Advancing medical science and developing new medicinal therapies can come in many forms and from various innovators. In 2020, the forefront of this type of attention came from the accelerated research and development process of multiple COVID-19 vaccines.
However, other innovative therapies have also been developed in 2020 and in years prior. Many of these therapies are considered to be novel drugs.
Novel drugs are prime examples of continuous advancement and innovation in the world of pharmacy practice in more recent years, and they are spotlighted here.
What is a novel drug?
Novel drugs, also referred to as new molecular entities or new compounds, are entities that are dissimilar from any previously-approved United States Food and Drug Administration (FDA) drug product. These drugs are innovative products that offer a new method of treating certain medical conditions. An official description is quoted below from the FDA.
“Certain drugs are classified as new molecular entities (“NMEs”) for purposes of FDA review. Many of these products contain active moieties that have not been approved by FDA previously, either as a single ingredient drug or as part of a combination product; these products frequently provide important new therapies for patients.” – United States Food and Drug Administration (FDA)
Novel drugs are typically created to help treat complex and/or medical conditions. For example, some of the more recent novel drug approvals in 2020 were approved to treat Ebola, advanced prostate cancer, HER2+ breast cancer, actinic keratosis of the face or scalp, hereditary angioedema, neuroblastomas, hyperoxaluria type 1, spinal muscular atrophy, and countless others. Patients with medical condition(s) that may be undertreated or untreated, such as these, rely on innovators to produce novel drugs that can help to more optimally treat their medical condition(s).
However, there are many different categories of drug approvals that the FDA performs. Novel drug approvals are only one of these categories. For a comparison of novel drugs to other drug types, please reference Table 1.
Table 1. Example FDA Drug Categorizations (Not All-Inclusive)
| Drug Type | Description |
| Novel Drugs | This category is the focus of this article. Novel drugs generally are new chemical entities/structures that are dissimilar from any previously-approved FDA drug product. These drugs are innovative products (or products with an innovative delivery/administration mechanism) that offer a new method of treating certain indicated medical conditions. Drugs that are considered to be novel drugs can also fall into other buckets mentioned below (i.e. biologics, brand-name drugs, etc). |
| Biologics | Biologic products are a diverse category of medicines and they are generally large, complex molecules. Biologics can be a virus, therapeutic serum, vaccine, blood component or derivative, allergenic product, protein, toxin, a trivalent organic arsenic compound, etc., applicable to the prevention, treatment, or cure of a disease or condition of humans. |
| Emergency Use Authorization Therapies (EUA) | An EUA is a mechanism to facilitate the availability and use of certain medicines, including vaccines, during public health emergencies. Under an EUA, the FDA may allow the use of unapproved medical products, or unapproved uses of approved medical products when certain criteria have been met, including that there are no adequate, approved, and available alternatives to help treat the public health emergency at-hand. |
| Brand Name Drugs | Prescription drugs that are marketed with a specific brand name by the company that manufactures it (which is usually the company which develops and patents it). When patents run out, generic versions of many popular drugs are marketed at lower cost by other companies. |
| Generic Name Drugs | A generic drug is a medication created to be the same as an existing approved brand-name drug in dosage form, safety, strength, route of administration, quality, and performance characteristics. Generic medicines work the same as brand-name medicines. However, a generic drug may have certain minor differences from the brand-name product, such as different inactive ingredients. |
| Authorized Generic Drugs | An authorized generic drug is the same as the brand-name drug but does not use the brand name on the label; it is also slightly different from a traditional generic drug. An authorized generic is considered to be therapeutically equivalent to its brand-name drug because it is the same drug. Reference: FDA Listing of Current Authorized Generics (last updated 28 December 2020) |
| Nonprescription Drugs | Also known as over-the-counter (OTC) drugs, these are products that are considered by the FDA to be safe and effective for use by the general public without seeking treatment by a health professional |
To help quantify how many novel drugs have been approved on an annual basis between 2011 and 2020, reference Image 1. For a listing of medical advances in the form of novel drug approvals for the last half-decade, reference Table 2.
Image 1: FDA Center for Drug Evaluation and Research (CDER) Novel Drug Approvals Between 2011 and 2020
Disclaimer: Table 2 only includes products reviewed by the FDA Center for Drug Evaluation and Research (CDER). This listing does not contain vaccines, allergenic products, blood and blood products, plasma derivatives, cellular and gene therapy products, or other products approved by the FDA Center for Biologics Evaluation and Research (CBER).
Table 2. Novel Drug Approvals
| Year | Approval Quantity | List of Novel Drugs Brand Name (Generic Name) |
| 2015 | 45 | Zurampic (lesinurad) Uptravi (selexipag) Bridion (sugammadex) Alecensa (alectinib) Kanuma (sebelipase alfa) Empliciti (elotuzumab) Portrazza (necitumumab) Ninlaro (ixazomib) Darzalex (daratumumab) Tagrisso (osimertinib) Cotellic (cobimetinib) Genvoya (elvitegravir, cobicistat, emtricitabine, and tenofovir alafenamide) Nucala (mepolizumab) Strensiq (asfotase alfa) Yondelis (trabectedin) Veltassa (patiromer for oral suspension) Praxbind (idarucizumab) Aristada (aripiprazole lauroxil) Tresiba (insulin degludec) Lonsurf (trifluridine and tipiracil) Vraylar (cariprazine) Xuriden (uridine triacetate) Varubi (rolapitant) Repatha (evolocumab) Addyi (flibanserin) Daklinza (daclatasvir) Odomzo (sonidegib) Praluent (alirocumab) Rexulti (brexpiprazole) Entresto (sacubitril/valsartan) Orkambi (lumacaftor 200 mg/ivacaftor 125 mg) Kengreal (cangrelor) Viberzi (eluxadoline) Kybella (deoxycholic acid) Corlanor (ivabradine) Cholbam (cholic acid) Unituxin (dinutuximab) Cresemba (isavuconazonium sulfate) Avycaz (ceftazidime-avibactam) Farydak (panobinostat) Lenvima (lenvatinib) Ibrance (palbociclib) Natpara (parathyroid horomone) Cosentyx (secukinumab) Savaysa (edoxaban) |
| 2016 | 22 | Spinraza (nusinersen) Rubraca (rucaparib) Eucrisa (crisaborole) Zinplava (bezlotoxumab) Lartruvo (olaratumab) Exondys 51 (eteplirsen) Adlyxin (lixisenatide) Xiidra (lifitegrast ophthalmic solution) Epclusa (sofosbuvir and velpatasvir) NETSPOT (gallium Ga 68 dotatate) Axumin (fluciclovine F 18) Ocaliva (obeticholic acid) Zinbryta (daclizumab) Tecentriq (atezolizumab) Nuplazid (pimavanserin) Venclexta (venetoclax) Defitelio (defibrotide sodium) Cinqair (reslizumab) Taltz (ixekizumab) Anthim (obiltoxaximab) Briviact (brivaracetam) Zepatier (elbasvir and grazoprevir) |
| 2017 | 46 | Giapreza (angiotensin II) Macrilen (macimorelin acetate) Steglatro (ertugliflozin) Rhopressa (netarsudil) Xepi (ozenoxacin) Ozempic (semaglutide) Hemlibra (emicizumab) Mepsevii (vestronidase alfa-vjbk) Fasenra (benralizumab) Prevymis (letermovir) Vyzulta (latanoprostene bunod ophthalmic solution) Calquence (acalabrutinib) Verzenio (abemaciclib) Solosec (secnidazole) Aliqopa (copanlisib) Benznidazole (benznidazole) Vabomere (meropenem and vaborbactam) Besponsa (inotuzumab ozogamicin) Mavyret (glecaprevir and pibrentasvir) Idhifa (enasidenib) Vosevi (sofosbuvir, velpatasvir and voxilaprevir) Nerlynx (neratinib maleate) Tremfya (guselkumab) Bevyxxa (betrixaban) Baxdela (delafloxacin) Kevzara (sarilumab) Radicava (edaravone) Imfinzi (durvalumab) Tymlos (abaloparatide) Rydapt (midostaurin) Alunbrig (brigatinib) Brineura (cerliponase alfa) Ingrezza (valbenazine) Austedo (deutetrabenazine) Ocrevus (ocrelizumab) Dupixent (dupilumab) Zejula (niraparib) Symproic (naldemedine) Bavencio (avelumab) Xadago (safinamide) Kisqali (ribociclib) Xermelo (telotristat ethyl) Siliq (brodalumab) Emflaza (deflazacort) Parsabiv (etelcalcetide) Trulance (plecanatide) |
| 2018 | 59 | Ultomiris (ravulizumab) Elzonris (tagraxofusp-erzs) Asparlas (calaspargase pegol-mknl) Motegrity (prucalopride) Xospata (gilteritinib) Firdapse (amifampridine) Vitrakvi (larotrectinib) Daurismo (glasdegib) Gamifant (emapalumab-lzsgemapalumab-lzsg) Aemcolo (rifamycin) Yupelri (revefenacin) Lorbrena (lorlatinib) Xofluza (baloxavir marboxil) Talzenna (talazoparib) Tegsedi (inotersen) Revcovi (elapegademase-lvlr) Nuzyra (omadacycline) Seysara (sarecycline) Libtayo (cemiplimab-rwlc) Vizimpro (dacomitinib) Emgality (galcanezumab-gnlm) Copiktra (duvelisib) Ajovy (fremanezumab-vfrm) Lumoxiti (moxetumomab pasudotox-tdfk) Pifeltro (doravirine) Xerava (eravacycline) Takhzyro (lanadelumab) Oxervate (cenegermin-bkbj) Diacomit (stiripentol) Galafold (migalastat) Annovera (segesterone acetate and ethinyl estradiol vaginal system) Onpattro (patisiran) Poteligeo (mogamulizumab-kpkc) Mulpleta (lusutrombopag) Omegaven (fish oil triglycerides) Orilissa (elagolix sodium) Krintafel (tafenoquine) Tibsovo (ivosidenib) TPOXX (tecovirimat) Braftovi (encorafenib) Mektovi (binimetinib) Zemdri (plazomicin) Epidioloex (cannabidiol) Moxidectin (moxidectin) Olumiant (baricitinib) Palynziq (pegvaliase-pqpz) Doptelet (avatrombopag) Lokelma (sodium zirconium cyclosilicate) Aimovig (erenumab-aooe) Lucemyra (lofexidine hydrochloride) Akynzeo (fosnetupitant and palonosetron) Crysvita (burosumab-twza) Tavalisse (fostamatinib) Ilumya (tildrakizumab) Trogarzo (ibalizumab-uiyk) Erleada (apalutamide) Symdeko (tezacaftor and ivacaftor) Biktarvy (bictegravir, emtricitabine, and tenofovir alafenamide) Lutathera (lutetium Lu 177 dotatate) |
| 2019 | 48 | Ubrelvy (ubrogepant) Enhertu (fam-trastuzumab deruxtecan-nxki) Dayvigo (lemborexant) Caplyta (lumateperone tosylate) TissueBlue (Brilliant Blue G Ophthalmic Solution) Padcev (enfortumab vedotin-ejfv) Vyondys 53 (golodirsen) Oxbryta (voxelotor) Xcopri (cenobamate) Givlaari (givosiran) Adakveo (crizanlizumab-tmca) Fetroja (cefiderocol) Brukinsa (zanubrutinib) Reblozyl (luspatercept–aamt) ExEm Foam (air polymer-type A) Trikafta (elexacaftor, ivacaftor, and tezacaftor) Reyvow (lasmiditan) Fluorodopa F 18 (fluorodopa F 18) Scenesse (afamelanotide) Beovu (brolucizumab–dbll) Aklief (trifarotene) Ibsrela (tenapanor) Nourianz (istradefylline) Ga-68-DOTATOC (Ga-68-DOTATOC) Xenleta (lefamulin) Rinvoq (upadacitinib) Inrebic (fedratinib) Rozlytrek (entrectinib) Wakix (pitolisant) Pretomanid (pretomanid) Turalio (pexidartinib) Nubeqa (darolutamide) Accrufer (ferric maltol) Recarbrio (imipenem, cilastatin and relebactam) Xpovio (selinexor) Vyleesi (bremelanotide) Polivy (polatuzumab vedotin-piiq) Piqray (alpelisib) Vyndaqel (tafamidis meglumine) Skyrizi (risankizumab-rzaa) Balversa (erdafitinib) Evenity (romosozumab-aqqg) Mayzent (siponimod) Sunosi (solriamfetol) Zulresso (brexanolone) Egaten (triclabendazole) Cablivi (caplacizumab-yhdp) Jeuveau (prabotulinumtoxinA-xvfs) |
| 2020 | 53 | Gemtesa (vibegron) Ebanga (ansuvimab-zykl)Orgovyx (relugolix) Margenza (margetuximab (anti-HER2 mAb)) Klisyri (tirbanibulin) Orladeyo (berotralstat) Gallium 68 PSMA-11 (Gallium 68 PSMA-11) Danyelza (naxitamab-gqgk)Imcivree (setmelanotide) Oxlumo (lumasiran) Zokinvy (lonafarnib) Veklury (remdesivir) Inmazeb (atoltivimab, maftivimab, and odesivimab-ebgn) Gavreto (pralsetinib) Detectnet (copper Cu 64 dotatate injection) Sogroya (somapacitan-beco) Winlevi (clascoterone) Enspryng (satralizumab-mwge) Viltepso (viltolarsen) Olinvyk (oliceridine) Evrysdi (risdiplam) Lampit (nifurtimox) Blenrep (belantamab mafodotin-blmf) Monjuvi (tafasitamab-cxix) Xeglyze (abametapir) Inqovi (decitabine and cedazuridine) Rukobia (fostemsavir) Byfavo (remimazolam) Dojolvi (triheptanoin) Zepzelca (lurbinectedin) Uplizna (inebilizumab-cdon) Tauvid (flortaucipir F18) Artesunate (artesunate) Cerianna (fluoroestradiol F18) Qinlock (ripretinib) Retevmo (selpercatinib) Tabrecta (capmatinib) Ongentys (opicapone) Trodelvy (sacituzumab govitecan-hziy) Pemazyre (pemigatinib) Tukysa (tucatinib) Koselugo (selumetinib) Zeposia (ozanimod) Isturisa (osilodrostat) Sarclisa (isatuximab) Nurtec ODT (rimegepant) Barhemsys (amisulpride) Vyepti (eptinezumab-jjmr) Nexletol (bempedoic acid) Pizensy (lactitol) Tazverik (tazemetostat) Tepezza (teprotumumab-trbw) Ayvakit (avapritinib) |
Related links:
- FDA 2015 Novel Drugs Summary
- FDA 2016 Novel Drugs Summary
- FDA 2017 Novel Drugs Summary
- FDA 2018 Novel Drugs Summary
- FDA 2019 Novel Drugs Summary
- FDA 2020 Novel Drugs Summary
References:
- New Drugs at FDA: CDER’s New Molecular Entities and New Therapeutic Biological Products. United States Food and Drug Administration (FDA). Last updated 8 January 2021. Accessed 10 January 2021. Accessible via: https://www.fda.gov/drugs/development-approval-process-drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products
- Biological Product Definitions. United States Food and Drug Administration (FDA). Accessed 9 January 2021. Accessible via: https://www.fda.gov/files/drugs/published/Biological-Product-Definitions.pdf
- Drug Applications for Over-the-Counter (OTC) Drugs. United States Food and Drug Administration (FDA). Last updated 31 March 2020. Accessed 9 January 2021. Accessible via: https://www.fda.gov/drugs/types-applications/drug-applications-over-counter-otc-drugs#:~:text=Over%2Dthe%2Dcounter%20
- FDA List of Authorized Generic Drugs. United States Food and Drug Administration (FDA). Last updated 28 December 2021. Accessed 10 January 2021. Accessible via: https://www.fda.gov/drugs/abbreviated-new-drug-application-anda/fda-list-authorized-generic-drugs
- Generic Drug Facts. United States Food and Drug Administration (FDA). Last updated 1 June 2018. Accessed 10 January 2021. Accessible via: https://www.fda.gov/drugs/generic-drugs/generic-drug-facts#:~:text=A%20generic%20drug%20is%20a,%2C%20quality%2C%20and%20performance%20characteristics.
About The Author
