Novel FDA Drug Approvals - 2nd 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 | April | May | June

April 2023 – Novel FDA Drug Approvals

Tofersen

• Drug: Tofersen
• Trade name: Qalsody
• Therapeutic Areas: amyotrophic lateral sclerosis (ALS) MeSH D000690
• Manufacturer: Biogen
• FDA Approval Date: April 25, 2023 (Abbreviated New Drug Application)
• Classification: Oligonucleotide
• Drug Class: Antisense oligonucleotides
• Clinical Trials: https://app.pharmakb.com/drugs/Tofersen#clinical
• Adverse Events: https://app.pharmakb.com/drugs/Tofersen#safety
• Description: Tofersen, sold under the brand name Qalsody, is a medication used for the treatment of amyotrophic lateral sclerosis (ALS). Tofersen is an antisense oligonucleotide that targets the production of superoxide dismutase 1, an enzyme whose mutant form is commonly associated with ALS. It is administered as an intrathecal injection into the spinal cord.
• FDA News Release: https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-treatment-amyotrophic-lateral-sclerosis-associated-mutation-sod1-gene

May 2023 – Novel FDA Drug Approvals

Pegunigalsidase alfa  

• Drug: Pegunigalsidase alfa
• Trade name: Elfabrio
• Therapeutic Areas: Fabry disease MeSH 000795
• Manufacturer: Protalix BioTherapeutics
• FDA Approval Date: May 9, 2023
• Classification: Enzyme
• Drug Class: Enzymes; PEGylated compounds, covalent attachment of macrogol (polyethylene glycol) polymer
• Clinical Trials: https://app.pharmakb.com/drugs/Pegunigalsidase%20alfa#clinical
• Adverse Events: https://app.pharmakb.com/drugs/Pegunigalsidase%20alfa#safety
• Description: Pegunigalsidase alfa, sold under the trade name Elfabrio, is an experimental medication under investigation as an enzyme replacement therapy for the treatment of Fabry disease. It is a recombinant human α-galactosidase-A.
• FDA News Release: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761161s000lbl.pdf

Fezolinetant

• Drug: Fezolinetant
• Trade name: Veozah  
• Therapeutic Areas: Hot flashes MeSH D019584
• Manufacturer: Astellas Pharma
• FDA Approval Date: May 12, 2023
• Classification: Small molecule
• Drug Class: tachykinin (neurokinin) receptor antagonists: NK3 receptor antagonists
• Clinical Trials: https://app.pharmakb.com/drugs/Fezolinetant#clinical
• Adverse Events: https://app.pharmakb.com/drugs/Fezolinetant#safety
• Description: Fezolinetant (INN; former developmental code name ESN-364), sold under the trade name Veozah, is a small-molecule, orally active, selective neurokinin-3 (NK3) receptor antagonist which is under development by for the treatment of sex hormone-related disorders. It is developed by Astellas Pharma which acquired it from Ogeda (formerly Euroscreen) in April 2017.
• FDA News Release: https://www.fda.gov/news-events/press-announcements/fda-approves-novel-drug-treat-moderate-severe-hot-flashes-caused-menopause

Epcoritamab

• Drug: Epcoritamab
• Trade name: Epkinly  
• Therapeutic Areas: Large b-cell lymphoma diffuse MeSH D016403; follicular lymphoma D008224
• Manufacturer: Genmab A/S
• FDA Approval Date: May 19, 2023 (FDA accelerated approval)
• Classification: Antibody
• Drug Class: monoclonal antibodies
• Clinical Trials: https://app.pharmakb.com/drugs/Epcoritamab#clinical
• Adverse Events: https://app.pharmakb.com/drugs/Epcoritamab#safety
• Description: Epcoritamab, sold under the trade name Epkinly, is an antibody pharmaceutical. It is currently being investigated in clinical studies. It is known to target T-cell surface glycoprotein CD3 epsilon chain and B-lymphocyte antigen CD20.
• FDA News Release: https://www.fda.gov/drugs/drug-approvals-and-databases/fda-grants-accelerated-approval-epcoritamab-bysp-relapsed-or-refractory-diffuse-large-b-cell

Sulbactam

• Drug: Sulbacta
• Trade name: Xacduro
• Therapeutic Areas: Infections MeSH D007239
• Manufacturer: Entasis Therapeutics
• FDA Approval Date: May 23, 2023
• Classification: Small molecule
• Drug Class: Beta-lactamase inhibitors
• Clinical Trials: https://app.pharmakb.com/drugs/Sulbactam#clinical
• Adverse Events: https://app.pharmakb.com/drugs/Sulbactam#safety
• Description: Sulbactam, sold under the trade name of Xacduro, is used to treat hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia caused by susceptible isolates of Acinetobacter baumannii-calcoaceticus complex.
• FDA News Release: https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-pneumonia-caused-certain-difficult-treat-bacteria.

Nirmatrelvir, ritonavir

• Drug: Nirmatrelvir, ritonavir
• Trade name: Paxlovid  
• Therapeutic Areas: Covid-19 MeSH D000086382; infections MeSH D007239; digestive system diseases MeSH D004066; urogenital diseases MeSH D000091642
• Manufacturer: Pfizer
• FDA Approval Date: May 25, 2023
• Classification: Small molecule
• Drug Class: Antivirals: HIV protease inhibitors (saquinavir type)
• Clinical Trials: https://app.pharmakb.com/drugs/Ritonavir#clinical
• Adverse Events: https://app.pharmakb.com/drugs/Ritonavir#safety
• Description: Ritonavir is an L-valine derivative that is L-valinamide in which alpha-amino group has been acylated by a [(2-isopropyl-1,3-thiazol-4-yl)methyl]methylcarbamoyl group and in which a hydrogen of the carboxamide amino group has been replaced by a (2R,4S,5S)-4-hydroxy-1,6-diphenyl-5-{[(1,3-thiazol-5-ylmethoxy)carbonyl]amino}hexan-2-yl group. A CYP3A inhibitor and antiretroviral drug from the protease inhibitor class used to treat HIV infection and AIDS, it is often used as a fixed-dose combination with another protease inhibitor, lopinavir. Also used in combination with dasabuvir sodium hydrate, ombitasvir and paritaprevir (under the trade name Viekira Pak) for treatment of chronic hepatitis C virus genotype 1 infection as well as cirrhosis of the liver. It has a role as an antiviral drug, a HIV protease inhibitor, an environmental contaminant and a xenobiotic. It is a member of 1,3-thiazoles, a L-valine derivative, a carbamate ester, a member of ureas and a carboxamide.
• FDA News Release: https://www.fda.gov/news-events/press-announcements/fda-approves-first-oral-antiviral-treatment-covid-19-adults

Flotufolastat F 18

• Drug: piflufolastat f-18
• Trade name: Posluma
• Therapeutic Areas: PET imaging agent
• Manufacturer: Blue Earth Diagnostics
• FDA Approval Date: May 25, 2023
• Classification: Small molecule
• Drug Class: Enzyme inhibitors: folate hydrolose (also called Prostate Specific Membrane Antigen (PSMA) or glutamate carboxypeptidase II) inhibitors.
• Clinical Trials: https://app.pharmakb.com/drugs/Piflufolastat%20f-18#clinical
• Adverse Events: https://app.pharmakb.com/drugs/Piflufolastat%20f-18#safety
• Description: Flotufolastat F 18, sold under the trade name of Posluma, is a radiohybrid (PET) prostate-specific membrane antigen (PSMA)-targeted positron emission tomography (PET)-imaging agent indicated for PSMA-positive lesions in men with prostate cancer with suspected metastasis who are candidates for initial definitive therapy or with suspected recurrence based on elevated serum prostate‐specific antigen PSA level.
• FDA News Release: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/216023s000lbl.pdf

Sotagliflozin

• Drug: sotagliflozin
• Trade name: Inpefa
• Therapeutic Areas: Heart failure MeSH D006333; Type 1 diabetes mellitus MeSH D003924
• Manufacturer: Lexicon Pharmaceuticals
• FDA Approval Date: May26, 2023
• Classification: Small molecule
• Drug Class: Sotagliflozin is an inhibitor of SGLT2 and SGLT1.
• Clinical Trials: https://app.pharmakb.com/drugs/Sotagliflozin#clinical
• Adverse Events: https://app.pharmakb.com/drugs/Sotagliflozin#safety
• Description: Sotagliflozin, sold under the trade name of Inpefa, is a sodium-glucose cotransporter 2 (SGLT2) inhibitor indicated to reduce the risk of cardiovascular death, hospitalization for heart failure, and urgent heart failure visit in adults with heart failure or type 2 diabetes mellitus, chronic kidney disease, and other cardiovascular risk factors. It is delivered orally.
• FDA News Release: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/216203s000lbl.pdf

June 2023 – Novel FDA Drug Approvals

Glofitamab-gxbm

• Drug: Glofitamab
• Trade name: Columvi
• Therapeutic Areas: Large B-cell lymphoma MeSH D009369
• Manufacturer: Genentech
• FDA Approval Date: June 15, 2023
• Classification: Antibody
• Drug Class: Monoclonal antibodies
• Clinical Trials: https://app.pharmakb.com/drugs/Glofitamab#clinical
• Adverse Events: https://app.pharmakb.com/drugs/Glofitamab#safety
• Description: Glofitamab, sold under the brand name Columvi, is a bispecific monoclonal antibody used for the treatment of diffuse large B-cell lymphoma. It is a bispecific CD20-directed CD3 T-cell engager.
• FDA News Release: https://www.fda.gov/drugs/drug-approvals-and-databases/fda-grants-accelerated-approval-glofitamab-gxbm-selected-relapsed-or-refractory-large-b-cell

Ritlecitinib

• Drug: Ritlecitinib
• Trade name: Litfulo
• Therapeutic Areas: Severe alopecia areata in adults. MeSH D000506
• Manufacturer: Pfizer
• FDA Approval Date: June 23, 2023
• Classification: Small molecule
• Drug Class: Small molecule kinase inhibitor
• Clinical Trials: https://app.pharmakb.com/drugs/Ritlecitinib#clinical
• Adverse Events: https://app.pharmakb.com/drugs/Ritlecitinib#safety
• Description: Ritlecitinib, sold under the trade name Litfulo, is a kinase inhibitor indicated for the treatment of severe alopecia areata in adults and adolescents 12 years and older.
• FDA News Release: https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2023/215830Orig1s000ltr.pdf

Rozanolixizumab

• Drug: Rozanolixizumab
• Trade name: Rystiggo
• Therapeutic Areas: Myasthenia gravis MeSH D009157
• Manufacturer: UCB
• FDA Approval Date: June 26, 2023    
• Classification: Antibody
• Drug Class: Monoclonal antibodies
• Clinical Trials: https://app.pharmakb.com/drugs/Rozanolixizumab#clinical
• Adverse Events: Rozahttps://app.pharmakb.com/drugs/Rozanolixizumab#clinicaltrials#safety
• Description: Rozanolixizumab-noli, sold under the trade name Rystiggo, is used to treat generalized myasthenia gravis in adults who are anti-acetylcholine receptor- or anti-muscle-specific tyrosine kinase antibody-positive.
• FDA News Release: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761286s000lbl.pdf

Somatrogon-ghla

• Drug: Somatrogon
• Trade name: Ngenla
• Therapeutic Areas: Physiological phenomena MeSH D010829
• Manufacturer: Pfizer
• FDA Approval Date: June 27, 2023
• Classification: Protein
• Drug Class: Growth hormone derivitives
• Clinical Trials: https://app.pharmakb.com/drugs/Somatrogon#clinical  
• Adverse Events: https://app.pharmakb.com/drugs/Somatrogon#safety  
• Description: Somatrogon, sold under the trade name Ngenla, is used to treat growth failure due to inadequate secretion of endogenous growth hormone.
• FDA News Release: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761184Orig1s000Corrected_lbl.pdf