FDA on a Roll: Drug Approvals in H1 2018

Contributors : Garima Kaul, Ph.D, M.Pharm; Senior Director, BioPharma Insights, Market Assessment

Publish date: 09 Aug, 2018

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Is the pharma industry finally out of the R&D and approval slump or does it continue to struggle to bring a product from bench to bed side?

2017 marked a great year for the pharmaceutical industry in terms of FDA approvals. The FDA’s Center for Drug Evaluation and Research (CDER) approved 46 new molecular entities (NMEs) and biologics in 2017, compared with only 22 approvals in 2016. This was the highest number of approvals made since 1996 (59), and the average of 32 approvals over the last 10 years. This high rate of approval is even more impressive, considering that the cost of bringing a molecule to the market can be as high as USD 2.6 billion owing to technical, regulatory and economic challenges.1 This blog studies whether last year’s momentum continues in 2018 and summarizes approval activity for the first six months.

In the first half of 2018, the FDA approved 16 NMEs and 8 biologics license applications (BLAs)2, 4 of which DRG forecasts to be blockbusters by 2025. In total, 69 approvals have been made in all submission categories (type 1 to 10), including three supplements to BLAs (Figure 1). In addition to the new drug applications (NDAs), 329 abbreviated NDAs (ANDAs) have been approved in the past six months. Table 1 lists the NMEs approved by the CDER in the first half of 2018.

With another six NME approvals made in July 2018, the FDA seems to be on a roll. With about 50 NMEs in the pre-registration stage, 2018 is expected to see many approvals and many blockbusters launch. The change in FDA leadership by the Trump administration may be a major reason for these approval advancements. Not only branded drugs, the FDA plans to get the generic products also faster to the patients (8-10 months vs. 4 years in the past). De-regularization, cutting down multiple review cycles, and streamlining the approval process are some of the steps that the FDA plans to take to reduce the approval time.

Classification Meaning Classification Meaning
Type 1 New molecular entity Type 8 Rx to OTC
Type 2 New active ingredient Type 9 New indication or claim, drug not to be marketed under type 9 NDA after approval
Type 3 New dosage form Type 10 New indication or claim, drug to be marketed under type 10 NDA after approval
Type 4 New combination Type 1/4 Type 1, New molecular entity, and Type 4, New combination
Type 5 New formulation or other differences Type 2/3 Type 2, New active ingredient, and Type 3, New dosage form
Type 6 New indication or claim, same applicant [no longer used] Type 2/4 Type 2, New active ingredient and Type 4, New combination
Type 7 Previously marketed but without an approved NDA Type 3/4 Type 3, New Dosage Form, and Type 4, New combination


Table 1. NMEs and BLAs Approved in H1 20182

Approval Date Drug Name Active Ingredients Company Indication Therapy Area Review Priority
01/26/2018 Lutathera Lutetium dotatate Lu-177 AAA USA Treatment of somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including foregut, midgut, and hindgut neuroendocrine tumors in adults Oncology Priority, Orphan
02/07/2018 Biktarvy Bictegravir sodium; emtricitabine; tenofovir alafenamide fumarate Gilead Sciences Treatment 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 Antiviral Priority
02/12/2018 Symdeko (copackaged) Ivacaftor; ivacaftor, tezacaftor Vertex Pharms Treatment of patients with cystic fibrosis (CF) aged 12 years and 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 - respiratory Priority, Orphan
02/14/2018 Erleada Apalutamide Janssen Biotech Treatment of patients with non-metastatic, castration-resistant prostate cancer (nm-CRPC) Oncology Priority
04/17/2018 Tavalisse Fostamatinib disodium Rigel Pharms Treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment Rare disorder - oncology Standard, Orphan
04/19/2018 Akynzeo Fosnetupitant chloride hydrochloride, palonosetron hydrochloride Helsinn Healthcare Prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of cancer chemotherapy, including, but not limited to, highly emetogenic chemotherapy Oncology - antiemetic Standard
05/16/2018 Lucemyra Lofexidine hydrochloride US Worldmeds Mitigation of opioid withdrawal symptoms to facilitate abrupt opioid discontinuation in adults. CNS Priority
05/18/2018 Lokelma Sodium zirconium cyclosilicate AstraZeneca Treatment of hyperkalemia in adults Metabolic disorder Standard
05/21/2018 Doptelet Avatrombopag maleate Akarx Treatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure. Hematology disorders Priority, Orphan
05/31/2018 Olumiant Baricitinib Eli Lilly Treatment of adult patients with moderately to severely active rheumatoid arthritis who have had an inadequate response to one or more TNF antagonist therapies. Immune disorder Standard
06/13/2018 Moxidectin Moxidectin Medicines Development for Global Health Treatment of onchocerciasis due to Onchocerca volvulus in patients aged 12 years and older. Anthelmintic Priority, Orphan
06/25/2018 Zemdri Plazomicin Achaogen Treatment of patients ≤ 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. Antibacterial Priority
06/25/2018 Epidiolex Cannabidiol GW Research Treatment of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS) in patients 2 years of age and older CNS Priority, Orphan
06/27/2018 Braftovi Encorafenib Array Biopharma Indicated, 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 Oncology Standard, Orphan
06/27/2018 Mektovi Binimetinib Array Biopharma Indicated, 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
Oncology Standard, Orphan
5/3/2018 Andexxa Coagulation Factor Xa (Recombinant), Inactivated Portola Pharmaceuticals

Indicated for patients treated with rivaroxaban and apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding. Hematology disorders BLA approved by Center for Biologics Evaluation and Research (CBER)


Potential Blockbuster NMEs and BLA Approved in H1 2018

Biktarvy (bictegravir sodium; emtricitabine; tenofovir alafenamide fumarate)

Company Gilead Sciences
Mechanism of action/class Combination of integrase strand transfer inhibitor and nucleoside reverse transcriptase inhibitor
Indication Indicated as a complete regimen for the treatment 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
Review Priority
Dosage form Once daily tablet

Biktarvy, a triple combination of bictegravir sodium (50 mg), emtricitabine (200 mg), and tenofovir alafenamide fumarate (25 mg), is expected to be one of the biggest launches of 2018. It is the smallest integrase strand transfer inhibitor-based triple-therapy single tablet regime available and would allow Gilead to compete directly with GSK’s Triumeq (dolutegravir/abacavir/lamivudine). Presence of tenofovir alafenamide fumarate leads to better long-term renal and bone safety and the absence of a booster makes Biktarvy a safer alternative to Stribild and particularly Genvoya. In June 2018, Biktarvy was also granted the Marketing Authorization by the European Commission for the treatment of HIV-1 infection.

Erleada (apalutamide)

Company Janssen Biotech (J&J)
Mechanism of action/class Androgen receptor inhibitor
Indication Treatment of patients with non-metastatic, castration-resistant prostate cancer (nm-CRPC)
Review Priority
Dosage form Once daily tablet

Erleada is the first FDA-approved therapy for nm-CRPC and is expected to help J&J fight against upcoming generics of its blockbuster Zytiga, further strengthening its positioning in the prostate cancer market. Phase III studies are also underway to expand its use in combination with Zytiga across several prostate cancer patient populations. The drug was originated by Aragon Pharmaceuticals and acquired by J&J in 2013. The approval comes after its major efficacy outcomes in Phase III trial, SPARTAN, and significant improvement in secondary endpoints. The drug demonstrated a 72% reduction in risk of distant metastasis or death and an increase by more than 2 years in median metastasis-free survival.

Olumiant (baricitinib)

Company Eli Lilly, Incyte
Mechanism of action/class JAK (janus kinase) inhibitor
Indication Treatment of adult patients with moderately to severely active rheumatoid arthritis who have had an inadequate response to one or more TNF antagonist therapies
Review Standard
Dosage form Once daily tablet

In May 2018, the FDA brought an end to the saga of resubmissions by hesitantly approving Eli Lilly’s once-daily oral JAK inhibitor, Olumiant, for moderate-to-severe rheumatoid arthritis. It is the second JAK inhibitor in the US market, approved about six years after Pfizer’s Xeljanz (tofacitinib). In Europe, however, both the doses were approved a month earlier than Xeljanz, giving it the advantage of being first in class, especially in Germany. The drug comes with a black-box warning and its dose-dependent safety concerns led to 2 mg per day dosage approval instead of 4 mg. The FDA continues to have concerns about its safety owing to its associated thromboembolic imbalance and risk/benefit profile, especially with the 4 mg dosage. DRG forecasts JAK inhibitors to be the fastest-growing class in rheumatoid arthritis; approval of Olumiant would act as a guide for AbbVie’s and Gilead/Galapagos’s upadacitinib and filgotinib, respectively. Many inflammatory and autoimmune diseases involve JAK-dependent cytokines, suggesting wide application of JAK inhibitors in inflammatory conditions. With a price of approximately $25,000 per year (around 60% less than AbbVie’s Humira), Olumiant is expected to be a blockbuster.

Aimovig (erenumab)

Company Amgen and Novartis
Mechanism of action/class Fully human monoclonal antibody to calcitonin gene-related peptide (CGRP)
Indication Preventive treatment of migraine in adults
Review Orphan
Dosage form Subcutaneous injection once monthly

Erenumab (subcutaneous administration of monthly dose of 70 or 140 mg) has been observed to significantly reduce migraine frequency and use of migraine medication, over a period of 6 months. Early benefits were observed in patients starting from month 1.4 The drug exhibited efficacy in both episodic and chronic migraine patients during its clinical studies and is the only FDA-approved drug to block CGRP receptor. The drug can be self-administered with an autoinjector, SureClick®.5 The drug is co-developed by Novartis and Amgen; Amgen holds commercialization rights in the United States, Canada, and Japan and Novartis holds rights in Europe and rest of the world.6 


Key Trends in FDA Approvals

Is getting into orphan drug research, a faster way to bring drugs to the market?

In 2018, the trend of developing orphan drugs treating rare indications continues and is perhaps rising.

  • In the first half of the year, among the NMEs and BLAs approved (type 1 and type 1/4), 38% were orphan drugs, compared with 39% for all of 2017 and 41% in 2016.7,8
  • 20 of the pre-registered NMEs, have an orphan drug designation for one or more indications.

The high prices this category of drugs can demand have been luring companies to add these indications to their portfolios. Sales of orphan drugs have also been forecast to grow tremendously and outpace all other medications. The traditional blockbuster model is fading away, giving way to high-priced therapies treating rare diseases. It is good to see that these indications are no longer neglected. There now seems to be a tug-of-war between the clinical value these drugs bring to patients and the budget impact made by these expensive drugs.

The FDA continues to offer priority review benefits to drugs that could provide significant advancement (safety and efficacy) in treatment.

  • As in 2017, the number of priority review designations continues to be high. 42% of the NMEs and BLAs received priority review by CDER in the first half of 2018 (vs. 61% in 2017, 68% in 2016). Only 29% of the NMEs and BLAs received standard review.

The trend holds true for cancer drugs as well, with 25% of NMEs and BLAs approved in 2018 being cancer drugs. Furthermore, among the pre-registered drugs, 25% are for oncology indications. Cancer drugs, specialty medicines, orphan drugs are hot topics that are expected to continue to make noise and to highly impact healthcare expenditure.

It is debatable, though, whether speeding the approvals will be beneficial for both the industry and patients. More approvals can lead to increasing the burden on prescribers in terms of understanding more products and evaluating their safety and efficacy. Also, faster drug approvals by reducing the barriers can put patients to risk. However, faster approvals can mean more drugs reaching the bedside and reduced regulatory hurdles may lower the drug development cost eventually benefiting the patients and payers. Generic drug makers are also delighted by this move promised by the FDA. Speedy approval process is expected to reduce the burden on companies, promote competition and innovation. Cheaper medicines with quick and easier access is surely a welcomed step.


  1. The pharmaceutical industry and global health: facts and figures 2017, https://www.ifpma.org/wp-content/uploads/2017/02/IFPMA-Facts-And-Figures-2017.pdf. Accessed on July 12, 2018.
  2. Drugs@FDA, https://www.accessdata.fda.gov/scripts/cder/daf/.
  3. Eli Lilly's Olumiant Gets FDA Nod for Rheumatoid Arthritis, https://www.nasdaq.com/article/eli-lillys-olumiant-gets-fda-nod-for-rheumatoid-arthritis-cm973005. Accessed on July 30, 2018.
  4. Goadsby PJ, et.al., A controlled trial of erenumab for episodic migraine, N Engl J Med, 2017.
  5. Novartis press release, May 2018, https://novartis.gcs-web.com/Novartis-and-Amgen-announce-FDA-approval-of-Aimovig-erenumab-a-novel-treatment-developed-specifically-for-migraine-prevention. Accessed on July 30, 2018.
  6. Aimovig (erenumab) for the treatment of episodic migraine, https://www.drugdevelopment-technology.com/projects/aimovig-erenumab-treatment-episodic-migraine/. Accessed on July 30, 2018.
  7. 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.
  8. 2016 Novel drugs summary, https://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DrugInnovation/UCM536693.pdf. Accessed on July 12, 2018.

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. JAK inhibitor safety is front and center during regulatory approval, DRG Blog
  5. Aimovig, Emgality: Disease Landscape & Forecast, Migraine, November 2018, Decision Resources Group.

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About the Author:

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.

Infographic: Key Trends in Patient Rx Decision Making