Pegcetacoplan: First Approval
Pegcetacoplan (Empaveli™) is a PEGylated pentadecapeptide developed by Apellis Pharmaceuticals for the treatment of complement-mediated diseases. It binds to complement component 3 (C3) and its activation fragment C3b, controlling the cleavage of C3 and the generation of the downstream effectors of...
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| Veröffentlicht in: | Drugs (New York, N.Y.) N.Y.), 2021-08, Vol.81 (12), p.1423-1430 |
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| description | Pegcetacoplan (Empaveli™) is a PEGylated pentadecapeptide developed by Apellis Pharmaceuticals for the treatment of complement-mediated diseases. It binds to complement component 3 (C3) and its activation fragment C3b, controlling the cleavage of C3 and the generation of the downstream effectors of complement activation and thus both C3b-mediated extravascular haemolysis and terminal complement-mediated intravascular haemolysis. Pegcetacoplan is the first C3-targeted paroxysmal nocturnal haemoglobinuria (PNH) therapy to be approved (in May 2021) in the USA, where it is indicated for the treatment of adults with PNH, including those switching from C5 inhibitor therapy with eculizumab and ravulizumab. A regulatory assessment of pegcetacoplan for the treatment of PNH is currently underway in the EU and Australia. Pegcetacoplan is also being investigated as a therapeutic option in other complement-mediated diseases, including age-related macular degeneration, C3 glomerulopathy and autoimmune haemolytic anaemia. The recommended dosage regimen of pegcetacoplan is 1080 mg twice weekly, administered as a subcutaneous infusion via an infusion pump with a ≥ 20 mL reservoir. This article summarizes the milestones in the development of pegcetacoplan leading to this first approval for the treatment of adults with PNH. |
| doi_str_mv | 10.1007/s40265-021-01560-8 |
| format | Article |
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It binds to complement component 3 (C3) and its activation fragment C3b, controlling the cleavage of C3 and the generation of the downstream effectors of complement activation and thus both C3b-mediated extravascular haemolysis and terminal complement-mediated intravascular haemolysis. Pegcetacoplan is the first C3-targeted paroxysmal nocturnal haemoglobinuria (PNH) therapy to be approved (in May 2021) in the USA, where it is indicated for the treatment of adults with PNH, including those switching from C5 inhibitor therapy with eculizumab and ravulizumab. A regulatory assessment of pegcetacoplan for the treatment of PNH is currently underway in the EU and Australia. Pegcetacoplan is also being investigated as a therapeutic option in other complement-mediated diseases, including age-related macular degeneration, C3 glomerulopathy and autoimmune haemolytic anaemia. The recommended dosage regimen of pegcetacoplan is 1080 mg twice weekly, administered as a subcutaneous infusion via an infusion pump with a ≥ 20 mL reservoir. This article summarizes the milestones in the development of pegcetacoplan leading to this first approval for the treatment of adults with PNH.</description><identifier>ISSN: 0012-6667</identifier><identifier>EISSN: 1179-1950</identifier><identifier>DOI: 10.1007/s40265-021-01560-8</identifier><identifier>PMID: 34342834</identifier><language>eng</language><publisher>Cham: Springer International Publishing</publisher><subject>AdisInsight Report ; Adults ; Agreements ; Amyotrophic lateral sclerosis ; Anemia ; Anemia, Hemolytic, Autoimmune - drug therapy ; Autoimmune hemolytic anemia ; Collaboration ; Complement ; Complement activation ; Complement C3 - antagonists & inhibitors ; Complement C3b - antagonists & inhibitors ; Complement component C3 ; Complement component C3b ; Complement Inactivating Agents - pharmacology ; Complement Inactivating Agents - therapeutic use ; Complement system ; Drug dosages ; Eye diseases ; Geographic Atrophy - drug therapy ; Glomerulonephritis - drug therapy ; Hemoglobin ; Hemoglobinuria, Paroxysmal - drug therapy ; Hemolysis ; Humans ; Infusion pumps ; Internal Medicine ; Macular degeneration ; Medicine ; Medicine & Public Health ; Patients ; Peptides ; Peptides, Cyclic - pharmacology ; Peptides, Cyclic - therapeutic use ; Pharmaceuticals ; Pharmacology/Toxicology ; Pharmacotherapy ; Streptococcus infections</subject><ispartof>Drugs (New York, N.Y.), 2021-08, Vol.81 (12), p.1423-1430</ispartof><rights>The Author(s), under exclusive licence to Springer Nature Switzerland AG 2021</rights><rights>2021. 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Aug 2021</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c375t-51321725939041f81e9241aa7eb90651494cc0c7a07ae793c8511133892996d93</citedby><cites>FETCH-LOGICAL-c375t-51321725939041f81e9241aa7eb90651494cc0c7a07ae793c8511133892996d93</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://link.springer.com/content/pdf/10.1007/s40265-021-01560-8$$EPDF$$P50$$Gspringer$$H</linktopdf><linktohtml>$$Uhttps://link.springer.com/10.1007/s40265-021-01560-8$$EHTML$$P50$$Gspringer$$H</linktohtml><link.rule.ids>314,780,784,27924,27925,41488,42557,51319</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/34342834$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Hoy, Sheridan M.</creatorcontrib><title>Pegcetacoplan: First Approval</title><title>Drugs (New York, N.Y.)</title><addtitle>Drugs</addtitle><addtitle>Drugs</addtitle><description>Pegcetacoplan (Empaveli™) is a PEGylated pentadecapeptide developed by Apellis Pharmaceuticals for the treatment of complement-mediated diseases. 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The recommended dosage regimen of pegcetacoplan is 1080 mg twice weekly, administered as a subcutaneous infusion via an infusion pump with a ≥ 20 mL reservoir. 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drug therapy</subject><subject>Hemoglobin</subject><subject>Hemoglobinuria, Paroxysmal - drug therapy</subject><subject>Hemolysis</subject><subject>Humans</subject><subject>Infusion pumps</subject><subject>Internal Medicine</subject><subject>Macular degeneration</subject><subject>Medicine</subject><subject>Medicine & Public Health</subject><subject>Patients</subject><subject>Peptides</subject><subject>Peptides, Cyclic - pharmacology</subject><subject>Peptides, Cyclic - therapeutic use</subject><subject>Pharmaceuticals</subject><subject>Pharmacology/Toxicology</subject><subject>Pharmacotherapy</subject><subject>Streptococcus infections</subject><issn>0012-6667</issn><issn>1179-1950</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2021</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><sourceid>ABUWG</sourceid><sourceid>AFKRA</sourceid><sourceid>BENPR</sourceid><sourceid>CCPQU</sourceid><recordid>eNp9kE1LAzEQhoMotlb_gKAIXrxEZ_K58VaKVaGgBz2HNE1Ly7a7JruC_97UrQoePA3DPPPO8BByinCNAPomCWBKUmBIAaUCWuyRPqI2FI2EfdIHQEaVUrpHjlJabVsjzSHpccEFK7jok7PnsPChcb6qS7e5vRgvY2ouhnUdq3dXHpODuStTONnVAXkd372MHujk6f5xNJxQz7VsqETOUDNpuAGB8wKDYQKd02FqQEkURngPXjvQLmjDfSERkfPCMGPUzPABuepy89m3NqTGrpfJhzK_FKo2WSalltxIVBm9_IOuqjZu8neZUiCAMwaZYh3lY5VSDHNbx-XaxQ-LYLfybCfPZnn2S54t8tL5LrqdrsPsZ-XbVgZ4B6Q82ixC_L39T-wn2Ix1Cw</recordid><startdate>20210801</startdate><enddate>20210801</enddate><creator>Hoy, Sheridan M.</creator><general>Springer International Publishing</general><general>Springer Nature B.V</general><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>3V.</scope><scope>4T-</scope><scope>7QO</scope><scope>7RV</scope><scope>7TK</scope><scope>7U7</scope><scope>7U9</scope><scope>7X7</scope><scope>7XB</scope><scope>88E</scope><scope>8FD</scope><scope>8FI</scope><scope>8FJ</scope><scope>8FK</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>AN0</scope><scope>BENPR</scope><scope>C1K</scope><scope>CCPQU</scope><scope>FR3</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>H94</scope><scope>K9.</scope><scope>KB0</scope><scope>M0S</scope><scope>M1P</scope><scope>NAPCQ</scope><scope>P64</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>7X8</scope></search><sort><creationdate>20210801</creationdate><title>Pegcetacoplan: First Approval</title><author>Hoy, Sheridan M.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c375t-51321725939041f81e9241aa7eb90651494cc0c7a07ae793c8511133892996d93</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2021</creationdate><topic>AdisInsight Report</topic><topic>Adults</topic><topic>Agreements</topic><topic>Amyotrophic lateral sclerosis</topic><topic>Anemia</topic><topic>Anemia, Hemolytic, Autoimmune - drug therapy</topic><topic>Autoimmune hemolytic anemia</topic><topic>Collaboration</topic><topic>Complement</topic><topic>Complement activation</topic><topic>Complement C3 - antagonists & inhibitors</topic><topic>Complement C3b - antagonists & inhibitors</topic><topic>Complement component C3</topic><topic>Complement component C3b</topic><topic>Complement Inactivating Agents - pharmacology</topic><topic>Complement Inactivating Agents - therapeutic use</topic><topic>Complement system</topic><topic>Drug dosages</topic><topic>Eye diseases</topic><topic>Geographic Atrophy - drug therapy</topic><topic>Glomerulonephritis - drug therapy</topic><topic>Hemoglobin</topic><topic>Hemoglobinuria, Paroxysmal - drug therapy</topic><topic>Hemolysis</topic><topic>Humans</topic><topic>Infusion pumps</topic><topic>Internal Medicine</topic><topic>Macular degeneration</topic><topic>Medicine</topic><topic>Medicine & Public Health</topic><topic>Patients</topic><topic>Peptides</topic><topic>Peptides, Cyclic - pharmacology</topic><topic>Peptides, Cyclic - therapeutic use</topic><topic>Pharmaceuticals</topic><topic>Pharmacology/Toxicology</topic><topic>Pharmacotherapy</topic><topic>Streptococcus infections</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Hoy, Sheridan M.</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>ProQuest Central (Corporate)</collection><collection>Docstoc</collection><collection>Biotechnology Research Abstracts</collection><collection>Nursing & Allied Health Database</collection><collection>Neurosciences Abstracts</collection><collection>Toxicology Abstracts</collection><collection>Virology and AIDS Abstracts</collection><collection>Health & Medical Collection</collection><collection>ProQuest Central (purchase pre-March 2016)</collection><collection>Medical Database (Alumni Edition)</collection><collection>Technology Research Database</collection><collection>Hospital Premium Collection</collection><collection>Hospital Premium Collection (Alumni Edition)</collection><collection>ProQuest Central (Alumni) (purchase pre-March 2016)</collection><collection>ProQuest Central (Alumni Edition)</collection><collection>ProQuest Central UK/Ireland</collection><collection>British Nursing Database</collection><collection>ProQuest Central</collection><collection>Environmental Sciences and Pollution Management</collection><collection>ProQuest One Community College</collection><collection>Engineering Research Database</collection><collection>Health Research Premium Collection</collection><collection>Health Research Premium Collection (Alumni)</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>Nursing & Allied Health Database (Alumni Edition)</collection><collection>Health & Medical Collection (Alumni Edition)</collection><collection>Medical Database</collection><collection>Nursing & Allied Health Premium</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>ProQuest One Academic Eastern Edition (DO NOT USE)</collection><collection>ProQuest One Academic</collection><collection>ProQuest One Academic UKI Edition</collection><collection>MEDLINE - Academic</collection><jtitle>Drugs (New York, N.Y.)</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Hoy, Sheridan M.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Pegcetacoplan: First Approval</atitle><jtitle>Drugs (New York, N.Y.)</jtitle><stitle>Drugs</stitle><addtitle>Drugs</addtitle><date>2021-08-01</date><risdate>2021</risdate><volume>81</volume><issue>12</issue><spage>1423</spage><epage>1430</epage><pages>1423-1430</pages><issn>0012-6667</issn><eissn>1179-1950</eissn><abstract>Pegcetacoplan (Empaveli™) is a PEGylated pentadecapeptide developed by Apellis Pharmaceuticals for the treatment of complement-mediated diseases. It binds to complement component 3 (C3) and its activation fragment C3b, controlling the cleavage of C3 and the generation of the downstream effectors of complement activation and thus both C3b-mediated extravascular haemolysis and terminal complement-mediated intravascular haemolysis. Pegcetacoplan is the first C3-targeted paroxysmal nocturnal haemoglobinuria (PNH) therapy to be approved (in May 2021) in the USA, where it is indicated for the treatment of adults with PNH, including those switching from C5 inhibitor therapy with eculizumab and ravulizumab. A regulatory assessment of pegcetacoplan for the treatment of PNH is currently underway in the EU and Australia. Pegcetacoplan is also being investigated as a therapeutic option in other complement-mediated diseases, including age-related macular degeneration, C3 glomerulopathy and autoimmune haemolytic anaemia. The recommended dosage regimen of pegcetacoplan is 1080 mg twice weekly, administered as a subcutaneous infusion via an infusion pump with a ≥ 20 mL reservoir. This article summarizes the milestones in the development of pegcetacoplan leading to this first approval for the treatment of adults with PNH.</abstract><cop>Cham</cop><pub>Springer International Publishing</pub><pmid>34342834</pmid><doi>10.1007/s40265-021-01560-8</doi><tpages>8</tpages></addata></record> |
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| ispartof | Drugs (New York, N.Y.), 2021-08, Vol.81 (12), p.1423-1430 |
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| source | MEDLINE; SpringerLink Journals - AutoHoldings |
| subjects | AdisInsight Report Adults Agreements Amyotrophic lateral sclerosis Anemia Anemia, Hemolytic, Autoimmune - drug therapy Autoimmune hemolytic anemia Collaboration Complement Complement activation Complement C3 - antagonists & inhibitors Complement C3b - antagonists & inhibitors Complement component C3 Complement component C3b Complement Inactivating Agents - pharmacology Complement Inactivating Agents - therapeutic use Complement system Drug dosages Eye diseases Geographic Atrophy - drug therapy Glomerulonephritis - drug therapy Hemoglobin Hemoglobinuria, Paroxysmal - drug therapy Hemolysis Humans Infusion pumps Internal Medicine Macular degeneration Medicine Medicine & Public Health Patients Peptides Peptides, Cyclic - pharmacology Peptides, Cyclic - therapeutic use Pharmaceuticals Pharmacology/Toxicology Pharmacotherapy Streptococcus infections |
| title | Pegcetacoplan: First Approval |
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