Pegcetacoplan

Pegcetacoplan, sold under the brand name Empaveli, among others, is a medication used to treat paroxysmal nocturnal hemoglobinuria[4][7][8][9][10] and geographic atrophy of the retina.[6][11] Pegcetacoplan is a complement inhibitor.[4][6]

Pegcetacoplan
Clinical data
Trade namesEmpaveli, Aspaveli, Syfovre
Other namesAPL-2
AHFS/Drugs.comMonograph
MedlinePlusa621045
License data
Pregnancy
category
Routes of
administration		Subcutaneous, intravitreal
Drug classComplement inhibitor
ATC code
Legal status
Legal status
Identifiers
CAS Number
DrugBank
UNII
KEGG
ChEMBL
Chemical and physical data
FormulaC170H248N50O47S4
Molar mass3872.40 g·mol−1

The most common side effects include injection-site reactions, infections, diarrhea, abdominal pain, respiratory tract infection, viral infection, and fatigue.[4][7]

Paroxysmal nocturnal hemoglobinuria is characterized by red blood cell destruction, anemia (red blood cells unable to carry enough oxygen to tissues), blood clots, and impaired bone marrow function (not making enough blood cells).[5]

Pegcetacoplan is the first treatment for paroxysmal nocturnal hemoglobinuria that binds to and inhibits complement protein C3.[5] Pegcetacoplan was approved for medical use in the United States in May 2021.[5] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.[12]

Medical uses

Pegcetacoplan is indicated to treat adults with paroxysmal nocturnal hemoglobinuria.[4][5][7]

In February 2023, the indication was updated to include the treatment of people with geographic atrophy secondary to age-related macular degeneration.[6][13]

Pharmacology

People with paroxysmal nocturnal hemoglobinuria have greater and uninhibited complement activity, which may leads to intravascular (inside blood vessels) or extravascular (within the liver or spleen) hemolysis.[4]

Adverse effects

Meningococcal (a type of bacteria) infections can occur in people taking pegcetacoplan.[5] Pegcetacoplan may also predispose individuals to serious infections, especially infections caused by encapsulated bacteria.[5] These infections include but are not limited to Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae.[4] Common adverse effects associated with the medication include stomach pain, vomiting, diarrhea, cold sores, common-cold like symptoms, tiredness as well as any itching, redness, or sensitivity at the injection site.[4] Pegcetacoplan may cause fetal harm.[4] Pegcetacoplan may also interfere with silica reagents in laboratory coagulation panels.[4]

History

The effectiveness of pegcetacoplan was evaluated in a study enrolling 80 participants with paroxysmal nocturnal hemoglobinuria and anemia who had been taking eculizumab, a treatment previously approved for paroxysmal nocturnal hemoglobinuria.[5]

The FDA granted the application for pegcetacoplan orphan drug designation.[12]

Society and culture

On 14 October 2021, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Aspaveli, intended for the treatment of adults with paroxysmal nocturnal hemoglobinuria.[14] The applicant for this medicinal product is Swedish Orphan Biovitrum AB (publ).[14] Pegcetacoplan was approved for medical use in the European Union in December 2021.[7][15]

References
  1. "Empaveli APMDS". Therapeutic Goods Administration (TGA). 17 February 2022. Retrieved 20 February 2022.
  2. "Updates to the Prescribing Medicines in Pregnancy database". Therapeutic Goods Administration (TGA). 21 December 2022. Archived from the original on 3 April 2022. Retrieved 2 January 2023.
  3. "Notice: Multiple Additions to the Prescription Drug List (PDL) [2023-03-08]". Health Canada. 8 March 2023. Retrieved 21 March 2023.
  4. "Empaveli- pegcetacoplan injection, solution". DailyMed. 10 August 2021. Archived from the original on 20 May 2022. Retrieved 18 February 2023.
  5. "FDA approves new treatment for adults with serious rare blood disease". U.S. Food & Drug Administration (FDA). 18 May 2021. Archived from the original on 17 October 2021. Retrieved 18 February 2023. Public Domain This article incorporates text from this source, which is in the public domain.
  6. "Syfovre (pegcetacoplan injection), for intravitreal use" (PDF). Archived (PDF) from the original on 22 February 2023. Retrieved 21 February 2023.
  7. "Aspaveli EPAR". European Medicines Agency (EMA). 6 September 2022. Archived from the original on 18 February 2023. Retrieved 18 February 2023.
  8. Heo YA (December 2022). "Pegcetacoplan: A Review in Paroxysmal Nocturnal Haemoglobinuria". Drugs. 82 (18): 1727–1735. doi:10.1007/s40265-022-01809-w. PMID 36459381. S2CID 254149706.
  9. Wong RS (2022). "Safety and efficacy of pegcetacoplan in paroxysmal nocturnal hemoglobinuria". Therapeutic Advances in Hematology. 13: 20406207221114673. doi:10.1177/20406207221114673. PMC 9340389. PMID 35923770.
  10. Xu B (April 2022). "Novel targeted C3 inhibitor pegcetacoplan for paroxysmal nocturnal hemoglobinuria". Clinical and Experimental Medicine. doi:10.1007/s10238-022-00830-3. PMID 35441351. S2CID 248245151.
  11. Liao DS, Metlapally R, Joshi P (September 2022). "Pegcetacoplan treatment for geographic atrophy due to age-related macular degeneration: a plain language summary of the FILLY study". Immunotherapy. 14 (13): 995–1006. doi:10.2217/imt-2022-0078. PMID 35860926. S2CID 250731980.
  12. Advancing Health Through Innovation: New Drug Therapy Approvals 2021 (PDF). U.S. Food and Drug Administration (FDA) (Report). 13 May 2022. Archived from the original on 6 December 2022. Retrieved 22 January 2023. Public Domain This article incorporates text from this source, which is in the public domain.
  13. "FDA Approves SYFOVRE™ (pegcetacoplan injection) as the First and Only Treatment for Geographic Atrophy (GA), a Leading Cause of Blindness" (Press release). Apellis Pharmaceuticals. 17 February 2023. Archived from the original on 17 February 2023. Retrieved 18 February 2023 – via GlobeNewswire.
  14. "Aspaveli: Pending EC decision". European Medicines Agency (EMA). 14 October 2021. Archived from the original on 18 October 2021. Retrieved 15 October 2021. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  15. "Aspaveli Product information". Union Register of medicinal products. Retrieved 3 March 2023.

Public Domain This article incorporates public domain material from the United States Department of Health and Human Services.

  • "Pegcetacoplan". Drug Information Portal. U.S. National Library of Medicine.
  • Clinical trial number NCT03500549 for "Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)" at ClinicalTrials.gov
  • Clinical trial number NCT03525613 for "A Study to Compare the Efficacy and Safety of Intravitreal APL-2 Therapy With Sham Injections in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration " at ClinicalTrials.gov
  • Clinical trial number NCT03525600 for "Study to Compare the Efficacy and Safety of Intravitreal APL-2 Therapy With Sham Injections in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration " at ClinicalTrials.gov
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