Tsoom Fwv Teb Chaws Asmeskas FDA tau muab thawj qhov kev tshuaj xyuas Hetlioz (melatonin receptor agonist): thawj zaug Smith-Maggie syndrome (SMS) tshuaj!

Tsis ntev los no Vanda Pharmaceuticals tshaj tawm tias Tsoomfwv Meskas Cov Khoom Noj thiab Tshuaj (FDA) tau lees txais ob daim ntawv thov xa los ntawm lub tuam txhab rau Smith-Magenis Syndrome (Smith-Magenis Syndrome, SMS) thiab tau tso cai kev tshuaj xyuas ua ntej.

Daim ntawv thov suav nrog kev thov tshuaj tshiab (sNDA) rau Hetlioz (tasimelteon) tsiav tshuaj thiab Daim Ntawv Thov Tshuaj Tshiab (NDA) rau Hetlioz (tasimelteon) cov kua tshuaj sib tov, uas tau siv los kho SMS cov laus thiab cov menyuam, ib qho. FDA tau tsim muaj Daim Ntawv Qhia Txog Tshuaj Rau Cov Neeg Siv Tshuaj (PDUFA) hnub tim lub Kaum Ob Hlis 1, 2020.

Mihael H. Polymeropoulos, MD, Thawj Tswj Hwm thiab CEO ntawm Vanda, hais tias:" FDA' s lees txais Hetlioz daim ntawv thov thiab kev saib xyuas qhov tseem ceeb yog lub cim tseem ceeb uas coj peb los ze zog muab cov kev kho mob tseem ceeb rau cov neeg mob SMS. Tam sim no, tsis muaj kev kho mob tau pom zoo. Cov tshuaj SMS, uas yog tus mob tsis tshua muaj, cuam tshuam rau 15,000 tus neeg hauv Tebchaws Meskas."

Smith-Margilli Syndrome (SMS) yog ib qho kev txhim kho kev loj hlob tshwm sim los ntawm kev txiav ib feem me me ntawm tib neeg 17p chromosome. Hauv qee qhov teeb meem tsawg tsawg, SMS yog tshwm sim los ntawm kev sib hloov taw tes hauv lub RAI1 gene, uas yog nyob hauv thaj av uas ploj lawm.

Khwv yees tias hauv Tebchaws Meskas, qhov tshwm sim ntawm SMS hauv cov menyuam yug tshiab yog 1 / 15,000-25,000. SMS yog ib txwm tsis muaj kev tau txais, tab sis vim de-novo deletion. Cov neeg uas SMS yuav muaj qee qhov teeb meem lub cev, lub hlwb, thiab kev coj cwj pwm. Cov tsos mob feem ntau ntawm SMS yog kev pw tsaug zog tsis zoo, thiab lub neej ntawm cov neeg mob thiab lawv tsev neeg muaj kev cuam tshuam loj heev.

Tasimelteon qauv qauv tsim (duab qhov chaw: link.springer.com)

Hetlioz (tasimelteon) yog melatonin receptor agonist. Hauv Tebchaws Meskas thiab European Union, Hetlioz tau pom zoo rau kev kho mob tsis-24-teev pw tsaug zog tsis meej (tsis yog-24-teev-pw-wakedisorder," Tsis-24" ;," tsis- 24") hauv cov neeg mob uas muaj lub qhov muag tsis pom kev, uas yog Thawj Cov Tshuaj Siv rau qhov ntsuas no.

Tam sim no, Hetlioz tseem tsis tau pom zoo los ntawm ib lub koom haum tswj kev lag luam rau kev kho SMS.