau kev tshuaj xyuas qhov tseem ceeb ntawm cov tshuaj tshiab uas tsis ua raws li qhov tseem ceeb ntawm kev tshuaj xyuas, thiab tuaj yeem muag lossis xa mus.

Raws li kev txhim kho ntawm kev loj hlob txhua xyoo (AGV), qhov taw qhia tau pom zoo raws li cov txheej txheem kev pom zoo nrawm. Kev pom zoo txuas ntxiv rau qhov ntsuas no yuav yog nyob ntawm kev txheeb xyuas thiab piav qhia ntawm cov txiaj ntsig kho mob hauv kev tshawb fawb pom tseeb. Txhawm rau kom ua tau raws li qhov yuav tsum tau ua tom qab kev lag luam, BioMarin npaj siab yuav siv cov ntawv txuas txuas ntxiv txuas ntxiv mus rau kev sib piv nrog cov keeb kwm yav dhau los.

Hauv European Union, Voxzogo tau pom zoo thaum Lub Yim Hli 2021 rau kev kho mob achondroplasia hauv cov menyuam yaus hnub nyoog qis dua 2 xyoos kom kaw daim phiaj loj hlob. Hauv Tebchaws Meskas thiab European Union, Voxzogo tau tso cai Orphan Drug Designation (ODD) rau kev kho mob achondroplasia. Kaw ntawm lub phaj loj hlob tshwm sim tom qab puberty, thaum tus neeg laus qhov siab kawg mus txog.

Voxzogo yog thawj cov tshuaj pom zoo los kho cov menyuam yaus nrog achondroplasia. Nws tuaj yeem kho lub hauv paus ntawm tus kab mob. Nws sawv cev rau kev kho mob loj heev thiab muaj peev xwm muaj txiaj ntsig zoo rau cov neeg mob' nyob. Cov tshuaj nquag ntawm Voxzogo yog vosoritide, uas yog hloov kho C-hom natriuretic peptide (CNP) uas txhawb nqa cov pob txha endochondral los ntawm kev tswj hwm kev loj hlob ntawm fibroblast receptor 3 (FGFR3) taw qhia thiab ncaj qha lub hom phiaj kev loj hlob ntawm pob txha mos Lub sab hauv pathophysiology ntawm insufficiency.

Qhov kev pom zoo no yog ua raws li cov txiaj ntsig ntawm lub ntiaj teb randomized, ob qhov muag tsis pom kev, placebo-tswj Theem 3 txoj kev tshawb fawb thiab qhib daim ntawv lo txuas ntxiv ntawm Phase 3 txoj kev tshawb no. Txoj kev tshawb no tau tso npe rau 121 tus menyuam yaus uas muaj achondroplasia hnub nyoog 5 txog 14.9 xyoo. Lub hauv paus txhais tau tias AGV ntawm pawg placebo thiab pawg Voxzogo yog 4.06 cm / xyoo thiab 4.26 cm / xyoo, feem. Hauv 52nd lub lim tiam ntawm kev kho mob, qhov kev hloov ntawm AGV hauv pawg placebo los ntawm lub hauv paus yog -0.17 cm / xyoo, thiab kev hloov hauv AGV hauv pawg Voxzogo los ntawm lub hauv paus yog 1.40 cm / xyoo, ua rau pawg Voxzogo piv nrog cov pab pawg placebo raws li AGV. Kev txhim kho tseem ceeb hauv tsev kawm ntawv yog 1.57 cm / xyoo. Tom qab 52 lub lis piam ntawm ob qhov muag tsis pom kev placebo-tswj Phase 3 txoj kev tshawb fawb, 58 cov neeg mob uas tau pib random muab rau Voxzogo nkag mus rau lub sijhawm qhib-daim ntawv nthuav dav. Hauv cov neeg mob ua raws li 2 xyoos tom qab randomization, kev txhim kho ntawm AGV tseem tsis hloov pauv.