Novel FDA Drug Approvals - 4th Quarter of 2023 - PharmaKB Report

The Pharmaceutical KnowledgeBase (PharmaKB.com) provides research and financial intelligence about companies and their approved drugs as well as drug candidates in clinical trials. In this quarterly PharmaKB report, we provide an update about novel drugs approved by the US FDA in of this year.  By referencing new drugs as “novel” the FDA is confirming that the medicine being approved contains ingredients and/or a mechanism of action that the FDA hasn’t previously approved. 

2023 | October | November | December

October 2023 – Novel FDA Drug Approvals

Etrasimod

• Drug: Etrasimod
• Trade name: Velsipity
• Therapeutic Area: Ulcerative colitis MeSH D003093.
• Manufacturer: Pfizer Labs
• FDA Approval Date: October 12, 2023
• Classification: Small molecule
• Drug Class: Immunomodulators
• Clinical Trials: https://www.app.pharmakb.com/drug-report/etrasimod#clinical
• Adverse Events: e.g.  https://www.app.pharmakb.com/drug-report/etrasimod#safety
• Description: Etasimod, sold under the trade name Velsipity, is used to treat moderately to severely active ulcerative colitis in adults. It is taken by mouth.
• FDA News Release: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/216956s000lbl.pdf

Zilucoplan

• Drug: Zilucoplan
• Trade name: Zilbrysq    
• Therapeutic Area: Myasthenia gravis MeSH DOO9157
• Manufacturer: UCB
• FDA Approval Date: October 17, 2023
• Classification: Protein
• Drug Class: Complement ligand inhibitors
• Clinical Trials:     https://www.app.pharmakb.com/drug-report/zilucoplan#clinical
• Adverse Events: https://www.app.pharmakb.com/drug-report/zilucoplan#safety
• Description: Zilucoplan, sold under the trade name Zilbrysq, is used to treat generalized myasthenia gravis in adults who are anti-acetylcholine receptor (AChR) antibody positive. It is administered subcutaneously.
• FDA News Release: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/216834s000lbl.pdf

Bimekizumab

• Drug: Bimekizumab
• Trade name: BIMZELX    
• Therapeutic Area: Plaque psoriasis MeSH D011565
• Manufacturer: UCB
• FDA Approval Date: October 17, 2023
• Classification: Antibody
• Drug Class: Monoclonal antibody (Cytokine inhibitor)
• Clinical Trials:     https://www.app.pharmakb.com/drug-report/bimekizumab#clinical
• Adverse Events: e.g.  https://www.app.pharmakb.com/drug-report/bimekizumab#safety
• Description: Bimekizumab, sold under the trade name BIMZELX, is a humanized anti-IL17A, anti-IL-17F, and anti-IL17AF monoclonal antibody used to treat moderate to severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy, administered subcutaneously.
• FDA News Release: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761151s000lbl.pdf

Vamorolone

• Drug: Vamorolone
• Trade name: Agamree    
• Therapeutic Area: Duchenne muscular dystrophy MeSH D020388
• Manufacturer: Santhera Pharmaceuticals
• FDA Approval Date: October 26, 2023
• Classification: Small molecule
• Drug Class: Steroids (non prednisolone derivatives)
• Clinical Trials:     https://www.app.pharmakb.com/drug-report/vamorolone #clinical
• Adverse Events: e.g.  https://www.app.pharmakb.com/drug-report/vamorolone #safety
• Description: Vamorolone, sold under the trade name Agamree, is used to treat Duchenne muscular dystrophy. It is taken by mouth.
• FDA News Release: e.g https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215239s000lbl.pdf

Mirikizumab-mrkz

• Drug: Mirikizumab
• Trade name: Omvoh
• Therapeutic Area: Ulcerative colitis MeSH D003093
• Manufacturer: Eli Lilly
• FDA Approval Date: October 26, 2023
• Classification: Antibody
• Drug Class: Monoclonal antibody
• Clinical Trials:     https://www.app.pharmakb.com/drug-report/mirikizumab-mrkz#clinical
• Adverse Events: e.g.  https://www.app.pharmakb.com/drug-report/mirikizumab-mrkz#safety
• Description: Mirikizumab-mrkz, sold under the trade name Omvoh, is a mineralocorticoid receptor antagonist used to treat ulcerative colitis. It is taken by mouth.
• FDA News Release: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761279s000lbl.pdf

Toripalimab-tpzi

• • Drug: Toripalimab-tpzi
  • Trade name: Loqtorzi      
  • Therapeutic Area: Nasopharyngeal carcinoma MeSH D000077274
  • Manufacturer: Coherus BioSciences
  • FDA Approval Date: October 27, 2023
  • Classification: Antibody
  • Drug Class: Monoclonal antibody
  • Clinical Trials:     https://www.app.pharmakb.com/drug-report/toripalimab#clinical
  • Adverse Events: e.g.  https://www.app.pharmakb.com/drug-report/toripalimab #safety
  • Description: Toripalimab, sold under the trade name Loqtorzi, is used to treat recurrent or metastatic nasopharyngeal carcinoma when used together with or following other therapies. It is administered intravenously.
  • FDA News Release: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-toripalimab-tpzi-nasopharyngeal-carcinoma

November 2023 – Novel FDA Drug Approvals

Fruquintinib

• Drug: Fruquintinib
• Trade name: Fruzaqla    
• Therapeutic Area: Colorectal cancer MeSH D015179
• Manufacturer: Takeda Pharmaceuticals
• FDA Approval Date: November 8, 2023
• Classification: Small molecule
• Drug Class: Tyrosine kinase inhibitors
• Clinical Trials:     https://www.app.pharmakb.com/drug-report/fruquintinib#clinical
• Adverse Events: e.g.  https://www.app.pharmakb.com/drug-report/fruquintinib#safety
• Description: Fruquintini, sold under the trade name Fruzaqla, is used to treat refractory, metastatic colorectal cancer. It is taken by mouth.
• FDA News Release: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-fruquintinib-refractory-metastatic-colorectal-cancer

Taurolidine, heparin

• Drug: Taurolidine
• Trade name: Defencath    
• Therapeutic Area: Bloodstream infection Mesh D055499
• Manufacturer: CorMedix
• FDA Approval Date: November 15, 2023
• Classification: Small molecule
• Drug Class: Thiadiazinane
• Clinical Trials:     https://www.app.pharmakb.com/drug-report/taurolidine #clinical
• Adverse Events: e.g.  https://www.app.pharmakb.com/drug-report/taurolidine #safety
• Description: Taurolidine and heparin, sold under the trade name, Defencath is used as a catheter lock solution to reduce catheter-related bloodstream infections in adult patients with kidney failure who are receiving chronic hemodialysis through a central venous catheter. Defencath is intended for use as a catheter lock solution only and is not meant to be injected into the body or used as a catheter lock flush product.
• FDA News Release: https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-new-drug-under-special-pathway-patients-receiving-hemodialysis  

Repotrectinib

• Drug: Repotrectinib
• Trade name: Augtyro    
• Therapeutic Area: Non-small-cell lung cancer MeSH D002289
• Manufacturer: Bristol-Myers Squibb
• FDA Approval Date: November 15, 2023
• Classification: Small molecule
• Drug Class: Kinase inhibitor
• Clinical Trials:      https://www.app.pharmakb.com/drug-report/repotrectinib #clinical
• Adverse Events: e.g.  https://www.app.pharmakb.com/drug-report/repotrectinib #safety
• Description: Repotrectinib, sold under the trade name Augtyro, is used to treat locally advanced or metastatic ROS1-positive non-small cell lung cancer. It is taken by mouth.
• FDA News Release: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-repotrectinib-ros1-positive-non-small-cell-lung-cancer#:~:text=On%20November%2015%2C%202023%2C%20the,cell%20lung%20cancer%20(NSCLC).

Efbemalenograstim alfa

• Drug: Efbemalenograstim alfa
• Trade name: Ryzneuta    
• Therapeutic Area: Neutropenia MeSH D009503
• Manufacturer: Archotech Biopharma
• FDA Approval Date: November 16, 2023
• Classification: Molecule
• Drug Class: Colony-stimulating factor
• Clinical Trials:     https://www.app.pharmakb.com/drug-report/efbemalenograstimalfa#clinical
• Adverse Events: https://www.app.pharmakb.com/drug-report/efbemalenograstimalfa#safety
• Description: Efbemalenograstim alfa, sold under the trade name Ryzneuta, is used to decrease the incidence of infection, as manifested by febrile neutropenia, in adult patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with a clinically significant incidence of febrile neutropenia. It is administered subcutaneously.
• FDA News Release: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761134s000lbl.pdf  

Capivasertib

• Drug: Capivasertib
• Trade name: Truqap    
• Therapeutic Area: Breast cancer MeSH D001943
• Manufacturer: AstraZeneca
• FDA Approval Date: November 16, 2023
• Classification: Small molecule
• Drug Class: Serine/threonine kinase inhibitors
• Clinical Trials:     https://www.app.pharmakb.com/drug-report/capivasertib#clinical
• Adverse Events: e.g.  https://www.app.pharmakb.com/drug-report/capivasertib#safety
• Description: Capivasertib, sold under the brand name Truqap, is an anti-cancer medication used with fulvestrant for adult patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative locally advanced or metastatic breast cancer with one or more PIK3CA/AKT1/PTEN-alterations, as detected by an FDA-approved test, following progression on at least one endocrine-based regimen in the metastatic setting or recurrence on or within 12 months of completing adjuvant therapy used for the treatment of breast cancer. It is taken by mouth.
• FDA News Release: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-capivasertib-fulvestrant-breast-cancer#:~:text=On%20November%2016%2C%202023%2C%20the,one%20or%20more%20PIK3CA%2FAKT1%2F

Nirogacestat

• Drug: Nirogacestat
• Trade name: Ogsiveo    
• Therapeutic Area: Aggressive fibromatosis MeSH D018222
• Manufacturer: SpringWorks Therapeutics
• FDA Approval Date: November 28, 2023
• Classification: Small molecule
• Drug Class: Enzyme inhibitors: gamma secretase inhibitors
• Clinical Trials:   https://www.app.pharmakb.com/drug-report/nirogacestat#clinical
• Adverse Events: e.g.  https://www.app.pharmakb.com/drug-report/nirogacestat #safety
• Description: Nirogacestat, sold under the brand name Ogsiveo, is an anti-cancer medication used for the treatment of desmoid tumors. It is a selective gamma secretase inhibitor that is taken by mouth.
• FDA News Release: https://www.fda.gov/news-events/press-announcements/fda-approves-first-therapy-rare-type-non-cancerous-tumors

December 2023 – Novel FDA Drug Approvals

Iptacopan

• Drug: Iptacopan
• Trade name: FABHALTA
• Therapeutic Area: Paroxysmal hemoglobinuria MeSH D006457
• Manufacturer: Novartis
• FDA Approval Date: December 5, 2023
• Classification: Small molecule
• Drug Class: Complement receptor antagonists/complement inhibitors
• Clinical Trials:     https://www.app.pharmakb.com/drug-report/iptacopan#clinical
• Adverse Events: e.g.  https://www.app.pharmakb.com/drug-report/iptacopan#safety
• Description: Iptacopan , sold under the brand name Fabhalta, is a complement factor B inhibitor used for the treatment of paroxysmal nocturnal hemoglobinuria. It is taken by mouth.
• FDA News Release: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/218276s000lbl.pdf

Birch triterpenes

• Drug: Birch triterpenes
• Trade name: Filsuvez
• Therapeutic Area: Epidermolysis bullosa MeSH D004820
• Manufacturer: Chiesi Global Rare Disease
• FDA Approval Date: December 18, 2023
• Classification: Isoprenoid
• Drug Class: COX-2 inhibitor
• Clinical Trials:     https://www.app.pharmakb.com/drug-report/birchtriterpenes#clinical  
• Adverse Events: https://www.app.pharmakb.com/drug-report/birchtriterpenes#safety
• Description: Birch triterpenes, sold under the Filsuvez, is used to treat wounds associated with dystrophic and junctional epidermolysis bullosa. It is administered topically.
• FDA News Release: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215064s000lbl.pdf

Eplontersen

• Drug: Eplontersen
• Trade name: Wainua    
• Therapeutic Area: Amyloidosis MeSH D000686
• Manufacturer: Ionis Pharmaceuticals
• FDA Approval Date: December 21, 2024
• Classification: Oligonucleotide
• Drug Class: Antisense oligonucleotides
• Clinical Trials:     https://www.app.pharmakb.com/drug-report/eplontersen#clinical
• Adverse Events: e.g.  https://www.app.pharmakb.com/drug-report/eplontersen#safety
• Description: Eplontersen, sold under the brand name Wainua, is a medication used for the treatment of transthyretin-mediated amyloidosis. It is a transthyretin-directed antisense oligonucleotide. It was developed to treat hereditary transthyretin amyloidosis by Ionis Pharmaceuticals and AstraZeneca. It is taken by mouth.
• FDA News Release: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/217388s000lbl.pdf