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New Drugs Approved in 2021

Published:February 27, 2022DOI:https://doi.org/10.1016/j.amjmed.2022.01.055

      Abstract

      In 2021, the US Food and Drug Administration (FDA) approved 50 novel drugs. Thirty-seven of the 50 (74%) novel drug approvals were reviewed and approved through an expedited review pathway, and 26 of the 50 (52%) were approved for treatment of a rare disease. This review includes a summary of the novel drugs approved by the FDA in 2021.

      Keywords

      Clinical Significance
      • This summary of novel drugs approved by the US Food and Drug Administration in 2021 provides clinicians with pertinent prescribing information for each new drug.
      • Clinicians will find this information useful when discussing these new drugs with their patients, who may request them as part of their care.
      • This information may be useful as clinicians work with hospitals and other health care organizations that are considering addition of these drugs to their prescribing formularies.
      In 2021, the US Food and Drug Administration (FDA) approved 50 novel drugs.

      US Food and Drug Administration (FDA). Novel drug approvals for 2021. Available at:https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2021. Accessed January 5, 2022.

      Several expedited review pathways, including accelerated approval, priority review, fast track, and breakthrough therapy, allow for approval processes that allow drugs to be available earlier than had they undergone review in the traditional pathway. Ultimately, this allows for expedited approval and availability of drugs that treat a serious condition or fill an unmet medical need. Of the novel drug approvals in 2021, 37 of the 50 (74%) drugs were processed via one of the expedited review pathways, and 26 of the 50 (52%) were approved for treatment of a rare disease.

      US Food and Drug Administration (FDA). New drug therapy approvals 2021. Available at: https://www.fda.gov/media/155227/download. Accessed January 7, 2022.

      The 50 novel drugs approved in 2021 are summarized in the Table.
      TableSummary of 50 Novel Drugs Approved in 2021
      Brand (Generic)IndicationClinical PearlsPackage Insert
      Cardiology
      Verquvo (vericiguat)To reduce cardiovascular death and heart failure hospitalization in adults with symptomatic chronic HF and EF <45%Contraindicated in pregnancyhttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214377s000lbl.pdf
      Dermatology
      Adbry (tralokinumab-ldrm)Atopic dermatitisMonitor for hypersensitivity reactions or conjunctivitishttps://www.leo-pharma.us/Files/Billeder/US%20Website%20Product%20PIs/AdbryPI.pdf
      Bylvay (odevixibat)PruritusMay increase the risk hepatotoxicityhttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/215498s000lbl.pdf
      Korsuva (difelikefalin)Pruritus with chronic kidney disease undergoing hemodialysisMay cause somnolencehttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214916s000lbl.pdf
      Livmarli (maralixibat)Cholestatic pruritus associated with Alagille syndromeMay increase the risk of hepatotoxicity or gastrointestinal adverse reactionshttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214662s000lbl.pdf
      Endocrine
      Kerendia (finerenone)Reduction of kidney and heart complication in chronic kidney disease associated with type 2 diabetesCheck for drug–drug interactionshttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/215341s000lbl.pdf
      Skytrofa (lonapegsomatropin-tcgd)Short stature due to inadequate endogenous growth hormoneMonitor for hypersensitivity reactions or intracranial hypertensionhttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761177Orig1s000lbl.pdf
      Voxzogo (vosoritide)AchondroplasiaNot recommended in renal impairmenthttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214938s000lbl.pdf
      Zegalogue (dasiglucagon)Severe hypoglycemiaCheck for drug–drug interactionshttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214231s000lbl.pdf
      Genetic
      Amondys 45 (casimersen)Duchenne muscular dystrophyMay cause nephrotoxicityhttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/213026lbl.pdf
      Evkeeza (evinacumab-dgnb)Homozygous familial hypercholesterolemiaMay cause hypersensitivity reactionshttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761181s000lbl.pdf
      Leqvio (inclisiran)Heterozygous familial hypercholesterolemia or clinical atherosclerotic cardiovascular diseaseThe effect on cardiovascular morbidity and mortality has not been determinedhttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214012lbl.pdf
      Nexviazyme (avalglucosidase alfa-ngpt)Late-onset Pompe diseaseRisk of acute cardiorespiratory failurehttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761194s000lbl.pdf
      Nulibry (fosdenopterin)Molybdenum cofactor deficiency type AMay cause photosensitivityhttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214018s000lbl.pdf
      Welireg (belzutifan)von Hippel-Lindau diseaseMay cause anemia or hypoxiahttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/215383s000lbl.pdf
      Hematology
      Besremi (ropeginterferon alfa-2b-njft)Polycythemia veraCheck for drug-drug interactionshttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761166s000lbl.pdf
      Empaveli (pegcetacoplan)Paroxysmal nocturnal hemoglobinuriaAvailable only through a restricted program called Empaveli Risk Evaluation and Mitigation Strategyhttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/215014s000lbl.pdf
      Rezurock (belumosudil)Chronic graft-versus-host diseaseCheck for drug-drug interactionshttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214783s000lbl.pdf
      Infectious diseases
      Brexafemme (ibrexafungerp)Vulvovaginal candidiasisContraindicated in pregnancyhttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214900s000lbl.pdf
      Cabenuva (cabotegravir and rilpivirine)HIVMonitor for allergic and post-injection reactions, increase in liver enzymes, and depressionhttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/212888s000lbl.pdf
      Fexinidazole (fexinidazole)TrypanosomiasisMay cause hepatotoxicity, neuropsychiatric adverse reactions, or QT prolongationhttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214429s000lbl.pdf
      Livtencity (maribavir)CytomegalovirusCheck for drug–drug interactionshttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/215596lbl.pdf
      Neurology
      Aduhelm (aducanumab-avwa)Alzheimer diseaseMay cause hypersensitivity reactionshttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761178s000lbl.pdf
      Ponvory (ponesimod)Relapsing forms of multiple sclerosisMay increase the risk of infections, bradyarrhythmias/AV conduction delays or macular edemahttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/213498s000lbl.pdf
      Qulipta (atogepant)Prevent episodic migrainesCheck for drug–drug interactionshttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/215206Orig1s000lbl.pdf
      Vyvgart (efgartigimod alfa-fcab)Myasthenia gravisDelay administration in patients with an active infectionhttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761195s000lbl.pdf
      OB/GYN
      Nextstellis (drospirenone and estetrol)To prevent pregnancyDecreased efficacy with body mass index ≥30 kg/m2https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214154s000lbl.pdf
      Oncology
      Cosela (trilacicilib)To mitigate chemotherapy-induced myelosuppression in small cell lung cancerMonitor for hypersensitivity reactions or pneumonitishttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214200s000lbl.pdf
      Cytalux (pafolacianine)Diagnostic agent for ovarian cancerUse only 5% dextrose injection for dilutionhttps://www.accessdata.fda.gov/drugsatfda_docs/label/2020/214907s000lbl.pdf
      Exkivity (mobocertinib)Non-small cell lung cancerMonitor for cardiac toxicity or pneumonitishttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/215310s000lbl.pdf
      Fotivda (tivozanib)Renal cell carcinomaMonitor for signs or symptoms of cardiac failurehttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/212904s000lbl.pdf
      Jemperli (dostarlimab-gxly)Endometrial cancerMay increase risk of immune-related adverse reaction in any organ system or tissuehttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761174s000lbl.pdf
      Lumakras (sotorasib)Non-small cell lung cancerMay increase the risk hepatotoxicity or pneumonitishttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214665s000lbl.pdf
      Pepaxto (melphalan flufenamide)Relapsed or refractory multiple myelomaMonitor for signs and symptoms of myelosuppressionhttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214383s000lbl.pdf
      Pylarify (piflufolastat F 18)Diagnostic agent for men with prostate cancerMay increase risk for image misinterpretationhttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214793s000lbl.pdf
      Rybrevant (amivantamab-vmjw)Subset of non-small cell lung cancerMonitor for signs and symptoms of infusion-related reactionshttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761210s000lbl.pdf
      Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn)Acute lymphoblastic leukemia or Lymphoblastic lymphomaMonitor for hypersensitivity reactions, hepatotoxicity, or pancreatitishttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761179s000lbledt.pdf
      Scemblix (asciminib)Chronic myeloid leukemiaMonitor for signs and symptoms of myelosuppression and cardiac toxicityhttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/215358s000Orig1lbl.pdf
      Tepmetko (tepotinib)Non-small cell lung cancerMay increase the risk hepatotoxicity or pneumonitishttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214096s000lbl.pdf
      Tivdak (tisotumab vedotin-tftv)Recurrent metastatic cervical cancerMonitor for signs and symptoms of peripheral neuropathyhttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761208Orig1s000lbledt.pdf
      Truseltiq (infigratinib)CholangiocarcinomaMay cause retinal pigment epithelial detachmenthttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214622s000lbl.pdf
      Ukoniq (umbralisib)Marginal zone lymphoma and follicular lymphomaMay increase the risk of infections, neutropenia or hepatotoxicityhttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/213176s000lbl.pdf
      Zynlonta (loncastuximab tesirine-lpyl)Certain types of relapsed or refractory diffuse large B-cell lymphomaMonitor for signs and symptoms of myelosuppression and cutaneous reactionshttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761196s000lbl.pdf
      Psychiatry
      Azstarys (serdexmethylphenidate and dexmethylphenidate)ADHDPretreatment screening for cardiac diseasehttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/212994s000lbl.pdf
      Lybalvi (olanzapine and samidorphan)Schizophrenia and bipolar type 1Check for drug–drug interactionshttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/213378s000lbl.pdf
      Qelbree (viloxazine)ADHDMay increase suicidal thoughts and behaviorhttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/211964s000lbl.pdf
      Respiratory
      Tezspire (tezepelumab-ekko)AsthmaDo not discontinue corticosteroids use abruptlyhttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761224s000lbl.pdf
      Rheumatology
      Lupkynis (voclosporin)Lupus nephritisMonitor for prolonged QT interval, nephrotoxicity, neurotoxicity, or hypertension. Contraindicated with strong CYP 3A4 inhibitors.https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/213716s000lbl.pdf
      Saphnelo (anifrolumab-fnia)Moderate to severe systemic lupus erythematosusMonitor for hypersensitivity reactions or serious infectionshttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761123s000lbl.pdf
      Tavneos (avacopan)Anti-neutrophil cytoplasmic autoantibody-associated vasculitisMay increase the risk of infections, HBV reactivation, or hepatotoxicityhttps://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214487s000lbl.pdf
      ADHD = attention-deficit/hyperactivity disorder; AV = atrioventricular; CYP = cytochrome P450; EF = ejection fraction; HBV = hepatitis B vaccine; HF = heart failure; HIV = human immunodeficiency virus; OB/GYN = Obstetrics/Gynecology.
      First-in-class is a classification indicating a mechanism of action different from existing therapies, occurring in 27 of the 50 (54%) approved drugs in 2021. One notable first-in-class example is Besremi (ropeginterferon alfa-2b-njft; PharmaEssentia USA, Burlington, Mass), indicated for treatment of polycythemia vera, a condition that causes the bone marrow to make too many red blood cells. Another first-in-class approval is LIVTENCITY (maribavir; Takeda Pharmaceuticals, Lexington, Mass), indicated for post-transplant cytomegalovirus infection that is refractory to treatment with ganciclovir, valganciclovir, cidofovir, or foscarnet. The therapeutic area with the most novel drug approvals (16 of 50) in 2021 was oncology. Lung cancer accounts for 5 of the new drug approvals.
      The FDA also approved biologicals in 2021, which are not considered drugs and not reviewed in detail here. Notable among the approved biologicals are PreHevbrio (hepatitis B vaccine [recombinant]; VBI Vaccines, Cambridge, Mass) for prevention of hepatitis B virus; Comirnaty (COVID-19 vaccine, mRNA; Pfizer, New York, NY) for prevention of coronavirus disease 2019; TICOVAC (Tick-Borne Encephalitis Vaccine; Pfizer, New York NY) for prevention of tick-borne encephalitis; VAXNEUVANCE (pneumococcal 15-valent conjugate vaccine; Merck, Whitehouse Station, NJ) for prevention of Streptococcus pneumonia; Prevnar 20 (pneumococcal 20-valent conjugate vaccine; Pfizer, New York, NY) for prevention of Streptococcus pneumonia; ABECMA (idecabtagene vicleucel; Bristol-Myers Squibb, Summit, NJ) for treatment of relapsed or refractory multiple myeloma; and Breyanzi (lisocabtagene maraleucel; Bristol-Myers Squibb, Summit, NJ) for treatment of relapsed or refractory large B-cell lymphoma.
      Moreover, the FDA approved 4 biosimilar drugs. Among these are Semglee (insulin glargine-yfgn; Mylan, Morgantown, WV), Rezvoglar (insulin glargine-aglr; Ely Lilly, Indianapolis, Ind), Byooviz (ranibizumab-nuna, Biogen, Cambridge, Mass), and Yusimry (adalimumab-aqvh; Coherus BioSciences, Redwood City, Calif).
      In 2017, the FDA announced the Drug Competition Action Plan to address the high drug costs through market competition of generic drugs. The 3 objectives were to improve the efficiency of the generic drug development, review, and approval process, maximize scientific and regulatory clarity, and close loopholes for brand-name drug companies that delay generic competition.

      US Food and Drug Administration (FDA). FDA drug competition action plan. Available at:https://www.fda.gov/drugs/guidance-compliance-regulatory-information/fda-drug-competition-action-plan. Accessed January 9, 2022.

      The Competitive Generic Therapy (CGT) designation qualifies a generic drug to receive an expedited development and review of its application.

      US Food and Drug Administration (FDA). FDA in brief: FDA reaches milestone in competitive generic therapy drug approvals. Available at:https://www.fda.gov/news-events/press-announcements/fda-brief-fda-reaches-milestone-competitive-generic-therapy-drug-approvals. Accessed January 5, 2022.

      Additionally, generic drug applications that receive a CGT designation may be eligible for a 180-day period of marketing exclusivity if it is the first approved product for that CGT and the product must be commercially marketed within 75 calendar days after the date of approval or the exclusivity will be forfeited.

      US Food and Drug Administration (FDA). FDA in brief: FDA reaches milestone in competitive generic therapy drug approvals. Available at:https://www.fda.gov/news-events/press-announcements/fda-brief-fda-reaches-milestone-competitive-generic-therapy-drug-approvals. Accessed January 5, 2022.

      This marketing exclusivity blocks approval of competitive abbreviated new drug applications, but only begins when the first CGT product is marketed. This provides an incentive to market the CGT quickly after it is first approved. Of the 80 first-time generic drug approvals in 2021, 51 received the CGT designation.

      US Food and Drug Administration (FDA). First generic drug approvals. Available at: https://www.fda.gov/drugs/drug-and-biologic-approval-and-ind-activity-reports/first-generic-drug-approvals. Accessed January 5, 2022.

      ,

      US Food and Drug Administration (FDA). First competitive generic therapy approvals. Available at: https://www.fda.gov/drugs/generic-drugs/competitive-generic-therapy-approvals. Accessed January 5, 2022.

      Since the program's inception, 119 generic drugs received the CGT designation.

      References

      1. US Food and Drug Administration (FDA). Novel drug approvals for 2021. Available at:https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2021. Accessed January 5, 2022.

      2. US Food and Drug Administration (FDA). New drug therapy approvals 2021. Available at: https://www.fda.gov/media/155227/download. Accessed January 7, 2022.

      3. US Food and Drug Administration (FDA). FDA drug competition action plan. Available at:https://www.fda.gov/drugs/guidance-compliance-regulatory-information/fda-drug-competition-action-plan. Accessed January 9, 2022.

      4. US Food and Drug Administration (FDA). FDA in brief: FDA reaches milestone in competitive generic therapy drug approvals. Available at:https://www.fda.gov/news-events/press-announcements/fda-brief-fda-reaches-milestone-competitive-generic-therapy-drug-approvals. Accessed January 5, 2022.

      5. US Food and Drug Administration (FDA). First generic drug approvals. Available at: https://www.fda.gov/drugs/drug-and-biologic-approval-and-ind-activity-reports/first-generic-drug-approvals. Accessed January 5, 2022.

      6. US Food and Drug Administration (FDA). First competitive generic therapy approvals. Available at: https://www.fda.gov/drugs/generic-drugs/competitive-generic-therapy-approvals. Accessed January 5, 2022.