#

Dailypharm Live Search Close
  • Qalsody receives orphan drug designation in Korea
  • by Eo, Yun-Ho | translator Alice Kang | Aug 8, 2024 09:24am
New drug for ALS, an area that had no available treatment option
Approved in the US in 2023, and then in Europe recently
Lack of treatment options draws attention to commercialized drugs
¡®Qalsody (tofersen),¡¯ a new drug for Lou Gehrig's disease, was designated as an orphan drug in Korea.

The Ministry of Food and Drug Safety (MFDS) recently announced the designation through an orphan drug designation notice.

More specifically, the drug is indicated for amyotrophic lateral sclerosis (Lou Gehrig's disease) associated with a mutation in the SOD1 (Superoxide Dismutase 1) gene.

Qalsody, which was developed by Biogen, is an antisense oligonucleotide (ASO) drug that blocks the messenger ribonucleic acid (mRNA) associated with the SOD1 gene mutation to prevent its expression.

The drug was approved by the U.S. FDA in June last year, followed by the European EMA in May.

The efficacy data for Qa
  • 0
Reader Comment
0
Anonymity comment Write Operate Rule
Colse

´ñ±Û ¿î¿µ¹æ½ÄÀº

´ñ±ÛÀº ½Ç¸í°ÔÀç¿Í À͸í°ÔÀç ¹æ½ÄÀÌ ÀÖÀ¸¸ç, ½Ç¸íÀº À̸§°ú ¾ÆÀ̵𰡠³ëÃâµË´Ï´Ù. À͸íÀº ÇʸíÀ¸·Î µî·Ï °¡´ÉÇϸç, ´ë´ñ±ÛÀº À͸íÀ¸·Î µî·Ï °¡´ÉÇÕ´Ï´Ù.

´ñ±Û ³ëÃâ¹æ½ÄÀº

´ñ±Û ¸í¿¹ÀÚ¹®À§¿ø(ÆÊ-ÄڴϾð-Çʱâ¸ð¾ç ¾ÆÀÌÄÜ)À¸·Î À§ÃËµÈ µ¥Àϸ®ÆÊ ȸ¿øÀÇ ´ñ±ÛÀº ¡®°Ô½ÃÆÇÇü º¸±â¡¯¿Í ¡¯ÆîÃ帱âÇü¡¯ ¸®½ºÆ®¿¡¼­ Ç×»ó ÃÖ»ó´Ü¿¡ ³ëÃâµË´Ï´Ù. »õ·Î¿î ´ñ±ÛÀ» ¿Ã¸®´Â ÀϹÝȸ¿øÀº ¡®°Ô½ÃÆÇÇü¡¯°ú ¡®ÆîÃ帱âÇü¡¯ ¸ðµÎ ÆÊÄڴϾð ȸ¿øÀÌ ¾´ ´ñ±ÛÀÇ ÇÏ´Ü¿¡ ½Ç½Ã°£ ³ëÃâµË´Ï´Ù.

´ñ±ÛÀÇ »èÁ¦ ±âÁØÀº

´ÙÀ½ÀÇ °æ¿ì »çÀü Å뺸¾øÀÌ »èÁ¦ÇÏ°í ¾ÆÀ̵ð ÀÌ¿ëÁ¤Áö ¶Ç´Â ¿µ±¸ °¡ÀÔÁ¦ÇÑÀÌ µÉ ¼öµµ ÀÖ½À´Ï´Ù.

  • ÀúÀ۱ǡ¤ÀÎ°Ý±Ç µî ŸÀÎÀÇ ±Ç¸®¸¦ Ä§ÇØÇÏ´Â °æ¿ì

    »ó¿ë ÇÁ·Î±×·¥ÀÇ µî·Ï°ú °ÔÀç, ¹èÆ÷¸¦ ¾È³»ÇÏ´Â °Ô½Ã¹°

    ŸÀÎ ¶Ç´Â Á¦3ÀÚÀÇ ÀúÀÛ±Ç ¹× ±âŸ ±Ç¸®¸¦ Ä§ÇØÇÑ ³»¿ëÀ» ´ãÀº °Ô½Ã¹°

  • ±Ù°Å ¾ø´Â ºñ¹æ¡¤¸í¿¹¸¦ ÈѼÕÇÏ´Â °Ô½Ã¹°

    ƯÁ¤ ÀÌ¿ëÀÚ ¹× °³Àο¡ ´ëÇÑ ÀνŠ°ø°ÝÀûÀÎ ³»¿ëÀÇ ±Û ¹× Á÷Á¢ÀûÀÎ ¿å¼³ÀÌ »ç¿ëµÈ °æ¿ì

    ƯÁ¤ Áö¿ª ¹× Á¾±³°£ÀÇ °¨Á¤´ë¸³À» Á¶ÀåÇÏ´Â ³»¿ë

    »ç½Ç È®ÀÎÀÌ ¾ÈµÈ ¼Ò¹®À» À¯Æ÷ ½ÃŰ´Â °æ¿ì

    ¿å¼³°ú ºñ¾î, ¼Ó¾î¸¦ ´ãÀº ³»¿ë

    Á¤´ç¹ý ¹× °øÁ÷¼±°Å¹ý, °ü°è ¹ý·É¿¡ ÀúÃ˵Ǵ °æ¿ì(¼±°üÀ§ ¿äû ½Ã Áï½Ã »èÁ¦)

    ƯÁ¤ Áö¿ªÀ̳ª ´Üü¸¦ ºñÇÏÇÏ´Â °æ¿ì

    ƯÁ¤ÀÎÀÇ ¸í¿¹¸¦ ÈѼÕÇÏ¿© ÇØ´çÀÎÀÌ »èÁ¦¸¦ ¿äûÇÏ´Â °æ¿ì

    ƯÁ¤ÀÎÀÇ °³ÀÎÁ¤º¸(Áֹεî·Ï¹øÈ£, ÀüÈ­, »ó¼¼ÁÖ¼Ò µî)¸¦ ¹«´ÜÀ¸·Î °Ô½ÃÇÏ´Â °æ¿ì

    ŸÀÎÀÇ ID ȤÀº ´Ð³×ÀÓÀ» µµ¿ëÇÏ´Â °æ¿ì

  • °Ô½ÃÆÇ Ư¼º»ó Á¦ÇѵǴ ³»¿ë

    ¼­ºñ½º ÁÖÁ¦¿Í ¸ÂÁö ¾Ê´Â ³»¿ëÀÇ ±ÛÀ» °ÔÀçÇÑ °æ¿ì

    µ¿ÀÏ ³»¿ëÀÇ ¿¬¼Ó °ÔÀç ¹× ¿©·¯ ±â»ç¿¡ Áߺ¹ °ÔÀçÇÑ °æ¿ì

    ºÎºÐÀûÀ¸·Î º¯°æÇÏ¿© ¹Ýº¹ °ÔÀçÇÏ´Â °æ¿ìµµ Æ÷ÇÔ

    Á¦¸ñ°ú °ü·Ã ¾ø´Â ³»¿ëÀÇ °Ô½Ã¹°, Á¦¸ñ°ú º»¹®ÀÌ ¹«°üÇÑ °æ¿ì

    µ·¹ú±â ¹× Á÷¡¤°£Á¢ »ó¾÷Àû ¸ñÀûÀÇ ³»¿ëÀÌ Æ÷ÇÔµÈ °Ô½Ã¹°

    °Ô½Ã¹° Àбâ À¯µµ µîÀ» À§ÇØ ³»¿ë°ú ¹«°üÇÑ Á¦¸ñÀ» »ç¿ëÇÑ °æ¿ì

  • ¼ö»ç±â°ü µîÀÇ °ø½ÄÀûÀÎ ¿äûÀÌ ÀÖ´Â °æ¿ì

  • ±âŸ»çÇ×

    °¢ ¼­ºñ½ºÀÇ Çʿ伺¿¡ µû¶ó ¹Ì¸® °øÁöÇÑ °æ¿ì

    ±âŸ ¹ý·ü¿¡ ÀúÃ˵Ǵ Á¤º¸ °ÔÀ縦 ¸ñÀûÀ¸·Î ÇÒ °æ¿ì

    ±âŸ ¿ø¸¸ÇÑ ¿î¿µÀ» À§ÇØ ¿î¿µÀÚ°¡ ÇÊ¿äÇÏ´Ù°í ÆÇ´ÜµÇ´Â ³»¿ë

  • »ç½Ç °ü°è È®ÀÎ ÈÄ »èÁ¦

    ÀúÀÛ±ÇÀڷκÎÅÍ Çã¶ô¹ÞÁö ¾ÊÀº ³»¿ëÀ» ¹«´Ü °ÔÀç, º¹Á¦, ¹èÆ÷ÇÏ´Â °æ¿ì

    ŸÀÎÀÇ ÃÊ»ó±ÇÀ» Ä§ÇØÇϰųª °³ÀÎÁ¤º¸¸¦ À¯ÃâÇÏ´Â °æ¿ì

    ´ç»ç¿¡ Á¦°øÇÑ ÀÌ¿ëÀÚÀÇ Á¤º¸°¡ ÇãÀ§ÀÎ °æ¿ì (ŸÀÎÀÇ ID, ºñ¹Ð¹øÈ£ µµ¿ë µî)

  • ¡ØÀÌ»óÀÇ ³»¿ëÁß ÀϺΠ»çÇ׿¡ Àû¿ëµÉ °æ¿ì ÀÌ¿ë¾à°ü ¹× °ü·Ã ¹ý·ü¿¡ ÀÇÇØ Á¦À縦 ¹ÞÀ¸½Ç ¼öµµ ÀÖÀ¸¸ç, ¹Î¡¤Çü»ç»ó ó¹úÀ» ¹ÞÀ» ¼öµµ ÀÖ½À´Ï´Ù.

    ¡ØÀ§¿¡ ¸í½ÃµÇÁö ¾ÊÀº ³»¿ëÀÌ´õ¶óµµ ºÒ¹ýÀûÀÎ ³»¿ëÀ¸·Î ÆÇ´ÜµÇ°Å³ª µ¥Àϸ®ÆÊ ¼­ºñ½º¿¡ ¹Ù¶÷Á÷ÇÏÁö ¾Ê´Ù°í ÆÇ´ÜµÇ´Â °æ¿ì´Â ¼± Á¶Ä¡ ÀÌÈÄ º» °ü¸® ±âÁØÀ» ¼öÁ¤ °ø½ÃÇϰڽÀ´Ï´Ù.

    ¡Ø±âŸ ¹®ÀÇ »çÇ×Àº µ¥Àϸ®ÆÊ ¿î¿µÀÚ¿¡°Ô ¿¬¶ôÁֽʽÿÀ. ¸ÞÀÏ ÁÖ¼Ò´Â dailypharm@dailypharm.comÀÔ´Ï´Ù.

Write
Write
recently Agree DisAgree
If you want to see the full article, please JOIN US (click)