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Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment

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¡Ø º» »óǰÀº ¿µ¹® ÀÚ·á·Î Çѱ۰ú ¿µ¹® ¸ñÂ÷¿¡ ºÒÀÏÄ¡ÇÏ´Â ³»¿ëÀÌ ÀÖÀ» °æ¿ì ¿µ¹®À» ¿ì¼±ÇÕ´Ï´Ù. Á¤È®ÇÑ °ËÅ並 À§ÇØ ¿µ¹® ¸ñÂ÷¸¦ Âü°íÇØÁֽñ⠹ٶø´Ï´Ù.

¼¼°èÀÇ ¹ßÀÛ¼º¾ß°£Ç÷»ö´¢Áõ(PNH) Ä¡·á ½ÃÀåÀº 2030³â±îÁö 84¾ï ´Þ·¯¿¡ µµ´Þ

2023³â¿¡ 41¾ï ´Þ·¯·Î ÃßÁ¤µÇ´Â ¼¼°èÀÇ ¹ßÀÛ¼º¾ß°£Ç÷»ö´¢Áõ(PNH) Ä¡·á ½ÃÀåÀº 2023-2030³âÀÇ ºÐ¼® ±â°£¿¡ CAGR 10.7%·Î ¼ºÀåÇϸç, 2030³â¿¡´Â 84¾ï ´Þ·¯¿¡ ´ÞÇÒ °ÍÀ¸·Î ¿¹ÃøµË´Ï´Ù. º» ¸®Æ÷Æ®¿¡¼­ ºÐ¼®ÇÑ ºÎ¹®ÀÇ ÇϳªÀÎ Åõ¾à Ä¡·á´Â CAGR 9.0%¸¦ ±â·ÏÇϸç, ºÐ¼® ±â°£ Á¾·á½Ã¿¡´Â 55¾ï ´Þ·¯¿¡ ´ÞÇÒ °ÍÀ¸·Î ¿¹ÃøµË´Ï´Ù. ±âŸ Ä¡·á ºÐ¾ßÀÇ ¼ºÀå·üÀº ºÐ¼® ±â°£¿¡ CAGR 14.4%·Î ÃßÁ¤µË´Ï´Ù.

¹Ì±¹ ½ÃÀåÀº 11¾ï ´Þ·¯·Î ÃßÁ¤, Áß±¹Àº CAGR 10.1%·Î ¼ºÀå ¿¹Ãø

¹Ì±¹ÀÇ ¹ßÀÛ¼º¾ß°£Ç÷»ö´¢Áõ(PNH) Ä¡·á ½ÃÀåÀº 2023³â¿¡ 11¾ï ´Þ·¯·Î ÃßÁ¤µË´Ï´Ù. ¼¼°è 2À§ÀÇ °æÁ¦´ë±¹ÀÎ Áß±¹Àº 2030³â±îÁö 13¾ï ´Þ·¯ÀÇ ½ÃÀå ±Ô¸ð¿¡ ´ÞÇÒ °ÍÀ¸·Î ¿¹ÃøµÇ¸ç, ºÐ¼® ±â°£ÀÎ 2023-2030³âÀÇ CAGRÀº 10.1%ÀÔ´Ï´Ù. ±âŸ ÁÖ¸ñÇØ¾ß ÇÒ Áö¿ªº° ½ÃÀåÀ¸·Î´Â ÀϺ»°ú ij³ª´Ù°¡ ÀÖÀ¸¸ç, ºÐ¼® ±â°£ Áß CAGRÀº °¢°¢ 9.1%¿Í 8.9%·Î ¿¹ÃøµË´Ï´Ù. À¯·´¿¡¼­´Â µ¶ÀÏÀÌ CAGR 7.8%·Î ¼ºÀåÇÒ °ÍÀ¸·Î ¿¹ÃøµË´Ï´Ù.

¼¼°èÀÇ ¹ßÀÛ¼º¾ß°£Ç÷»ö´¢Áõ(PNH) Ä¡·á ½ÃÀå - ÁÖ¿ä µ¿Çâ°ú ÃËÁø¿äÀÎ Á¤¸®

¹ßÀÛ¼º¾ß°£Ç÷»ö´¢Áõ(PNH)ÀÇ È¿°úÀû Ä¡·á°¡ ÇʼöÀûÀÎ ÀÌÀ¯´Â?

¹ßÀÛ¼º¾ß°£Ç÷»ö´¢Áõ(PNH)ÀÇ È¿°úÀûÀÎ Ä¡·á´Â ÀÌ Èñ±ÍÇÑ Ç÷¾× Áúȯ¿¡ ¼ö¹ÝµÇ´Â ½É°¢ÇÏ°í »ý¸íÀ» À§ÇùÇÏ´Â ÇÕº´ÁõÀ¸·Î ÀÎÇØ ÇʼöÀûÀ̸ç, PNH´Â ÀûÇ÷±¸°¡ Á¶±â¿¡ ¶³¾îÁ® ³ª°¡ ¿ëÇ÷¼º ºóÇ÷°ú Ç÷ÀüÁõÀ» À¯¹ßÇÏ°í ½ÉÇÒ °æ¿ì Àå±â ¼Õ»ó°ú »ç¸Á¿¡ À̸£°Ô ÇÕ´Ï´Ù. ÀÌ ÁúȯÀº ¸é¿ª ¸Å°³ ÀûÇ÷±¸ ÆÄ±«¸¦ µ¿¹ÝÇϹǷΠȯÀÚ´Â Ç×»ó ÇÇ·Î, º¹Åë, È£Èí°ï¶õ, Ç÷Àü°ú °°Àº ¼è¾à Áõ»óÀÇ À§Çè¿¡ ³ëÃâµÇ¾î ÀÖ½À´Ï´Ù. Ä¡·áÇÏÁö ¾ÊÀ¸¸é ÀÌ·¯ÇÑ ÇÕº´ÁõÀº ȯÀÚÀÇ »îÀÇ Áú¿¡ ½É°¢ÇÑ ¿µÇâÀ» ¹ÌÄ¡°í ³úÁ¹ÁßÀ̳ª Àå±â ºÎÀü°ú °°Àº »ý¸íÀ» À§ÇùÇÏ´Â »óȲÀ» ÃÊ·¡ÇÒ ¼ö ÀÖÀ¸¸ç, PNHÀÇ Ä¡·á´Â ¿ëÇ÷ ¿¹¹æ, Ç÷Àü »ç°Ç °¨¼Ò ¹× Áõ»ó °ü¸®¿¡ ÁßÁ¡À» µÎ¾î ȯÀÚÀÇ °á°ú¸¦ °³¼±ÇÏ´Â µ¥ ÃÊÁ¡À» ¸ÂÃß¾ú½À´Ï´Ù.

PNHÀÇ Ä¡·á´Â ÀϹÝÀûÀ¸·Î ¸é¿ª°è¸¦ Ç¥ÀûÀ¸·Î ÇÏ´Â Ä¡·á, ƯÈ÷ ¸é¿ª°è°¡ ÀûÇ÷±¸¸¦ °ø°ÝÇÏ´Â °ÍÀ» ¸·´Â º¸Ã¼ ¾ïÁ¦Á¦¸¦ »ç¿ëÇÕ´Ï´Ù. ¿¡Å©¸®ÁÖ¸¿(¼Ö¸®¸®½º)À̳ª ¶óºê¸±ÁÖ¸¿(¿ïÆ®¶ó¸¶ºê)°ú °°Àº º¸Ã¼ ¾ïÁ¦Á¦ÀÇ µµÀÔÀº ¿ëÇ÷·üÀ» ³·Ãß°í Ç÷ÀüÁõ À§ÇèÀ» °¨¼Ò½ÃÅ´À¸·Î½á PNH °ü¸®¿¡ º¯È­¸¦ °¡Á®¿ÔÀ¸¸ç, ȯÀڵ鿡°Ô ´õ ±ä ¼ö¸í°ú ´õ ³ªÀº »îÀÇ ÁúÀ» Á¦°øÇß½À´Ï´Ù. Ç÷ÀüÁõ, ƯÈ÷ Á¤¸ÆÇ÷ÀüÁõÀÇ À§ÇèÀÌ ³ôÀº PNH¿¡¼­ º¸Ã¼ ¾ïÁ¦Á¦´Â ÀÌ·¯ÇÑ ÇÕº´ÁõÀ» ÁÙÀ̰í ȯÀÚ°¡ º¸´Ù ¾ÈÁ¤ÀûÀÎ »îÀ» ¿µÀ§ÇÒ ¼ö ÀÖµµ·Ï ÇÏ´Â µ¥ ¸Å¿ì Áß¿äÇÑ ¿ªÇÒÀ» ÇÕ´Ï´Ù. Áß¿äÇÕ´Ï´Ù.

º¸Ã¼ ¾ïÁ¦Á¦ ¿Ü¿¡µµ ºóÇ÷À» °ü¸®Çϱâ À§ÇÑ ¼öÇ÷°ú Ç÷Àü Çü¼ºÀ» ¿¹¹æÇϱâ À§ÇÑ ¾à¼öó¸®¿Í °°Àº ÁöÁö¿ä¹ýÀÌ PNHÀÇ °ü¸®¿¡ Áß¿äÇÑ ¿ªÇÒÀ» ÇÕ´Ï´Ù. ¼öÇ÷Àº Çì¸ð±Û·Îºó ³óµµ¸¦ À¯ÁöÇϰí ÇǷΰ¨, ¼è¾à°¨ µî ºóÇ÷ °ü·Ã Áõ»ó¿¡ ´ëóÇÏ´Â µ¥ µµ¿òÀÌ µÇ¸ç, Ç×ÀÀ°íÁ¦´Â Ç÷Àü À§ÇèÀ» °¨¼Ò½ÃŰ´Â µ¥ µµ¿òÀÌ µË´Ï´Ù. ´Ù°¢ÀûÀÎ Á¢±ÙÀÌ ÇÊ¿äÇϸç, È¿°úÀûÀÎ Ä¡·á´Â ȯÀÚ¸¦ ¾ÈÁ¤½Ã۰í, ÇÕº´ÁõÀ» ¿¹¹æÇϸç, ±Ã±ØÀûÀ¸·Î PNH ȯÀÚÀÇ »îÀÇ ÁúÀ» Çâ»ó½ÃŰ´Â µ¥ ÇʼöÀûÀÔ´Ï´Ù.

±â¼úÀû, ¾à¸®ÇÐÀûÀÎ ¹ßÀüÀº PNH Ä¡·á¸¦ ¾î¶»°Ô º¯È­½Ãų °ÍÀΰ¡?

±â¼úÀû, ¾à¸®ÇÐÀûÀÎ ¹ßÀüÀº ´õ ¿À·¡ Áö¼ÓµÇ´Â È¿°ú, °³¼±µÈ ¾ÈÀü¼º, ȯÀÚ ÆíÀǼºÀ» °®Ãá º¸´Ù È¿°úÀûÀÎ Ä¡·á¹ýÀ» Á¦°øÇÔÀ¸·Î½á PNH Ä¡·á¸¦ º¯È­½Ã۰í ÀÖÀ¸¸ç, PNH Ä¡·áÀÇ °¡Àå Áß¿äÇÑ Çõ½Å Áß Çϳª´Â ¿¡Å¬¸®ÁÖ¸¿°ú °°Àº Ãʱâ Ä¡·áÁ¦º¸´Ù Åõ¿© Ƚ¼ö¸¦ ÁÙÀÏ ¼ö ÀÖ´Â ¶óºê¸®ÁÖ¸¿°ú °°Àº Àå±â Áö¼ÓÇü º¸Ã¼ ¾ïÁ¦Á¦ÀÇ °³¹ßÀÔ´Ï´Ù. Åõ¿© Ƚ¼ö¸¦ ÁÙÀÏ ¼ö ÀÖ´Â ¶óºê¸±ÁÖ¸¿°ú °°Àº Àå½Ã°£ ÀÛ¿ëÇÏ´Â º¸Ã¼ ¾ïÁ¦Á¦ÀÇ °³¹ßÀÔ´Ï´Ù. ¶óºê¸±ÁÖ¸¿Àº Åõ¾à °£°ÝÀ» 2ÁÖ °£°Ý¿¡¼­ 8ÁÖ °£°ÝÀ¸·Î ¿¬ÀåÇÏ¿© ȯÀÚ¿¡°Ô ÆíÀǼºÀ» Á¦°øÇϰí Ä¡·á ¼øÀÀµµ¸¦ Çâ»ó½Ãŵ´Ï´Ù. ÀÌ·¯ÇÑ Åõ¿© ÀÏÁ¤ ¿¬ÀåÀº ÀæÀº º´¿ø ¹æ¹®ÀÌ ¾î·Æ°í ½Ã°£ÀÌ ¸¹ÀÌ °É¸®´Â ȯÀڵ鿡°Ô ƯÈ÷ À¯¿ëÇÕ´Ï´Ù. ÀæÀº Åõ¾àÀÇ ºÎ´ãÀ» ÁÙÀÓÀ¸·Î½á Àå½Ã°£ ÀÛ¿ëÇÏ´Â º¸Ã¼ ¾ïÁ¦Á¦´Â º¸´Ù °ü¸®Çϱ⠽¬¿î Ä¡·á ·çƾÀ» Áö¿øÇÏ¿© º¸´Ù ȯÀÚ Ä£È­ÀûÀÎ Ä¡·á °æÇèÀ» Á¦°øÇÕ´Ï´Ù.

C3 ¾ïÁ¦Á¦ ¹× ´ëü °æ·Î ¾ïÁ¦Á¦¿Í °°Àº ´ëü °æ·Î¸¦ Ç¥ÀûÀ¸·Î ÇÏ´Â »õ·Î¿î º¸Ã¼ ¾ïÁ¦Á¦´Â ±âÁ¸ Ä¡·áÁ¦¿¡ ³»¼ºÀ» º¸À̴ ȯÀڵ鿡°Ô À¯¸ÁÇÑ ´ë¾ÈÀ¸·Î ¶°¿À¸£°í ÀÖ½À´Ï´Ù. ¿¹¸¦ µé¾î Áö¸£ÄÚÇöõ°ú Æä±×¼¼Å¸ÄÚÇöõÀº º¸Ã¼ °æ·ÎÀÇ Ãʱ⠴ܰ迡¼­ º¸Ã¼ °æ·Î¸¦ Â÷´ÜÇϵµ·Ï ¼³°èµÇ¾î ±âÁ¸ C5 ¾ïÁ¦Á¦¿¡ ºÎºÐÀû ¶Ç´Â ºÒÃæºÐÇÑ ¹ÝÀÀÀ» º¸À̴ ȯÀڵ鿡°Ô º¸´Ù ±¤¹üÀ§ÇÑ ¾ïÁ¦ È¿°ú°¡ ÀÖÀ¸¸ç, µµ¿òÀÌ µÉ ¼ö ÀÖ½À´Ï´Ù. ÀÌ·¯ÇÑ ´ëü °æ·Î ¾ïÁ¦Á¦´Â ¿ëÇ÷À» º¸´Ù È¿°úÀûÀ¸·Î ¿¹¹æÇÏ°í ¼öÇ÷ÀÇ Çʿ伺À» ÁÙÀÌ´Â °ÍÀ» ¸ñÇ¥·Î Çϰí ÀÖÀ¸¸ç, ÇöÀç Ä¡·á¹ýÀ¸·Î ¿ÏÀüÇÑ °üÇØ¿¡ µµ´ÞÇÏÁö ¸øÇÑ È¯Àڵ鿡°Ô Èñ¸ÁÀ» ÁÖ°í ÀÖ½À´Ï´Ù. ÀÌ·¯ÇÑ »õ·Î¿î ¾ïÁ¦Á¦°¡ ÀÓ»ó½ÃÇè ´Ü°è¿¡ µµ´ÞÇϸé PNH Ä¡·á ¿É¼ÇÀ» È®ÀåÇÏ°íº¸´Ù Á¾ÇÕÀûÀÎ º¸Ã¼ ¾ïÁ¦°¡ ÇÊ¿äÇÑ È¯ÀÚ¿¡°Ô ÇØ°áÃ¥À» Á¦°øÇÒ ¼ö ÀÖ½À´Ï´Ù.

À¯ÀüÀÚ Ä¡·á¿Í CRISPR¿¡ ±â¹ÝÇÑ ¿¬±¸µµ PNHÀÇ Àå±âÀûÀÎ ÇØ°áÃ¥ÀÌ µÉ ¼ö ÀÖ´Â ¿¬±¸·Î ¹ßÀüÇϰí ÀÖ½À´Ï´Ù. ¾ÆÁ÷ ¿¬±¸ Ãʱ⠴ܰ迡 ÀÖÁö¸¸, À¯ÀüÀÚ Ä¡·á´Â ÀÏȸ¼º Ä¡·á·Î ¿ÏÄ¡Çϰųª Áö¼ÓÀûÀÎ ¾à¹°ÀÇ Çʿ伺À» Å©°Ô ÁÙÀÌ´Â °ÍÀ» ¸ñÇ¥·Î Çϰí ÀÖÀ¸¸ç, PNH¸¦ À¯¹ßÇÏ´Â À¯ÀüÀÚ µ¹¿¬º¯À̸¦ Á÷Á¢ Ç¥ÀûÀ¸·Î »ïÀ½À¸·Î½á À¯ÀüÀÚ Ä¡·á´Â ȯÀÚ¿¡°Ô ¿µ±¸ÀûÀÎ ÇØ°áÃ¥À» Á¦°øÇÒ ¼ö ÀÖ½À´Ï´Ù. ÀÌ·¯ÇÑ ±â¼úÀº ¾ÆÁ÷ °³¹ß ÁßÀÌÁö¸¸, Àå±âÀûÀÎ È¿°ú¸¦ ±â´ëÇÒ ¼ö ÀÖÀ¸¹Ç·Î Èï¹Ì·Î¿î ¿¬±¸ ºÐ¾ßÀÔ´Ï´Ù. À¯ÀüÀÚ Ä¡·á°¡ ¹ßÀüÇϸé PNH Ä¡·á¿¡ Çõ¸íÀ» ÀÏÀ¸Å°°í ȯÀڵ鿡°Ô Æò»ý ¾à¹°¿¡ ÀÇÁ¸ÇÏÁö ¾Ê´Â ¹Ì·¡¿¡ ´ëÇÑ Èñ¸ÁÀ» °¡Á®´Ù ÁÙ ¼ö ÀÖ½À´Ï´Ù.

PNH Ä¡·á´Â ȯÀÚ¿Í ÀÇ·á ½Ã½ºÅÛ¿¡ ¾î¶² ÀÌÁ¡ÀÌ Àִ°¡?

PNH Ä¡·á´Â Áõ»ó °ü¸®, »ý¸íÀ» À§ÇùÇÏ´Â ÇÕº´Áõ À§Çè °¨¼Ò, »îÀÇ Áú °³¼± µî ȯÀڵ鿡°Ô Å« ÀÌÁ¡À» Á¦°øÇϸç, ÀÇ·á ½Ã½ºÅÛ¿¡´Â ÀÔ¿ø °¨¼Ò, ȯÀÚ ¿¹ÈÄ °³¼± µî ´Ù¾çÇÑ ÀÌÁ¡À» Á¦°øÇÕ´Ï´Ù. ȯÀÚÀÇ °æ¿ì, ¿¡Å©¸®ÁÖ¸¿°ú ¶óºê¸±ÁÖ¸¿°ú °°Àº º¸Ã¼ ¾ïÁ¦Á¦´Â ¿ëÇ÷À» °¨¼Ò½Ã۰í ÇÇ·Î, ¼è¾à, È£Èí°ï¶õ°ú °°Àº Áõ»óÀ» ¿ÏÈ­ÇÔÀ¸·Î½á PNH °ü¸®¿¡ Å« º¯È­¸¦ °¡Á®¿Ô½À´Ï´Ù. ¿ëÇ÷À» °¨¼Ò½ÃÅ´À¸·Î½á ÀÌ·¯ÇÑ Ä¡·á´Â ȯÀÚ°¡ ´õ °Ç°­ÇÑ Çì¸ð±Û·Îºó ³óµµ¸¦ À¯ÁöÇϰí ÀÏ»ó Ȱµ¿¿¡ Âü¿©ÇÏ´Â ´É·ÂÀ» Çâ»ó½ÃŰ´Â µ¥ µµ¿òÀ̵Ǹç, PNH ȯÀÚÀÇ °æ¿ì È¿°úÀûÀÎ Áõ»ó °ü¸®´Â µ¶¸³¼ºÀ» À¯ÁöÇϰí ÀÏ»ó »ýȰ¿¡ ¹ÌÄ¡´Â ¿µÇâÀ» ÁÙÀ̱â À§ÇØ ¸Å¿ì Áß¿äÇÕ´Ï´Ù.

º¸Ã¼ ¾ïÁ¦Á¦´Â ¶ÇÇÑ PNH ȯÀÚÀÇ ÁÖ¿ä »ç¸Á ¿øÀÎ Áß ÇϳªÀÎ Ç÷ÀüÁõÀÇ À§ÇèÀ» ÁÙÀÌ´Â µ¥ Áß¿äÇÑ ¿ªÇÒÀ» ÇÕ´Ï´Ù. Ç÷Àü Çü¼ºÀ» ¿¹¹æÇÔÀ¸·Î½á ÀÌ·¯ÇÑ Ä¡·á´Â ³úÁ¹Áß, Æó»öÀüÁõ, Àå±â ¼Õ»ó°ú °°Àº ½É°¢ÇÑ ÇÕº´ÁõÀÇ °¡´É¼ºÀ» °¨¼Ò½Ãŵ´Ï´Ù. Ä¡·áµÇÁö ¾ÊÀº Ç÷ÀüÁõÀº Ä¡¸íÀûÀÎ °á°ú¸¦ ÃÊ·¡ÇÒ ¼ö ÀÖÀ¸¹Ç·Î ÀÌ·¯ÇÑ Ç÷Àü »ç°ÇÀÇ °¨¼Ò´Â ȯÀÚÀÇ Àå±â »ýÁ¸¿¡ ƯÈ÷ À¯ÀÍÇÕ´Ï´Ù. µû¶ó¼­ º¸Ã¼ ¾ïÁ¦Á¦¸¦ ÅëÇÑ È¿°úÀûÀÎ Ä¡·á´Â ÀÀ±Þ °³ÀÔÀÇ Çʿ伺À» ÁÙÀ̰í ȯÀÚÀÇ °Ç°­ »óŸ¦ ¾ÈÁ¤È­Çϸç Àå±â ¿¹Èĸ¦ °³¼±ÇÕ´Ï´Ù.

º¸°ÇÀÇ·á ½Ã½ºÅÛ¿¡¼­ PNH Ä¡·á´Â ÀæÀº ÀÔ¿ø ¹× ÀÀ±Þ Ä¡·áÀÇ Çʿ伺À» ÁÙ¿© ÇÕº´Áõ °ü¸®¿Í °ü·ÃµÈ Àüü ÀÇ·á ºñ¿ëÀ» Àý°¨Çϰí, ¶óºÒ¸®ÁÖ¸¿°ú °°Àº Ä¡·áÁ¦´Â Åõ¿© Ƚ¼ö¸¦ ÁÙ¿© º´¿ø ¹æ¹®°ú Á¤¸ÆÁÖ»ç Ƚ¼ö¸¦ ÁÙ¿© ÀÇ·á ÀÚ¿øÀ» È®º¸ÇÏ°í ºñ¿ë È¿À²À» ³ôÀÔ´Ï´Ù. ºñ¿ë È¿À²¼ºÀÌ Çâ»óµË´Ï´Ù. È¿°úÀûÀÌ°í ¿À·¡ Áö¼ÓµÇ´Â ¼Ö·ç¼ÇÀ» Á¦°øÇÔÀ¸·Î½á ÀÇ·á ½Ã½ºÅÛÀº ±Þ¼º±â °³ÀÔº¸´Ù ¿¹¹æ ÀÇ·á¿¡ ÁýÁßÇÒ ¼ö ÀÖÀ¸¸ç, ȯÀÚÀÇ ¿¹Èĸ¦ °³¼±ÇÒ ¼ö ÀÖ½À´Ï´Ù. ¶ÇÇÑ »õ·Î¿î Ä¡·á¹ýÀÌ PNH °ü¸®ÀÇ È¿°ú¸¦ °³¼±ÇÔ¿¡ µû¶ó ȯÀÚ´Â ¼öÇ÷À̳ª Ç×ÀÀ°íÁ¦¿Í °°Àº ÁöÁö¿ä¹ý¿¡ ´ú ÀÇÁ¸ÇÏ°Ô µÇ¾î ÀÇ·á ºñ¿ë°ú ÀÚ¿ø ¹èºÐÀ» ´õ¿í Àý°¨ÇÒ ¼ö ÀÖ½À´Ï´Ù.

¶ÇÇÑ ÀÓ»ó½ÃÇè¿¡¼­ °æ±¸¿ë º¸Ã¼ ¾ïÁ¦Á¦°¡ °³¹ßµÇ¸é, ƯÈ÷ ¼ö¾× ¼¾ÅÍ¿¡ ´ëÇÑ Á¢±Ù¼ºÀÌ Á¦ÇÑµÈ Áö¿ª¿¡¼­ ȯÀÚµéÀÇ Ä¡·á ÆíÀǼº°ú Á¢±Ù¼ºÀ» ´õ¿í Çâ»ó½Ãų ¼ö ÀÖ½À´Ï´Ù. °æ±¸¿ë Ä¡·áÁ¦¸¦ ÅëÇØ ȯÀÚµéÀº Áý¿¡¼­ º´À» °ü¸®ÇÒ ¼ö ÀÖ°Ô µÇ°í, ÀÇ·á±â°üÀ» ÀÚÁÖ ¹æ¹®ÇÏÁö ¾Ê¾Æµµ µË´Ï´Ù. ÀÌ·¯ÇÑ Á¢±Ù¼ºÀº ³óÃÌ Áö¿ªÀ̳ª ÀÇ·á ÇýÅÃÀÌ ºÎÁ·ÇÑ »ç¶÷µé¿¡°Ô´Â À̵¿¿¡ µû¸¥ ¹°·ù ¹× °æÁ¦Àû ºÎ´ã ¾øÀÌ ÇÊ¿äÇÑ Ä¡·á¸¦ ¹ÞÀ» ¼ö ÀÖ´Ù´Â Å« ÀÌÁ¡ÀÌ ÀÖ½À´Ï´Ù. ÀÇ·á½Ã½ºÅÛÀÇ °æ¿ì, °æ±¸¿ë Ä¡·á´Â ¼ö¾×¼¾ÅÍ¿Í º´¿øÀÇ ÀÚ¿ø¿¡ ´ëÇÑ ¼ö¿ä¸¦ ÁÙ¿© È¿À²ÀûÀÎ Ä¡·á Á¦°øÀ» ´õ¿í Áö¿øÇÏ°í ¸ðµç PNH ȯÀÚÀÇ Á¢±Ù¼ºÀ» Çâ»ó½Ãų ¼ö ÀÖ½À´Ï´Ù.

PNH Ä¡·á ½ÃÀåÀÇ ¼ºÀåÀ» °¡¼ÓÇÏ´Â ¿äÀÎÀº ¹«¾ùÀΰ¡?

Èñ±ÍÁúȯ¿¡ ´ëÇÑ ÀÎ½Ä Áõ°¡, Ç¥Àû Ä¡·áÀÇ ¹ßÀü, »õ·Î¿î Ä¡·á¹ý¿¡ ´ëÇÑ È¯ÀÚ Á¢±Ù¼º È®´ë, À¯ÀüÀÚ Ä¡·á ¿¬±¸ÀÇ ºÎ»ó µîÀÌ PNH Ä¡·á ½ÃÀåÀÇ ¼ºÀå µ¿·ÂÀÌ µÇ°í ÀÖ½À´Ï´Ù. ÀÇ·áÁø°ú ȯÀÚµé »çÀÌ¿¡¼­ PNH¿¡ ´ëÇÑ Àνİú ÀÌÇØ°¡ ³ô¾ÆÁü¿¡ µû¶ó Áø´ÜÀ²ÀÌ Çâ»óµÇ°í Á¤È®ÇÑ Ä¡·á¸¦ ´õ »¡¸® ½ÃÀÛÇÒ ¼ö ÀÖ°Ô µÇ¾ú½À´Ï´Ù. ȯÀÚ°¡ ´õ »¡¸® Áø´ÜÀ» ¹ÞÀ¸¸é ÇÕº´ÁõÀ» ÁÙÀ̰í Àå±âÀûÀÎ °á°ú¸¦ °³¼±ÇÒ ¼ö ÀÖ´Â »ý¸íÀ» ±¸ÇÏ´Â Ä¡·á¸¦ ¹ÞÀ» ¼ö ÀÖ½À´Ï´Ù. ¶ÇÇÑ È¯ÀÚ Áö¿ø ´ÜüÀÇ ÀÎ½Ä °³¼± Ȱµ¿Àº PNH ¿¬±¸¿¡ ´ëÇÑ ÅõÀÚ¸¦ ÃËÁøÇÏ¿© ÷´Ü Ä¡·á¹ý °³¹ß°ú Ä¡·á ¿É¼ÇÀÇ È®´ë·Î À̾îÁö°í ÀÖÀ¸¸ç, PNH¿¡ ´ëÇÑ Àνİú Áø´ÜÀÌ Çâ»óµÊ¿¡ µû¶ó È¿°úÀûÀÎ PNH Ä¡·á¿¡ ´ëÇÑ ¼ö¿ä´Â Áö¼ÓÀûÀ¸·Î Áõ°¡Çϰí ÀÖ½À´Ï´Ù.

Ç¥Àû Ä¡·á, ƯÈ÷ º¸Ã¼ ¾ïÁ¦Á¦ÀÇ ¹ßÀüÀº ¿ëÇ÷À» È¿°úÀûÀ¸·Î Á¦¾îÇϰí Ç÷Àü »ç°ÇÀÇ À§ÇèÀ» ÁÙÀÏ ¼ö ÀÖÀ¸¹Ç·Î Å« ÃËÁøÁ¦ ¿ªÇÒÀ» Çϰí ÀÖ½À´Ï´Ù. ¿¡Å¬¸®ÁÖ¸¿°ú ¶óºê¸®ÁÖ¸¿°ú °°Àº C5 ¾ïÁ¦Á¦ÀÇ ¼º°øÀº C3 ¾ïÁ¦Á¦ ¹× ±âŸ º¸Ã¼ °æ·Î ¾ïÁ¦Á¦¸¦ Æ÷ÇÔÇÑ Â÷¼¼´ë Ä¡·áÁ¦ °³¹ßÀÇ ±æÀ» ¿­¾ú½À´Ï´Ù. ÀÌ·¯ÇÑ »õ·Î¿î Ä¡·áÁ¦´Â Ä¡·á ¼±ÅÃÀÇ ÆøÀ» ³ÐÈ÷°í ±âÁ¸ Ä¡·áÁ¦·Î´Â È¿°ú°¡ ÃæºÐÇÏÁö ¾ÊÀº ȯÀÚÀÇ ¹ÌÃæÁ· ¼ö¿ä¸¦ ÃæÁ·½Ãų ¼ö ÀÖ½À´Ï´Ù. Èñ±ÍÁúȯ ¿¬±¸¿¡ ´ëÇÑ °ü½ÉÀÌ ³ô¾ÆÁö¸é¼­ Á¦¾à»çµéÀÌ PNH¿¡ ´ëÇÑ Çõ½ÅÀûÀÎ Ä¡·á¹ý¿¡ ´ëÇÑ ÅõÀÚ¸¦ ´Ã¸®¸é¼­ ½ÃÀå ¼ºÀå¿¡ ´õ¿í ¹ÚÂ÷¸¦ °¡Çϰí ÀÖ½À´Ï´Ù. Á¦¾à»çµéÀÌ »õ·Î¿î Ä¡·á¹ýÀ» Ãß±¸ÇÏ°í º¸Ã¼ ¾ïÁ¦Á¦ÀÇ À¯ÇüÀ» ´Ã¸®¸é¼­ PNH Ä¡·á ½ÃÀåÀº ¼ºÀåÇÒ °ÍÀ¸·Î º¸ÀÔ´Ï´Ù.

±ÔÁ¦ ´ç±¹ÀÇ ½ÂÀΰú º¸Çè Àû¿ë È®´ë¿¡ ÈûÀÔ¾î PNH Ä¡·áÁ¦¿¡ ´ëÇÑ È¯ÀÚ Á¢±Ù¼º È®´ëµµ ½ÃÀå ¼ºÀåÀÇ ¿øµ¿·ÂÀÌ µÇ°í ÀÖÀ¸¸ç, FDA ¹× EMA¿Í °°Àº ±ÔÁ¦±â°üÀº ¸¹Àº PNH Ä¡·áÁ¦¿¡ Èñ±ÍÀǾàǰ(Orphan Drug) ÁöÀ§¸¦ ºÎ¿©ÇÏ¿© ½ÂÀÎ ÀýÂ÷¸¦ °£¼ÒÈ­ÇÏ°í ´Ù¾çÇÑ Áö¿ª¿¡¼­ÀÇ Á¢±Ù¼ºÀ» ³ôÀ̰í ÀÖ½À´Ï´Ù. ½ÂÀÎ ÀýÂ÷¸¦ °£¼ÒÈ­ÇÏ°í ´Ù¾çÇÑ Áö¿ª¿¡¼­ÀÇ Á¢±Ù¼ºÀ» ³ôÀ̰í ÀÖ½À´Ï´Ù. º¸Ã¼ ¾ïÁ¦Á¦¿Í °°Àº °í°¡ÀÇ Ä¡·áÁ¦¿¡ ´ëÇÑ º¸Çè Àû¿ëµµ °³¼±µÇ¾î ÀÌÀü¿¡´Â Ä¡·áºñ¸¦ ÁöºÒÇÒ ¼ö ¾ø¾ú´ø ȯÀÚµéÀÌ Ä¡·áÁ¦¸¦ ´õ ½±°Ô ÀÌ¿ëÇÒ ¼ö ÀÖ°Ô µÇ¾ú½À´Ï´Ù. ¶ÇÇÑ ´õ ÀûÀº Ƚ¼ö·Î Åõ¿©ÇÒ ¼ö ÀÖ´Â Áö¼ÓÇü Ä¡·áÁ¦ÀÇ µµÀÔÀ¸·Î, ƯÈ÷ ¿Üµý Áö¿ªÀ̳ª Ä¡·á Á¢±Ù¼ºÀÌ ³·Àº Áö¿ªÀÇ È¯Àڵ鿡°Ô ´õ Æí¸®ÇÏ°í ½±°Ô Ä¡·á¹ÞÀ» ¼ö ÀÖ°Ô µÇ¾ú½À´Ï´Ù. ´Ù¾çÇÑ Áö¿ª¿¡¼­ Ä¡·á °á°ú°¡ °³¼±µÉ °ÍÀ¸·Î ¿¹»óµË´Ï´Ù.

À¯ÀüÀÚ Ä¡·á ¿¬±¸ÀÇ ÃâÇöÀº Àå±âÀûÀ¸·Î PNH Ä¡·á ½ÃÀåÀ» º¯È­½Ãų ¼ö ÀÖ´Â ÀáÀç·ÂÀ» °¡Áö°í ÀÖÀ¸¸ç, PNH¸¦ À¯¹ßÇÏ´Â ±Ùº»ÀûÀÎ À¯ÀüÀÚ µ¹¿¬º¯À̸¦ ÇØ°áÇÔÀ¸·Î½á À¯ÀüÀÚ Ä¡·á´Â Æò»ý Åõ¾àÀÇ Çʿ伺À» ¾ø¾Ö°í ´Ü ÇÑ ¹øÀÇ Ä¡·á·Î ¿ÏÄ¡ÇÒ ¼ö ÀÖ´Â Ä¡·á¹ýÀ» Á¦°øÇÒ ¼ö ÀÖ´Â ÀáÀç·ÂÀ» °¡Áö°í ÀÖ½À´Ï´Ù. À¯ÀüÀÚ Ä¡·á ¿¬±¸´Â ¾ÆÁ÷ Ãʱ⠴ܰ迡 ÀÖÁö¸¸, PNHÀÇ Áõ»ó°ú ÇÕº´ÁõÀ» Àå±âÀûÀ¸·Î ¿ÏÈ­ÇÒ ¼ö ÀÖ´Ù´Â Á¡¿¡¼­ Å« °ü½ÉÀ» ¹Þ°í ÀÖ½À´Ï´Ù. ±â¾÷ÀÌ PNH Ä¡·á¿¡ Àû¿ëÀ» ¸ð»öÇϸ鼭 À¯ÀüÀÚ Ä¡·á¿Í CRISPR ±â¹Ý ¼Ö·ç¼Ç¿¡ ´ëÇÑ ÅõÀÚ°¡ Áõ°¡Çϰí ÀÖ½À´Ï´Ù. ÀÌ·¯ÇÑ Ä¡·á¹ýÀÌ ¼º°øÇÑ´Ù¸é PNH ½ÃÀå¿¡ Çõ¸íÀ» ÀÏÀ¸ÄÑ È¯ÀÚ¿¡°Ô ¿µ±¸ÀûÀÎ ÇØ°áÃ¥À» Á¦°øÇÏ°í ºñ¿ëÀÌ ¸¹ÀÌ µå´Â Áö¼ÓÀûÀÎ Ä¡·á¿¡ ´ëÇÑ ÀÇÁ¸µµ¸¦ ³·Ãâ ¼ö ÀÖ½À´Ï´Ù. ÀÎÁöµµÀÇ Çâ»ó, Ç¥Àû Ä¡·áÀÇ ¹ßÀü, ȯÀÚ Á¢±Ù¼º Çâ»ó, À¯ÀüÀÚ Ä¡·á ¿¬±¸ µî ÀÌ·¯ÇÑ ¿äÀεéÀÌ °áÇÕÇÏ¿© PNH Ä¡·á ½ÃÀåÀº °­·ÂÇÑ ¼ºÀåÀ» ÀÌ·ç¸ç Èñ±ÍÁúȯ °ü¸® ¹× Çõ½ÅÀû ÇコÄɾîÀÇ Áß¿äÇÑ ºÐ¾ß·Î ÀÚ¸®¸Å±èÇϰí ÀÖ½À´Ï´Ù.

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  • Akari Therapeutics Plc
  • Alexion Pharmaceuticals, Inc.
  • Apellis Pharmaceuticals
  • AstraZeneca Plc
  • CANbridge Pharmaceuticals
  • Chugai Pharmaceutical Co., Ltd.
  • F. Hoffmann-La Roche AG
  • Genentech, Inc.
  • Kira Pharmaceuticals
  • Novartis AG

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KSA 24.11.27

Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market to Reach US$8.4 Billion by 2030

The global market for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment estimated at US$4.1 Billion in the year 2023, is expected to reach US$8.4 Billion by 2030, growing at a CAGR of 10.7% over the analysis period 2023-2030. Medication Treatment, one of the segments analyzed in the report, is expected to record a 9.0% CAGR and reach US$5.5 Billion by the end of the analysis period. Growth in the Other Treatments segment is estimated at 14.4% CAGR over the analysis period.

The U.S. Market is Estimated at US$1.1 Billion While China is Forecast to Grow at 10.1% CAGR

The Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment market in the U.S. is estimated at US$1.1 Billion in the year 2023. China, the world's second largest economy, is forecast to reach a projected market size of US$1.3 Billion by the year 2030 trailing a CAGR of 10.1% over the analysis period 2023-2030. Among the other noteworthy geographic markets are Japan and Canada, each forecast to grow at a CAGR of 9.1% and 8.9% respectively over the analysis period. Within Europe, Germany is forecast to grow at approximately 7.8% CAGR.

Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market - Key Trends & Drivers Summarized

Why Is Effective Treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH) Essential?

Effective treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH) is essential due to the severe and life-threatening complications associated with this rare blood disorder. PNH causes red blood cells to break apart prematurely, leading to hemolytic anemia, thrombosis, and, in severe cases, organ damage and death. Since the disease involves immune-mediated destruction of red blood cells, it places patients at constant risk for debilitating symptoms such as fatigue, abdominal pain, difficulty breathing, and blood clots. Without treatment, these complications can severely impact patients' quality of life and may result in life-threatening events like strokes and organ failure. Treatments for PNH focus on preventing hemolysis, reducing thrombotic events, and managing symptoms to improve patient outcomes.

PNH treatment typically involves therapies that target the immune system, specifically complement inhibitors, which prevent the immune system from attacking red blood cells. The introduction of complement inhibitors like eculizumab (Soliris) and ravulizumab (Ultomiris) has transformed PNH management by reducing the rate of hemolysis and decreasing the risk of thrombosis, providing patients with longer life expectancy and better quality of life. These treatments have become essential for managing the disease and preventing the serious complications associated with uncontrolled hemolysis. Given the high risk of thrombotic events in PNH, especially in veins, complement inhibitors are critical in reducing these complications and allowing patients to lead more stable lives.

In addition to complement inhibitors, supportive care plays a significant role in managing PNH, including blood transfusions to manage anemia and medications to prevent clot formation. Blood transfusions can help maintain hemoglobin levels, addressing anemia-related symptoms like fatigue and weakness, while anticoagulants can reduce the risk of blood clots. Advances in PNH treatment focus on reducing the need for supportive care and improving the efficacy of complement inhibitors. As the disease's complexity and risks require a multifaceted approach, effective treatments are vital for stabilizing patients, preventing complications, and ultimately enhancing the quality of life for those affected by PNH.

How Are Technological and Pharmacological Advancements Transforming PNH Treatment?

Technological and pharmacological advancements are transforming PNH treatment by offering more effective therapies with longer-lasting effects, improved safety, and enhanced patient convenience. One of the most significant innovations in PNH treatment is the development of long-acting complement inhibitors, such as ravulizumab, which allow for less frequent dosing than earlier treatments like eculizumab. Ravulizumab extends the dosing interval from every two weeks to every eight weeks, providing patients with greater convenience and improving adherence to treatment. This extended dosing schedule is especially beneficial for patients who find frequent hospital visits challenging and time-consuming. By reducing the burden of frequent infusions, long-acting complement inhibitors support more manageable treatment routines and provide a more patient-friendly experience.

New complement inhibitors targeting alternative pathways, such as C3 inhibitors and alternative pathway inhibitors, are emerging as promising options for patients who are resistant to existing treatments. Zilucoplan and pegcetacoplan, for example, are designed to block the complement pathway earlier in the cascade, providing broader inhibition that may benefit patients with partial or inadequate responses to traditional C5 inhibitors. These alternative pathway inhibitors aim to prevent hemolysis more effectively and reduce the need for blood transfusions, offering hope for patients who do not achieve full remission with current therapies. As these new inhibitors reach advanced clinical trials, they hold potential to expand PNH treatment options, providing a solution for those who require more comprehensive complement inhibition.

Gene therapy and CRISPR-based research are also advancing as potential long-term solutions for PNH, with the goal of correcting the genetic defect that causes the disease. Although still in early stages of research, gene therapy aims to provide a one-time treatment that can potentially cure or significantly reduce the need for ongoing medication. By directly targeting the genetic mutation that leads to PNH, gene therapy could offer a permanent solution for patients. While these technologies are still under development, their potential for long-lasting efficacy makes them an exciting area of research. As gene therapy advances, it may revolutionize PNH treatment, offering patients hope for a future without lifelong reliance on medication.

What Are the Benefits of PNH Treatments for Patients and Healthcare Systems?

PNH treatments provide significant benefits for patients by managing symptoms, reducing the risk of life-threatening complications, and improving quality of life, while healthcare systems benefit from reduced hospitalization and improved patient outcomes. For patients, complement inhibitors like eculizumab and ravulizumab have transformed PNH management by reducing hemolysis, which alleviates symptoms such as fatigue, weakness, and difficulty breathing. By reducing hemolysis, these treatments help patients maintain healthier hemoglobin levels, enhancing their ability to participate in daily activities. For patients with PNH, effective symptom management is critical for maintaining independence and reducing the impact of the disease on their daily lives.

Complement inhibitors also play a crucial role in reducing the risk of thrombosis, which is one of the leading causes of mortality in PNH patients. By preventing clot formation, these treatments decrease the likelihood of severe complications such as strokes, pulmonary embolisms, and organ damage. This reduction in thrombotic events is particularly beneficial for patients’ long-term survival, as untreated thrombosis can lead to fatal outcomes. Effective treatment with complement inhibitors thus reduces the need for emergency interventions, improving the stability of patients' health and providing a better long-term prognosis.

For healthcare systems, PNH treatments reduce the need for frequent hospitalizations and emergency care, lowering overall healthcare costs associated with managing complications. As treatments like ravulizumab allow for less frequent dosing, they decrease the frequency of hospital visits and infusion sessions, freeing up healthcare resources and improving cost efficiency. By providing effective, long-acting solutions, healthcare systems can focus on preventive care rather than acute interventions, which supports better patient outcomes. Furthermore, as new treatments improve the effectiveness of PNH management, patients are less reliant on supportive care like blood transfusions and anticoagulants, which further reduces healthcare expenses and resource allocation.

Additionally, the development of oral complement inhibitors in clinical trials could offer even greater convenience and access to treatment for patients, especially in regions where access to infusion centers is limited. Oral therapies would allow patients to manage their condition from home, eliminating the need for frequent travel to healthcare facilities. This accessibility can greatly benefit rural or underserved populations, providing them with the necessary treatment without the logistical and financial burden of travel. For healthcare systems, oral therapies would reduce the demand on infusion centers and hospital resources, further supporting efficient care delivery and improving accessibility for all PNH patients.

What Is Fueling the Growth in the PNH Treatment Market?

The growth in the PNH treatment market is driven by increased awareness of rare diseases, advancements in targeted therapies, expanded patient access to new treatments, and the emergence of gene therapy research. With greater awareness and understanding of PNH among healthcare providers and patients, diagnosis rates have improved, leading to earlier and more accurate treatment initiation. As patients are diagnosed sooner, they can begin receiving life-saving treatments that reduce complications and improve long-term outcomes. Awareness efforts, often driven by patient advocacy groups, have also encouraged more investment in PNH research, leading to the development of advanced therapies and expanded treatment options. As awareness and diagnosis improve, the demand for effective PNH treatments continues to grow.

Advancements in targeted therapies, particularly in complement inhibitors, are a major growth driver, as these treatments offer effective control of hemolysis and reduce the risk of thrombotic events. The success of C5 inhibitors like eculizumab and ravulizumab has paved the way for the development of next-generation therapies, including C3 inhibitors and other complement pathway inhibitors. These new therapies are expanding treatment options and addressing unmet needs for patients with incomplete responses to existing treatments. The market’s growth is further supported by increasing interest in rare disease research, which has encouraged pharmaceutical companies to invest in innovative treatments for PNH. As companies pursue novel therapies and expand the range of complement inhibitors, the PNH treatment market is set to grow.

Expanded patient access to PNH treatments, facilitated by regulatory approvals and broader insurance coverage, is also driving market growth. Regulatory agencies such as the FDA and EMA have granted orphan drug status to many PNH treatments, streamlining approval processes and increasing availability in different regions. Insurance coverage for high-cost therapies like complement inhibitors has also improved, making these treatments more accessible to patients who previously may not have been able to afford them. Additionally, the introduction of long-acting therapies, which require less frequent dosing, has made treatment more convenient and accessible for patients, particularly those in remote or underserved areas. As access to PNH treatments expands globally, the market is expected to grow, reaching more patients and improving outcomes across diverse regions.

The emergence of gene therapy research holds promise for transforming the PNH treatment market in the long term. By addressing the underlying genetic mutation that causes PNH, gene therapy has the potential to offer a one-time, curative treatment that eliminates the need for lifelong medication. Although still in early stages, gene therapy research has garnered significant interest due to its potential to provide long-lasting relief from PNH symptoms and complications. Investment in gene therapy and CRISPR-based solutions has increased as companies explore their application in PNH treatment. Should these therapies prove successful, they would revolutionize the PNH market, offering a permanent solution for patients and reducing reliance on costly, ongoing treatments. Together, these factors—greater awareness, targeted therapeutic advancements, increased patient access, and gene therapy research—are driving robust growth in the PNH treatment market, establishing it as a key area in rare disease management and innovative healthcare.

SCOPE OF STUDY:

The report analyzes the Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment market in terms of US$ Million by the following Treatment, and Geographic Regions/Countries:

Segments:

Treatment (Medication, Other Treatments)

Geographic Regions/Countries:

World; United States; Canada; Japan; China; Europe (France; Germany; Italy; United Kingdom; and Rest of Europe); Asia-Pacific; Rest of World.

Select Competitors (Total 42 Featured) -

  • Akari Therapeutics Plc
  • Alexion Pharmaceuticals, Inc.
  • Apellis Pharmaceuticals
  • AstraZeneca Plc
  • CANbridge Pharmaceuticals
  • Chugai Pharmaceutical Co., Ltd.
  • F. Hoffmann-La Roche AG
  • Genentech, Inc.
  • Kira Pharmaceuticals
  • Novartis AG

TABLE OF CONTENTS

I. METHODOLOGY

II. EXECUTIVE SUMMARY

  • 1. MARKET OVERVIEW
    • Influencer Market Insights
    • World Market Trajectories
    • Global Economic Update
    • Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment - Global Key Competitors Percentage Market Share in 2024 (E)
    • Competitive Market Presence - Strong/Active/Niche/Trivial for Players Worldwide in 2024 (E)
  • 2. FOCUS ON SELECT PLAYERS
  • 3. MARKET TRENDS & DRIVERS
    • Rising Prevalence of PNH Drives Demand for Novel Therapeutics
    • Advancements in Gene Therapy Drive PNH Treatment Adoption
    • Growing Use of Monoclonal Antibodies in PNH Treatment Expands Market Potential
    • Increasing Adoption of Hematopoietic Stem Cell Transplantation Spurs Demand
    • Rising Awareness Initiatives Enhance Early Diagnosis of PNH
    • Innovations in Eculizumab Alternatives Propel PNH Treatment Market
    • Expanding Applications of Complement Blockers Set the Stage for PNH Growth
    • Use of Advanced Diagnostic Tools Bodes Well for PNH Treatment Expansion
    • Growing Use of Biosimilars in PNH Treatment Expands Addressable Market
    • Demand for Supportive Therapies in PNH Treatment Propels Market Growth
  • 4. GLOBAL MARKET PERSPECTIVE
    • TABLE 1: World Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2023 through 2030 and % CAGR
    • TABLE 2: World Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
    • TABLE 3: World 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets for Years 2014, 2024 & 2030
    • TABLE 4: World Recent Past, Current & Future Analysis for Medication by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2023 through 2030 and % CAGR
    • TABLE 5: World Historic Review for Medication by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
    • TABLE 6: World 16-Year Perspective for Medication by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2014, 2024 & 2030
    • TABLE 7: World Recent Past, Current & Future Analysis for Other Treatments by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2023 through 2030 and % CAGR
    • TABLE 8: World Historic Review for Other Treatments by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
    • TABLE 9: World 16-Year Perspective for Other Treatments by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2014, 2024 & 2030
    • TABLE 10: World Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Analysis of Annual Sales in US$ Million for Years 2014 through 2030

III. MARKET ANALYSIS

  • UNITED STATES
    • Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in the United States for 2024 (E)
    • TABLE 11: USA Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
    • TABLE 12: USA Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
    • TABLE 13: USA 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
  • CANADA
    • TABLE 14: Canada Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
    • TABLE 15: Canada Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
    • TABLE 16: Canada 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
  • JAPAN
    • Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in Japan for 2024 (E)
    • TABLE 17: Japan Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
    • TABLE 18: Japan Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
    • TABLE 19: Japan 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
  • CHINA
    • Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in China for 2024 (E)
    • TABLE 20: China Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
    • TABLE 21: China Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
    • TABLE 22: China 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
  • EUROPE
    • Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in Europe for 2024 (E)
    • TABLE 23: Europe Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Million for Years 2023 through 2030 and % CAGR
    • TABLE 24: Europe Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
    • TABLE 25: Europe 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Geographic Region - Percentage Breakdown of Value Sales for France, Germany, Italy, UK and Rest of Europe Markets for Years 2014, 2024 & 2030
    • TABLE 26: Europe Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
    • TABLE 27: Europe Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
    • TABLE 28: Europe 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
  • FRANCE
    • Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in France for 2024 (E)
    • TABLE 29: France Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
    • TABLE 30: France Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
    • TABLE 31: France 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
  • GERMANY
    • Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in Germany for 2024 (E)
    • TABLE 32: Germany Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
    • TABLE 33: Germany Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
    • TABLE 34: Germany 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
  • ITALY
    • TABLE 35: Italy Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
    • TABLE 36: Italy Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
    • TABLE 37: Italy 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
  • UNITED KINGDOM
    • Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in the United Kingdom for 2024 (E)
    • TABLE 38: UK Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
    • TABLE 39: UK Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
    • TABLE 40: UK 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
  • REST OF EUROPE
    • TABLE 41: Rest of Europe Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
    • TABLE 42: Rest of Europe Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
    • TABLE 43: Rest of Europe 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
  • ASIA-PACIFIC
    • Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in Asia-Pacific for 2024 (E)
    • TABLE 44: Asia-Pacific Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
    • TABLE 45: Asia-Pacific Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
    • TABLE 46: Asia-Pacific 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
  • REST OF WORLD
    • TABLE 47: Rest of World Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
    • TABLE 48: Rest of World Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
    • TABLE 49: Rest of World 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030

IV. COMPETITION

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