EU Pom Zoo Aspaveli: Kev Kho Mob Ntawm Paroxysmal Nocturnal Hemoglobinuria (PNH)

Apellis Pharmaceuticals thiab Swedish Orphan Biovitrum AB (Sobi) tsis ntev los no tshaj tawm tias European Commission (EC) tau pom zoo Aspaveli (pegcetacoplan) rau kev kho mob ntawm paroxysmal pw tsaug zog los ntawm anemia tom qab tau txais C5 inhibitors tsawg kawg yog 3 lub hlis. Cov neeg laus nrog kev sib deev hemoglobinuria (PNH). Thaum lub Tsib Hlis 2021, cov tshuaj yog thawj zaug pom zoo hauv Tebchaws Meskas raws li lub npe lag luam Empaveli. Nws tuaj yeem siv rau cov neeg laus PNH uas tsis tau kho yav dhau los, thiab tseem tuaj yeem siv rau PNH cov neeg laus uas tau hloov kho los ntawm C5 inhibitors (Soliris thiab Ultomiris). Kev pom zoo ntawm pegcetacoplan rau kev lag luam yuav tsum txhim kho tus qauv ntawm PNH kev saib xyuas thiab kho dua tshiab ntawm PNH.

Nws yog ib qho tsim nyog hais tias pegcetacoplan yog thawj zaug thiab tsuas yog C3 tsom rau kev kho kom tau txais kev pom zoo ntawm kev tswj hwm. Hauv kaum xyoo dhau los los yog li ntawd, tib txoj kev kho mob ntawm PNH yog C5 inhibitors, tab sis ntau tus neeg mob tseem muaj kev pheej hmoo ntawm hypohemoglobinopathy, feem ntau ua rau qaug zog qaug zog thiab nquag ntshav. Hauv kev sim tshuaj, pegcetacoplan tuaj yeem muab kev tswj hwm ntau ntawm PNH, txhim kho lub neej ntawm PNH cov neeg mob los ntawm kev nce qib hemoglobin thiab txo qis kev xav tau ntawm cov ntshav.

pegcetacoplan tau txais kev pom zoo kev tswj hwm hauv Tebchaws Meskas thiab European Union, raws li cov txiaj ntsig ntawm lub taub hau mus rau lub taub hau 3 PEGASUS txoj kev tshawb fawb (NCT03500549) (saib: PEGASUS Phase 3 Top-Line Results Conference Call Presentation). Txoj kev tshawb no mus txog qhov kawg ntawm qhov kawg, qhia tau hais tias pegcetacoplan zoo dua Soliris, PNH tus qauv ntawm kev saib xyuas cov tshuaj: nws zoo dua li Soliris hauv kev hloov pauv ntawm qib hemoglobin los ntawm theem pib ntawm lub lim tiam 16, thiab qib hemoglobin kho tau nce 3.84. g/dL nyob nruab nrab (p<>

Tsis tas li ntawd, pegcetacoplan tau ua tiav qhov tsis muaj qhov ua tsis tau zoo hauv kev zam cov ntshav cov ntsiab lus kawg piv rau Soliris. 85% ntawm cov neeg mob nyob rau hauv pab pawg kho mob pegcetacoplan tsis muaj ntshav ntxiv nyob rau hauv 16 lub lis piam, piv nrog 15% hauv pab pawg kho mob Soliris. Nyob rau tib lub sijhawm, piv nrog cov neeg mob hauv pab pawg kho mob Soliris, cov neeg mob hauv pab pawg kho mob pegcetacoplan muaj qhov ua kom zoo dua qub ntawm cov cim tseem ceeb hemolysis, thiab cov qhab nia FACIT- qaug zog muaj kev txhim kho tseem ceeb hauv kev kho mob. Hauv txoj kev tshawb no, pegcetacoplan muaj kev nyab xeeb zoo li Soliris.

pegcetacoplan mechanism ntawm kev txiav txim

pegcetacoplan is the first therapy to show superior hemoglobin levels than Soliris, and up to 85% of patients treated with pegcetacoplan do not have a blood transfusion. Most of the PNH patients currently receiving Soliris treatment suffer from persistent anemia. The results of the PEGASUS study show that pegcetacoplan has the potential to become a new standard of care for patients with PNH.

Pegcetacoplan yog lub hom phiaj C3 inhibitor tsim los tswj kev ua kom muaj zog ntau ntxiv, uas yog qhov ua rau tshwm sim thiab kev loj hlob ntawm ntau yam kab mob loj. Pegcetacoplan yog ib qho hluavtaws cyclic peptide uas khi rau polyethylene glycol polymer thiab tshwj xeeb khi rau C3 thiab C3b. Tam sim no, pegcetacoplan tab tom tsim los kho ntau yam kab mob, suav nrog PNH, thaj chaw atrophy (GA) thiab C3 glomerupathies. Hauv Tebchaws Meskas, FDA tau tso cai rau pegcetacoplan ceev kev tsim nyog rau kev kho mob ntawm PNH thiab GA.

Tam sim no, tsis muaj tshuaj los kho GA. Apellis npaj yuav xa daim ntawv thov tshuaj tshiab rau kev txhaj tshuaj intravitreal ntawm pegcetacoplan los kho cov hnub nyoog ntsig txog macular degeneration (AMD) theem nrab GA hauv 2022.