FDA on a Roll, the Party Continues: Drug Approvals in 2018 (Part 2)

It was a golden year for the pharmaceutical industry in terms of FDA approvals. With 62 new molecular entity (NME) and biologic approvals in 2018, all records were broken to date (maximum being 59 in 1996).1,2 In the first part of this series, DRG observed that the FDA’s Center for Drug Evaluation and Research (CDER) had made, on average 32 approvals over the last ten years, with 46 approvals in 2017 and only 22 in 2016 (Figure 1). In H1 of 2018, 16 NMEs and 8 biologics license applications (BLAs) were approved by the FDA.3 With 42 NMEs and 20 biologics approved by CDER and the Center for Biologics Evaluation and Research (CBER), the FDA ended 2018 with flying colors before closing for Christmas (Tables 1 and 2). In total, 149 approvals have been made in all submission categories (type 1 to 10), including three BLA supplements.

It was no surprise that oncology topped the list with 18 approvals (Figure 1b) along with popularity of rare indications. Among the 62 approvals, 55% (34 NMEs and BLAs) were orphan designations; a tremendous increase from the past two years (39% for 2017 and 41% in 2016).4,5 The FDA continues to offer expedited review benefits to drugs, helping patients to gain quick access to much needed therapies. Among the 59 NMEs approved by CDER in 2018, 43 (73%) were designated to have either one or more of the expedited review categories - fast track, breakthrough, accelerated approval, and priority review. In 2018, 50% of drug approvals had priority review status. Many companies received approvals for their molecules. Pfizer was the only company to get four approvals (and in a span of three months). All four oncology products will strengthen its portfolio. A few other companies, such as AstraZeneca, Shionogi, Eli Lilly, and Array BioPharma, received approvals for two products each.

According to DRG estimates, eight of these molecules are expected to cross the US $ 1 billion mark onto the list of block busters. Of these molecules, five are biologics. In 2018 we saw the approvals of some major blockbusters including Gilead’s Biktarvy (bictegravir sodium, emtricitabine, and tenofovir alafenamide fumarate), a combination of integrase strand transfer inhibitor and nucleoside reverse transcriptase inhibitor, Janssen’s Erleada (apalutamide), an androgen receptor inhibitor, Eli Lilly’s Olumiant (baricitinib), a JAK inhibitor, and Regeneron/Sanofi’s Libtayo (cemiplimab), a fully human PD-1 monoclonal antibody (MAb). In addition to oncology drugs Erleada and Libtayo, two migraine drugs, Amgen/Novartis’s Aimovig (erenumab) and Eli Lilly’s Emgality (galcanezumab), are forecast to be block busters in three to five years.

Biktarvy, a once-daily, single-tablet option, is expected to be one of the biggest launches of the year and growth drivers for the HIV market. It will strongly compete with ViiV’s Triumeq (dolutegravir/abacavir/lamivudine). The improved long-term renal and bone safety seen with tenofovir alafenamide fumarate and the absence of a booster make Biktarvy a safer alternative to its competitors.

Erleada, the first FDA-approved therapy for nonmetastatic castration-resistant prostate cancer (nmCRPC) is expected to strengthen J&J’s position in the prostate cancer market. The drug exhibits a strong efficacy benefit and has been added to the NCCN guidelines as a category 1 recommendation for treatment of nmCRPC, which will significantly influence treatment of this disease.

Olumiant, the second JAK inhibitor in the U.S. market, was approved approximately six years after Pfizer’s Xeljanz (tofacitinib) for moderate to severe rheumatoid arthritis. With a price that is about 60% of Humira and the wide use of JAK inhibitors in inflammatory diseases, Olumiant is expected to become a blockbuster.

Libtayo, a fully human MAb targeting the immune checkpoint receptor PD-1, was approved in September by the FDA for the treatment of patients with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or radiation. This drug is the first approved treatment for advanced CSCC, the second most popular form of skin cancer. Results from both the phase II EMPOWER-CSCC-1 clinical trial and a Phase I trial led to its getting priority review. Additionally, Libtayo is under development for various advanced malignancies, such as NSCLC, solid tumors, and hematological cancers, either alone or in combination.

Aimovig, an anti-CGRP MAb, is the first therapy to be developed for migraine prevention and is a promising option for patients who cannot find relief or tolerate the side effects of currently available therapies. Apart from having first-mover advantage, the drug also differentiates from others by targeting the CGRP receptor rather than the CGRP molecule and thus is forecast to be a block buster. Aimovig offers a clean side-effect profile free from any warnings, precautions, and contraindications and can be self-administered with SureClick® autoinjector. Both Amgen and Novartis have made plans to ease the burden on patients as the drug faces access and reimbursement issues in the United States.

Emgality is a humanized MAb and CGRP antagonist that in addition to being approved for migraine prophylaxis is in development for cluster headache. The drug is also well tolerated and has a clean side effect profile, with no warnings or contraindications on its label. Like Aimovig, Emgality faces payer restrictions but Eli Lilly is offering patient-assistance programs for this drug. It can also be self-administered by patients at home once a month.

The increasing number of approvals raises the question of whether deregulation, process improvement, or higher risk-taking by companies leads to faster approvals. The FDA is certainly taking measures to bring medicines as quickly as possible to patients. In 2018, 42 of the 59 novel drugs approved (71 %) got their first approval in the United States before receiving approval in any other country. FDA’s CDER is now working more closely with companies, especially during designing their clinical studies and building NDAs for submission. This helps the companies to get approvals in the first cycle (6 to 12 months) of the approval process itself. In 2018, 95% of the novel drugs got approval in the first cycle, giving quick access of new drugs to the patients.6

A large number of expedited reviews, orphan drug designations, and approvals of high-priced drugs for rare indications are some of the trends shaping the future of pharmaceutical industry. Factors such as advancements in diagnostic techniques, investment in research in genomics, proteomics, and mapping of the human genome, and conducting biomarker-enriched clinical trials are going to increase precision-based drug approvals in future. The FDA is expected to bring in new innovative initiatives focusing on efficient and well-informed approvals, in a timely manner to bring safe and effective therapies to patients.




Table 1. NMEs Approved in 20181

S.No.Approval DateDrug NameActive IngredientsCompanyIndicationTherapy AreaReview Priority
11/26/2018Lutathera Lutetium dotatate Lu-177AAA USATreatment of somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including foregut, midgut, and hindgut neuroendocrine tumors in adults.OncologyPriority, Orphan
22/7/2018Biktarvy Bictegravir sodium; emtricitabine; tenofovir alafenamide fumarateGilead SciencesTreatment of HIV-1 infection in adults who have no antiretroviral treatment history or to replace the current antiretroviral regimen in those who are virologically suppressed (HIV-1 RNA less than 50 copies per mL) on a stable antiretroviral regimen for at least 3 months with no history of treatment failure and no known substitutions associated with resistance to the individual components of Biktarvy.AntiviralPriority
32/12/2018Symdeko (copackaged) Ivacaftor; tezacaftor/ ivacaftorVertex PharmaceuticalsTreatment of patients with cystic fibrosis (CF) aged 12 or older who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence.Rare disorder - respiratoryPriority, Orphan
42/14/2018Erleada ApalutamideJanssen BiotechTreatment of patients with nonmetastatic, castration resistant prostate cancer (nm-CRPC).OncologyPriority
54/17/2018Tavalisse Fostamatinib disodiumRigel PharmaceuticalsTreatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.Rare disorder - oncologyStandard, Orphan
64/19/2018Akynzeo Fosnetupitant chloride hydrochloride, palonosetron hydrochlorideHelsinn HealthcarePrevention of acute and delayed nausea and vomiting associated with initial and repeat courses of cancer chemotherapy, including, but not limited to, highly emetogenic chemotherapy.OncologyStandard
75/16/2018Lucemyra Lofexidine hydrochlorideUS WorldMedsMitigation of opioid withdrawal symptoms to facilitate abrupt opioid discontinuation in adults.CNSPriority
85/18/2018Lokelma Sodium zirconium cyclosilicateAstraZenecaTreatment of hyperkalemia in adultsMetabolic disorderStandard
95/21/2018Doptelet Avatrombopag maleateAkaRxTreatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure.Hematology disordersPriority, Orphan
105/31/2018Olumiant BaricitinibEli LillyTreatment of adult patients with moderately to severely active rheumatoid arthritis who have had an inadequate response to one or more TNF-antagonist therapies.Immune disorderStandard
116/13/2018Moxidectin MoxidectinMedicines Development for Global HealthTreatment of onchocerciasis due to Onchocerca volvulus in patients aged 12 or older.AnthelminticPriority, Orphan
126/25/2018Zemdri PlazomicinAchaogenTreatment of patients ≤ age 18 years with complicated urinary tract infections (cUTI) including Pyelonephritis caused by the following susceptible microorganism(s): Escherichia coli, Klebsiella pneumoniae, Proteus mirabilis, and Enterobacter cloacae.AntibacterialPriority
136/25/2018Epidiolex CannabidiolGW ResearchTreatment of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS) in patients aged 2 or older.CNSPriority, Orphan
146/27/2018Braftovi EncorafenibArray BioPharmaIndicated, in combination with binimetinib, for the treatment of patients with unresectable or metastatic melanoma with a BRAF V600E or V600K mutation, as detected by an FDA-approved test.OncologyStandard, Orphan
156/27/2018Mektovi BinimetinibArray BioPharmaIndicated, in combination with encorafenib, for the treatment of patients with unresectable or metastatic melanoma with a BRAF V600E or V600K mutation, as detected by an FDA-approved test.OncologyStandard, Orphan
167/13/2018TPOXXTecovirimatSiga TechnologiesTreatment of human smallpox disease caused by variola virus in adults and pediatric patients weighing at least 13 kgAntiviralPriority, Orphan
177/20/2018KrintafelTafenoquine succinateGlaxoSmithKlineRadical cure (prevention of relapse) of plasmodium vivax malaria in patients aged 16 or older who are receiving appropriate antimalarial therapy for acute P.vivax infection.Anti malarialPriority, Orphan
187/20/2018TibsovoIvosidenibAgios PharmaceuticalsTreatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test.OncologyPriority, Orphan
197/23/2018OrilissaElagolix sodiumAbbVieManagement of moderate to severe pain associated with endometriosis.PainPriority
207/27/2018OmegavenFish oil triglyceridesFresenius KabiSource of calories and fatty acids in pediatric patients with parenteral nutrition-associated cholestasis (PNAC).OtherPriority, Orphan
217/31/2018MulpletaLusutrombopagShionogiTreatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure.Hematology disordersPriority
228/10/2018GalafoldMigalastat hydrochlorideAmicus TherapeuticsTreatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant.RarePriority, Orphan
238/10/2018AnnoveraEthinyl estradiol, segesterone acetateTherapeuticsMDUse by females with reproductive potential to prevent pregnancy.Women’s healthStandard
248/10/2018OnpattroPatisiran sodiumAlnylam PharmaceuticalsTreatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.RarePriority, Orphan
258/20/2018DiacomitStiripentolBiocodexTreatment of seizures associated with Dravet syndrome (DS) in patients aged 2 years or older taking clobazam.CNSPriority, Orphan
268/27/2018XeravaEravacycline dihydrochlorideTetraphase PharmaceuticalsTreatment of complicated intra-abdominal infections (cIAI) caused by susceptible microorganismsAnti-infectivePriority
278/30/2018PifeltroDoravirineMSD MerckCombination with other antiretroviral agents for the treatment of HIV-1 infection in adult patients with no prior antiretroviral treatment history.Anti-viralStandard
289/24/2018CopiktraDuvelisibVerastemTreatment of adult patients with relapsed or refractory CLL or SLL after at least two prior therapies, treatment of adult patients with relapsed or refractory FL after at least two prior systemic therapies.OncologyPriority, Orphan
299/27/2018VizimproDacomitinibPfizerFirst-line treatment of patients with metastatic non-small-cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletion or exon 21 L858R substitution mutations as detected by an FDA-approved test.OncologyPriority, Orphan
3010/01/2018SeysaraSarecyclineAllerganTreatment of inflammatory lesions of non-nodular moderate to severe acne vulgaris in patients aged 9 years or older.DermatologyStandard
3110/02/2018NuzyraOmadacyclineParatek PharmsCommunity-acquired bacterial pneumonia (CABP), acute bacterial skin and skin structure infections (ABSSSI).Anti-infectivePriority
3210/05/2018TegsediInotersenIonis PharmaceuticalsTreatment of the polyneuropathy of hereditary transthyretinmediated amyloidosis in adults.RarePriority, Orphan
3310/16/2018TalzennaTalazoparibPfizerTreatment of adult patients with deleterious or suspected deleterious germline breast cancer susceptibility gene (BRCA)-mutated (gBRCAm) human epidermal growth factor receptor 2 (HER2)-negative locally advanced or metastatic breast cancer.OncologyPriority
3410/24/2018XofluzaBaloxavir marboxilShionogiTreatment of acute uncomplicated influenza in patients aged 12 years or older who have been symptomatic for no more than 48 hours.Anti-infectivePriority
3511/02/2018LorbrenaLorlatinibPfizerTreatment of patients with anaplastic lymphoma kinase (ALK)-positive, metastatic NSCLC.OncologyPriority, Orphan
3611/09/2018YupelriRevefenacinTheravance BiopharmaceuticalsMaintenance treatment of patients with chronic obstructive pulmonary disease (COPD)RespiratoryStandard
3711/16/2018AemcoloRifamycinCosmo TechnologiesTreatment of travelers’ diarrhea (TD) caused by non-invasive strains of Escherichia coli in adults.Anti-infectivePriority
3811/21/2018DaurismoGlasdegibPfizerTreatment of newly-diagnosed acute myeloid leukemia (AML) in adult patients who are >75 years old or who have comorbidities that preclude use of intensive induction chemotherapy.OncologyPriority, Orphan
3911/26/2018VitrakviLarotrectinibLoxo OncologyTreatment of adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (ntrk) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity and have no satisfactory alternative treatments or that have progressed following treatment.OncologyPriority, Orphan
4011/28/2018XospataGilteritinibAstellas PharmaTreatment of adult patients who have relapsed or refractory AML.OncologyStandard
4111/28/2018FirdapseAmifampridineCatalyst PharmaceuticalsTreatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults.Rare - CNSPriority, Orphan
4212/14/2018MotegrityPrucaloprideShireTreatment of chronic idiopathic constipation (CIC) in adults.Gastro intestinalStandard
Note: Based on data from Drugs@FDA and labels for drugs approved from January to December 2018.


Table 2. BLAs Approved in 2018 by CDER and CBER1,7

S.No.Approval DateDrug NameActive IngredientsCompanyIndicationTherapy AreaReview Priority
13/6/2018TrogarzoIbalizumab-uiykTaimed BiologicsTreatment of human immunodeficiency virus type 1 (HIV-1) infection in heavily treatment-experienced adults with multidrug resistant HIV-1 infection failing their current antiretroviral regimen.Anti-viralPriority, Orphan

23/20/2018IlumyaTildrakizumab-asmnMerckTreatment of adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy.Immune disorder
34/17/2018CrysvitaBurosumab-twzaUltragenyx PharmaceuticalTreatment of X-linked hypophosphatemia (XLH) in adults and for pediatric patients aged 1 or older.Metabolic disorderOrphan
45/3/2018AndexxaCoagulation factor Xa (recombinant), inactivatedPortola Pharmaceuticals

Indicated for patients treated with rivaroxaban and apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding.Hematology disorderBLA approved by CBER
55/17/2018AimovigErenumab-aooeAmgen, NovartisPreventive treatment of migraine in adults.CNS
65/24/2018PalynziqPegvaliase-pqpzBiomarin PharmIndicated to reduce blood phenylalanine concentrations in adult patients with phenylketonuria (PKU) who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management.Metabolic disorderOrphan
78/2/2018PanzygaImmune globulin intravenous (human)-ifasOctapharma Pharmazeutika Produktionsges.m.b.H.Treatment of primary humoral immunodeficiency (PI) in patients aged 2 years or older and chronic immune thrombocytopenic purpura (ITP) in adults.Hematology disorderBLA approved by CBER
88/8/2018PoteligeoMogamulizumab-kpkcKyowa KirinTreatment of adult patients with relapsed or refractory mycosis fungoides (MF) or Sézary syndrome (SS) after at least one prior systemic therapy.OncologyOrphan
98/22/2018OxervateCenegermin-bkbjDompe FarmaceuticiTreatment of neurotrophic keratitis.OphthalmologyOrphan
108/23/2018TakhzyroLanadelumab (SHP643)DyaxProphylaxis to prevent attacks of hereditary angioedema (HAE) in patients aged 12 years or older.CVSOrphan
118/29/2018JiviAntihemophilic Factor (recombinant) PEGylated-aucl - Factor VIII concentrateBayer HealthCareFor use in previously treated adults and adolescents (aged 12 years or older) with hemophilia A (congenital factor VIII deficiency) for: on demand treatment and control of bleeding episodes; perioperative management of bleeding; and routine prophylaxis to reduce the frequency of bleeding episodes.Hematology disorderBLA approved by CBER
129/13/2018LumoxitiMoxetumomab pasudotox-tdfkAstraZenecaTreatment of adult patients with relapsed or refractory hairy cell leukemia (HCL) who received at least two prior systemic therapies, including treatment with a purine nucleoside analogue (PNA).OncologyOrphan
139/14/2018AjovyFremanezumab-vfrmTevaPreventive treatment of migraine in adults.CNS
149/27/2018EmgalityGalcanezumab-gnlmEli LillyPreventive treatment of migraine in adults.CNS
159/28/2018LibtayoCemiplimab-rwlcRegeneron Pharmaceutical, SanofiTreatment of patients with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation.Oncology
1610/5/2018RevcoviElapegademase-lvlrLeadiant BiosciencesTreatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients.Rare - ImmuneOrphan
1711/20/2018GamifantEmapalumab-lzsgNovimmuneTeatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy.Hematology disorderOrphan
1812/20/2018AsparlasCalaspargase pegol-mknlServier PharmaceuticalTreatment of acute lymphoblastic leukemia in pediatric and young adult patients, from age 1 month to 21.OncologyOrphan
1912/21/2018UltomirisRavulizumab-cwvzAlexion PharmaceuticalTreatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).Hematology disorderOrphan
2012/21/2018ElzonrisTagraxofusp-erzsStemline TherapeuticsTreatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and pediatric patients aged 2 years or older.OncologyOrphan


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  1. Drugs@FDA, https://www.accessdata.fda.gov/scripts/cder/daf/.
  2. Mullard A, 2017 FDA drug approvals, Nature Reviews Drug Discovery, 19 Jan 2018.
  3. FDA on a Roll: Drug Approvals in H1 2018, Decision Resources Group, Blog, 9 August 2018 https://decisionresourcesgroup.com/drg-blog/fda-roll-drug-approvals-h1-2018/.
  1. Advancing health through innovation 2017 new drug therapy approvals, https://www.fda.gov/downloads/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ReportsBudgets/UCM591976.pdf. Accessed on July 12, 2018.
  2. 2016 Novel drugs summary, https://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DrugInnovation/UCM536693.pdf. Accessed on July 12, 2018.
  3. 2018: A Year of Innovation, Efficiency, and New Advances in Drug Therapy for the American Public, FDA Press Release, January 7, 2019.
  4. 2018 Biological License Application Approvals, https://www.fda.gov/BiologicsBloodVaccines/DevelopmentApprovalProcess/BiologicalApprovalsbyYear/ucm596371.htm. Accessed on January 5, 2019.

DRG Sources Used:

  1. Biktarvy: Disease Landscape & Forecast, Human Immunodeficiency Virus, October 2018, Decision Resources Group.
  2. Erleada: Disease Landscape & Forecast, Prostate Cancer, December 2018, Decision Resources Group.
  3. Olumiant: Disease Landscape & Forecast, Rheumatoid Arthritis, October 2018, Decision Resources Group.
  4. Libtayo: Real World Brand Tracker January 2016 to August 2018, December 2018, Decision Resources Group.
  5. Aimovig, Emgality: Disease Landscape & Forecast, Migraine, November 2018, Decision Resources Group.

Garima Kaul, PhD, MPharm., is a senior director in the market assessment team at Decision Resources Group. She has 15 years’ experience in the field of pharmaceutical business analytics and market research involving market assessment, competitive intelligence, opportunity assessment, social media analysis, and forecasting. She has supported many open innovation initiatives across the globe. Dr. Kaul has authored 22 publications in peer-reviewed international journals, 2 patent applications, and 1 book chapter. In addition, she has acted as a reviewer for many journals.

Contributors: Garima Kaul - Senior Director, Market Assessment
Published on: 11 January, 2019