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

Mechanism ntawm kev ua ntawm vosoritide

Achondroplasia yog qhov feem ntau tsis sib haum xeeb luv stature hauv tib neeg. Nws yog tus cwj pwm los ntawm kev qeeb endochondral ossification, ua rau qhov tsis sib haum xeeb luv luv thiab cov txheej txheem ntawm cov pob txha ntev, txha nraub qaum, ntsej muag, thiab pob txha taub hau. Qhov mob no yog tshwm sim los ntawm kev hloov pauv hauv fibroblast kev loj hlob yam tseem ceeb receptor 3 noob (FGFR3), uas yog tus tswj tsis zoo ntawm cov pob txha loj hlob.

Ntxiv nrog rau qhov qis qis qis, cov neeg mob achondroplasia tuaj yeem ntsib teeb meem kev noj qab haus huv loj, suav nrog foramen compression, pw tsaug zog apnea, khoov ob txhais ceg, lub ntsej muag hypoplasia, mus tas li rov qab viav vias, txha nraub qaum, thiab pob ntseg rov qab. Department of infection. Qee qhov teeb meem no yuav ua rau xav tau kev phais mob, xws li txha caj qaum decompression thiab straightening ntawm ob txhais ceg. Tsis tas li ntawd, cov kev tshawb fawb tau pom tias cov neeg tuag tau nce ntxiv rau txhua pab pawg hnub nyoog.

Ntau tshaj 80% ntawm cov niam txiv ntawm cov menyuam yaus uas muaj achondroplasia yog qhov nruab nrab, thiab tus kab mob no tshwm sim los ntawm kev hloov pauv ntawm caj ces. Ntiaj teb no tshwm sim ntawm achondroplasia yog hais txog ib tug ntawm 25,000 yug nyob.

Vosoritide yog C-hom natriuretic peptide (CNP) analog tau los ntawm tib neeg peptides thiab yog ib qho stimulator zoo ntawm endochondral ossification. Ntuj tib neeg peptide yog ib tug zoo regulator ntawm pob txha loj hlob. Vosoritide khi rau cov receptors tshwj xeeb thiab pib intracellular cov cim qhia uas cuam tshuam txoj hauv kev tshaj FGFR3.

Tam sim no, vosoritide tab tom raug tshuaj xyuas rau cov menyuam yaus uas lub phaj loj hlob tseem" qhib", feem ntau yog cov menyuam yaus hnub nyoog qis dua 18 xyoo. Cov neeg mob no suav txog kwv yees li 25% ntawm cov neeg mob achondroplasia. Hauv Latin America, Middle East, thiab feem ntau ntawm thaj av Asia-Pacific, tam sim no tsis muaj kev pom zoo rau cov tshuaj siv los kho achondroplasia.