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(2) タᐃ᰿ᣐ

1.9.3 ᵓ㐀ᘧ

࢔࣑ࣀ㓟㓄ิཬࡧࢪࢫࣝࣇ࢕ࢻ⤖ྜ㸸 L㙐

H㙐

H㙐E1㸸㒊ศⓗࣆࣟࢢࣝࢱ࣑ࣥ㓟㸹H㙐N302㸸⢾㙐⤖ྜ㸹H㙐K452㸸㒊ศⓗࣉࣟࢭࢩࣥࢢ L㙐C214㸫H㙐C225㸪H㙐C231㸫H㙐C231㸪H㙐C234㸫H㙐C234㸸ࢪࢫࣝࣇ࢕ࢻ⤖ྜ

EIVLTQSPAT LSLSPGERAT LSCRASQSVS SYLAWYQQKP GQAPRLLIYD ASNRATGIPA RFSGSGSGTD FTLTISSLEP EDFAVYYCQQ RSNWPPTFGQ GTKVEIKRTV AAPSVFIFPP SDEQLKSGTA SVVCLLNNFY PREAKVQWKV DNALQSGNSQ ESVTEQDSKD STYSLSSTLT LSKADYEKHK VYACEVTHQG LSSPVTKSFN RGEC

EVQLLESGGG LVQPGGSLRL SCAVSGFTFN SFAMSWVRQA PGKGLEWVSA ISGSGGGTYY ADSVKGRFTI SRDNSKNTLY LQMNSLRAED TAVYFCAKDK ILWFGEPVFD YWGQGTLVTV SSASTKGPSV FPLAPSSKST SGGTAALGCL VKDYFPEPVT VSWNSGALTS GVHTFPAVLQ SSGLYSLSSV VTVPSSSLGT QTYICNVNHK PSNTKVDKRV EPKSCDKTHT CPPCPAPELL GGPSVFLFPP KPKDTLMISR TPEVTCVVVD VSHEDPEVKF NWYVDGVEVH NAKTKPREEQ YNSTYRVVSV LTVLHQDWLN GKEYKCKVSN KALPAPIEKT ISKAKGQPRE PQVYTLPPSR EEMTKNQVSL TCLVKGFYPS DIAVEWESNG QPENNYKTTP PVLDSDGSFF LYSKLTVDKS RWQQGNVFSC SVMHEALHNH YTQKSLSLSP GK

ࢲࣛࢶ࣒࣐ࣈ 1.9 ୍⯡ⓗྡ⛠࡟ಀࡿᩥ᭩

୺࡞⢾㙐ࡢ᥎ᐃᵓ㐀㸸

C6466H9996N1724O2010S42㸦ࢱࣥࣃࢡ㉁㒊ศ㸪4ᮏ㙐㸧 H㙐㸸C2204H3404N582O672S16

L㙐㸸C1029H1598N280O333S5

Fuc GlcNAc Man

Gal 0-2 Man GlcNAc GlcNAc GlcNAc Man

ࢲࣛࢶ࣒࣐ࣈ 1.10 ẘ⸆࣭๻⸆➼ࡢᣦᐃᑂᰝ㈨ᩱࡢࡲ࡜ࡵ

1.10 ẘ⸆࣭๻⸆➼ࡢᣦᐃᑂᰝ㈨ᩱࡢࡲ࡜ࡵ

ẘ⸆࣭๻⸆➼ࡢᣦᐃᑂᰝ࡟ಀࡿ㈨ᩱ࡟ࡘ࠸࡚㸪ᮏ⏦ㄳ࡟ಀࡿෆᐜࢆḟ࣮࣌ࢪࡼࡾ♧ࡍࠋ

ࢲࣛࢶ࣒࣐ࣈ 1.10 ẘ⸆࣭๻⸆➼ࡢᣦᐃᑂᰝ㈨ᩱࡢࡲ࡜ࡵ

໬Ꮫྡ࣭

ูྡ

ࢲࣛࢶ࣒࣐ࣈࡣ㸪ࣄࢺCD38࡟ᑐࡍࡿ㑇ఏᏊ⤌᥮࠼ࣄࢺIgG1ࣔࣀࢡ࣮ࣟࢼࣝᢠయ࡛࠶

ࡿࠋࢲࣛࢶ࣒࣐ࣈࡣ㸪ࢳࣕ࢖ࢽ࣮ࢬࣁ࣒ࢫࢱ࣮༸ᕢ⣽⬊࡟ࡼࡾ⏘⏕ࡉࢀࡿࠋࢲࣛࢶ࣒

࣐ࣈࡣ㸪452ಶࡢ࢔࣑ࣀ㓟ṧᇶ࠿ࡽ࡞ࡿH㙐㸦Ț1㙐㸧2ᮏཬࡧ214࢔࣑ࣀ㓟ṧᇶ࠿ࡽ

࡞ࡿL㙐㸦ȡ㙐㸧2ᮏ࡛ᵓᡂࡉࢀࡿ⢾ࢱࣥࣃࢡ㉁㸦ศᏊ㔞㸸⣙148,000㸧࡛࠶ࡿࠋ 㸦ูྡ㸸ࢲࣛࢶ࣒࣐ࣈ㸦㑇ఏᏊ⤌᥮࠼㸧ཬࡧࡑࡢ〇๣㸧

ᵓ㐀ᘧ ࢔࣑ࣀ㓟㓄ิཬࡧ୺࡞⢾㙐ࡢ᥎ᐃᵓ㐀ࡣู⣬࡟グ㍕ࡍࡿࠋ

ຠ⬟࣭ຠ

෌Ⓨཪࡣ㞴἞ᛶࡢከⓎᛶ㦵㧊⭘

⏝ἲ࣭⏝

㏻ᖖ㸪ᡂே࡟ࡣࢲࣛࢶ࣒࣐ࣈ㸦㑇ఏᏊ⤌᥮࠼㸧࡜ࡋ࡚㸪1 ᅇ16 mg/kg ࢆ௨ୗࡢᢞ୚㛫 㝸࡛Ⅼ⁲㟼ὀࡍࡿࠋ

ࣞࢼࣜࢻ࣑ࢻཬࡧࢹ࢟ࢧ࣓ࢱࢰࣥే⏝ࡢሙྜ㸸

1 㐌㛫㛫㝸㸦1㹼8 㐌┠㸧㸪2㐌㛫㛫㝸㸦9㹼24 㐌┠㸧ཬࡧ4㐌㛫㛫㝸㸦25㐌┠௨㝆㸧

࣎ࣝࢸࢰ࣑ࣈཬࡧࢹ࢟ࢧ࣓ࢱࢰࣥే⏝ࡢሙྜ㸸

1 㐌㛫㛫㝸㸦1㹼9 㐌┠㸧㸪3 㐌㛫㛫㝸㸦10㹼24 㐌┠㸧ཬࡧ4 㐌㛫㛫㝸㸦25 㐌┠௨㝆㸧

๻⸆➼ࡢ ᣦᐃ ᕷ㈍ྡཬ ࡧ᭷ຠᡂ ศ࣭ศ㔞

ཎయ㸸ࢲࣛࢶ࣒࣐ࣈ㸦㑇ఏᏊ⤌᥮࠼㸧

〇๣㸸ࢲࣛࢨࣞࢵࢡࢫⅬ⁲㟼ὀ100 mg㸦1ࣂ࢖࢔ࣝ୰㸪ࢲࣛࢶ࣒࣐ࣈ㸦㑇ఏᏊ⤌

᥮࠼㸧ࢆ100 mgྵ᭷㸧㸪ࢲࣛࢨࣞࢵࢡࢫⅬ⁲㟼ὀ400mg㸦1ࣂ࢖࢔ࣝ୰㸪ࢲࣛࢶ

࣒࣐ࣈ㸦㑇ఏᏊ⤌᥮࠼㸧ࢆ400 mgྵ᭷㸧 ẘᛶ ༢ᅇᢞ୚ẘᛶ

ື≀✀ ᢞ୚⤒㊰ ᴫ␎ࡢ⮴Ṛ㔞㸦mg/kg㸧1)

ࢳࣥࣃࣥࢪ࣮ 㟼⬦ෆ ܴܲ㸸5

1) ༢ᅇᢞ୚ẘᛶヨ㦂ࢆᐇ᪋ࡋ࡚࠸࡞࠸ࡓࡵ㸪6㐌㛫཯᚟ᢞ୚ẘᛶヨ㦂ࡢึᅇᢞ୚᫬ࡢ⤖ᯝ࠿ࡽồࡵࡓࠋ

཯᚟ᢞ୚ẘᛶ

ື≀✀ ᢞ୚ᮇ㛫 ᢞ୚⤒㊰

ᢞ୚㔞 (mg/kg/㐌)

↓ẘᛶ㔞

(mg/kg/㐌) ୺࡞ᡤぢ

ࢳࣥࣃ

ࣥࢪ࣮

6㐌㛫 (1ᅇ/㐌)

㟼⬦ෆ

0 Ѝ 5 2)

0 Ѝ 25 3) < 5

Ӎ5: Ẽ⟶⢓ᾮЌ㸪᤼౽㸪ዲ୰⌫࣭APTT Ќ㸪ࣜࣥࣃ⌫࣭⾑ᑠᯈ࣭IgG࣭IgMЎ 5: Ṛஸ㸦ܲ1㸧

Ṛஸ౛࡟ࡳࡽࢀࡓᡤぢ㸸ࢧ࢖ࢺ࢝࢖ࣥ

ᨺฟ཯ᛂ㸦ࡃࡋࡷࡳ㸪TNF-Į࣭IL-6࣭

IFN-ȖЌ㸪Ẽ⟶ཬࡧ㰯Ꮝ࠿ࡽἻἓ≧ᾮ యࡢ኱㔞᤼ฟ㸧

25: ࢧ࢖ࢺ࢝࢖ࣥᨺฟ཯ᛂ㸦ࡃࡋࡷࡳ㸪⢓

ᾮЌ㸧㸪⢓⭷⵬ⓑ㸪ୗ⑩㸪㌾౽㸪㣗ḧ పୗ

2) ⁐፹ᢞ୚ࡢ1㐌㛫ᚋ࡟㸪ࢲࣛࢶ࣒࣐ࣈ 5 mg/kgࢆ30ศ㛫࠿ࡅ࡚㟼⬦ෆᣢ⥆ᢞ୚㸦௨ᚋ㸪1㐌㛫ࡈ࡜࡟

ࢲࣛࢶ࣒࣐ࣈ 5 mg/kgࢆィ6ᅇ㸪㟼⬦ෆᣢ⥆ᢞ୚㸧ࠋ

3) 5 mg/kgࡢึᅇ㟼⬦ෆᣢ⥆ᢞ୚᫬࡟ࢧ࢖ࢺ࢝࢖ࣥᨺฟ཯ᛂࡀㄆࡵࡽࢀ㸪㞤1౛ࡀṚஸࡋࡓࡓࡵ㸪㐣๫

࡞ࢧ࢖ࢺ࢝࢖ࣥᨺฟࢆண㜵ࡍࡿ┠ⓗ࡛㸪⁐፹ᢞ୚ࡢ2㐌㛫ᚋ࡟ࢲࣛࢶ࣒࣐ࣈ10 mgࢆ㟼⬦ෆ࡟࣮࣎ࣛ

ࢫ࡛๓ᢞ୚ࡋ㸪ࡑࡢ24᫬㛫ᚋ࡟ࢲࣛࢶ࣒࣐ࣈ 25 mg/kgࢆ1᫬㛫࠿ࡅ࡚㟼⬦ෆᣢ⥆ᢞ୚ࠋࢲࣛࢶ࣒࣐

ࣈࡢ2㹼6ᅇ┠ࡢ㟼⬦ෆᣢ⥆ᢞ୚᫬㛫ࡣ30ศ㛫ࠋ

ࢲࣛࢶ࣒࣐ࣈ 1.10 ẘ⸆࣭๻⸆➼ࡢᣦᐃᑂᰝ㈨ᩱࡢࡲ࡜ࡵ

๪స⏝ ๪స⏝Ⓨ⌧⋡㸦⮫ᗋ᳨ᰝ್␗ᖖࢆྵࡴ㸧 511/ 674౛㸻75.8%

㹙᪥ᮏࢆྵࡴᅜ㝿ඹྠ➨III ┦ヨ㦂㸦54767414MMY3003 ヨ㦂㸧㸪ᾏእ➨III ┦ヨ㦂 㸦54767414MMY3004 ヨ㦂㸧㸪ᾏእ➨II ┦ヨ㦂㸦54767414MMY2002 ヨ㦂 16mg/kg ࡢࡳ㸧㸪ᾏእ➨I/II ┦ヨ㦂㸦GEN501 ヨ㦂 Part 2 16mg/kgࡢࡳ㸧㹛

๪స⏝ࡢ✀㢮 ౛ᩘ

Infusion reaction 312/674㸦46.3%㸧

⾑ᑠᯈῶᑡ⑕* 112/674㸦16.6%㸧

⑂ປ* 92/674㸦13.6%㸧 တႿ* 82/674㸦12.2%㸧

࿧྾ᅔ㞴* 79/674㸦11.7%㸧 ዲ୰⌫ῶᑡ⑕ 73/674㸦10.8%㸧

ୖẼ㐨ឤᰁ* 73/674㸦10.8%㸧 ᝏᚰ* 63/674㸦9.3%㸧

ୗ⑩* 54/674㸦8.0%㸧

㈋⾑* 53/674㸦7.9%㸧

Ⓨ⇕* 45/674㸦6.7%㸧

჎ྤ* 45/674㸦6.7%㸧

*Infusion reactionࢆྵࡴ

఍♫ ࣖࣥࢭࣥࣇ࢓࣮࣐ᰴᘧ఍♫ 〇๣㸸〇㐀

ࢲࣛࢶ࣒࣐ࣈ 1.10 ẘ⸆࣭๻⸆➼ࡢᣦᐃᑂᰝ㈨ᩱࡢࡲ࡜ࡵ

ู⣬

࢔࣑ࣀ㓟㓄ิཬࡧࢪࢫࣝࣇ࢕ࢻ⤖ྜ㸸 L㙐

H㙐

H㙐E1㸸㒊ศⓗࣆࣟࢢࣝࢱ࣑ࣥ㓟㸹H㙐N302㸸⢾㙐⤖ྜ㸹H㙐K452㸸㒊ศⓗࣉࣟࢭࢩࣥࢢ㸹 L㙐C214㸫H㙐C225㸪H㙐C231㸫H㙐C231㸪H㙐C234㸫H㙐C234㸸ࢪࢫࣝࣇ࢕ࢻ⤖ྜ

ࢲࣛࢶ࣒࣐ࣈ 1.10 ẘ⸆࣭๻⸆➼ࡢᣦᐃᑂᰝ㈨ᩱࡢࡲ࡜ࡵ

୺࡞⢾㙐ࡢ᥎ᐃᵓ㐀㸸

Fuc GlcNAc Man

Gal 0-2 Man GlcNAc GlcNAc GlcNAc Man

㸦ู⣬㸧

ู⣬ᵝᘧ㸯

⏕≀⏤᮶་⸆ရཪࡣ≉ᐃ⏕≀⏤᮶་⸆ရࡢᣦᐃ㈨ᩱࡢࡲ࡜ࡵ

୍⯡ྡ㸸 ࢲࣛࢶ࣒࣐ࣈ㸦㑇ఏᏊ⤌᥮࠼㸧

㈍኎ྡ㸸 ࢲࣛࢨࣞࢵࢡࢫ®Ⅼ⁲㟼ὀ100 mg㸪ྠ400 mg

⏦ㄳ⪅㸸 ࣖࣥࢭࣥࣇ࢓࣮࣐ᰴᘧ఍♫

ຠ⬟࣭ຠᯝ㸸 ෌Ⓨཪࡣ㞴἞ᛶࡢከⓎᛶ㦵㧊⭘

⏝ἲ࣭⏝㔞㸸

㏻ᖖࠊᡂே࡟ࡣࢲࣛࢶ࣒࣐ࣈ㸦㑇ఏᏊ⤌᥮࠼㸧࡜ࡋ࡚ࠊ1 ᅇ 16 mg/kg ࢆ௨ୗࡢᢞ୚㛫㝸࡛Ⅼ⁲㟼ὀࡍࡿࠋ

ࣞࢼࣜࢻ࣑ࢻཬࡧࢹ࢟ࢧ࣓ࢱࢰࣥే⏝ࡢሙྜ㸸

1 㐌㛫㛫㝸㸦1㹼8 㐌┠㸧ࠊ2㐌㛫㛫㝸㸦9㹼24 㐌┠㸧ཬࡧ4㐌 㛫㛫㝸㸦25㐌┠௨㝆㸧

࣎ࣝࢸࢰ࣑ࣈཬࡧࢹ࢟ࢧ࣓ࢱࢰࣥే⏝ࡢሙྜ㸸

1 㐌㛫㛫㝸㸦1㹼9 㐌┠㸧ࠊ3 㐌㛫㛫㝸㸦10㹼24 㐌┠㸧ཬࡧ4 㐌㛫㛫㝸㸦25 㐌┠௨㝆㸧

⏕≀⏤᮶ཎᮦᩱࡢ౑⏝ࡢ᭷↓ ڦ౑⏝Ѝ ౑⏝ࡋ࡚࠸ࡿሙྜࡣ௨ୗࡢḍࢆグධ ڧ୙౑⏝

౑⏝ࡋࡓ⏕≀⏤᮶ཎᮦᩱ

ڧே⏤᮶⣽⬊࣭⤌⧊ࠊڧே⏤᮶ᡂศ㸦⾑ᾮࠊᒀࠊࡑࡢ௚㸧ࠊ ڦື≀⏤᮶⣽⬊࣭⤌⧊ࠊڧື≀⏤᮶ᡂศ㸦⾑ᾮࠊࡑࡢ௚㸧 ཎᮦᩱྡ㸹ࢳࣕ࢖ࢽ࣮ࢬࣁ࣒ࢫࢱ࣮༸ᕢ⣽⬊

⏕≀⏤᮶ཎᩱࡢ౑⏝┠ⓗ ڦᐟ୺⣽⬊ࠊڧᇵᆅῧຍ≀ࠊڧࡑࡢ௚ࡢ〇㐀ཎᮦᩱࠊ ڧ〇๣ῧຍ≀ࠊڧࡑࡢ௚㸦 㸧

ཎᮦᩱࡢ⏤᮶࡜࡞ࡿே࣭ື≀ࡢࢫ

ࢡ࣮ࣜࢽࣥࢢ࣭⟶⌮ࡢෆᐜ㸸 ྜ⮴ࡍࡿᇶ‽ࡢ࿌♧࣭㏻▱➼ࡀ࠶ࢀࡤᘬ⏝㸦 㸧 ᙜヱ⏕≀⏤᮶ཎᮦᩱ୙ά໬ฎ⌮➼

ࡢෆᐜ㸸 ู⣬ᵝᘧ2ཧ↷

࢘࢖ࣝࢫࢡࣜ࢔ࣛࣥࢫヨ㦂⤖ᯝࡢ

ᴫせ㸸 ูῧ2ཧ↷

〇㐀ᕤ⛬ࡢᴫせ㸦ࣇ࣮ࣟࢳ࣮ࣕ

ࢺ㸧㸸

㸦୙ά໬ฎ⌮࡟ࡣୗ⥺ࢆ௜ࡋࠊฎ

⌮᮲௳ࢆලయⓗ࡟グ㍕㸧

ูῧ1ཧ↷

ู⣬ᵝᘧ㸰

౑⏝ࡋࡓ⏕≀⏤᮶ཎᩱཪࡣᮦᩱࡢ

ྡ⛠ὀ㸯㸧㸪ὀ㸰㸧 ࢳࣕ࢖ࢽ࣮ࢬࣁ࣒ࢫࢱ࣮༸ᕢ㸦CHO㸧⣽⬊

౑⏝ࡋࡓ⏕≀⏤᮶ཎᩱཪࡣᮦᩱࡢ ศ㢮

ڧே⾑ᾮ⏤᮶ᡂศࠊڧே⣽⬊⤌⧊ࠊڧேᒀ⏤᮶ᡂศࠊڧே⏤

᮶ᡂศ㸦⾑ᾮࠊ⣽⬊⤌⧊ཪࡣᒀࢆ㝖ࡃࡶࡢ㸧ࠊڧ཯ⱄື≀⏤

᮶ᡂศࠊڦື≀⣽⬊⤌⧊ࠊ

ڧື≀⏤᮶ᡂศࠊڧࡑࡢ௚㸦 㸧

⏕≀⏤᮶ཎᩱཪࡣᮦᩱࡢ౑⏝┠ⓗ

ڧ〇๣᭷ຠᡂศࠊڦᐟ୺⣽⬊ࠊڧᇵᆅῧຍ≀ࠊ

ڧࡑࡢ௚ࡢ〇㐀ཎᩱཪࡣᮦᩱ㸦 㸧ࠊڧ〇๣ῧຍ≀ࠊ ڧࡑࡢ௚㸦 㸧

⏕≀⏤᮶ཎᩱཪࡣᮦᩱࡢ⏤᮶࡜࡞

ࡿே࣭ື≀ࡢࢫࢡ࣮ࣜࢽࣥࢢ࣭⟶

⌮ࡢෆᐜὀ㸱㸧

ᐟ୺⣽⬊㸦CHO⣽⬊㸧ࢆ⏝࠸࡚ࢲࣛࢶ࣒࣐ࣈ⏘⏕⣽⬊ᰴ

ࢆస〇ࡋ㸪MCBཬࡧWCBࢆᵓ⠏ࡋࡓࠋMCBཬࡧ WCBࡣ㸪ICH Q5AཬࡧQ5D࡟ᚑࡗ࡚⣧ᗘヨ㦂ࢆ⾜࠸㸪࠸ࡎ

ࢀࡶ⣽⳦㸪┿⳦㸪࣐࢖ࢥࣉࣛࢬ࣐ཬࡧ࢘ࢩཪࡣእ᮶ᛶ࢘࢖ࣝ

ࢫࡢΰධࡣㄆࡵࡽࢀ࡞࠿ࡗࡓࠋ

⏕≀⏤᮶ཎᩱཪࡣᮦᩱࡢ࢘࢖ࣝࢫ

➼ࡢ୙ά໬ཬࡧ㝖ཤฎ⌮➼ࡢෆᐜ

ὀ㸲㸧

ᐇ᪋ࡏࡎ

〇㐀ᕤ⛬ࡢᴫせ㸦ࣇ࣮ࣟࢳ࣮ࣕ

ࢺ㸧ὀ㸳㸧ὀ㸴㸧

㸦୙ά໬ཬࡧ㝖ཤฎ⌮࡟ࡣୗ⥺ࢆ

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〇㐀ᕤ⛬࡟࠾ࡅࡿ࢘࢖ࣝࢫࢡࣜ࢔ࣛࣥࢫ⬟ࡣ㸪ICH Q5A࡟ᚑ࠸࢘࢖ࣝࢫࢡࣜ࢔ࣛࣥࢫヨ㦂ࢆᐇ

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࢕࣮㸦Stage 4㸧ᕤ⛬㸪࢘࢖ࣝࢫ୙ά໬ཬࡧ 㸦Stage 5a㸧ᕤ⛬㸪㝜࢖࢜ࣥ஺᥮ࢡ࣐ࣟࢺࢢࣛࣇ

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⾲ 1

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⾲ 1 ࢘࢖ࣝࢫࢡࣜ࢔ࣛࣥࢫᣦᩘ

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