New Drug Approvals: Which Drugs Made the Mark in 2018?

By Patricia Van Arnum - DCAT Editorial Director

January 16, 2019

Last year (2018) was a record-setting year for approvals of new molecular entities (NMEs) by the FDA with 59 NMEs approved. DCAT Value Chain Insights examines the 2018 NME approvals, the product mix between small molecules and biologics, and which pharma companies led the pack.

Inside new drug approvals

In 2018, the US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) approved a record-high 59 new molecular entities (NMEs), surpassing the previous record of 53 NME approvals in 1996 and the 46 NMEs approved in 2017. The 59 NME approvals in 2018 was nearly double the 10-year (2009–2017) average of 33 NME approvals by FDA’s CDER. The record-number of NME approvals continues an upward trajectory for NME approvals (with the exception of 2016 when 22 NMEs were approved) over the last several years. FDA’s CDER approved 41 NMEs in 2014, 45 NMEs in 2015, 46 NMEs in 2017, and the 59 NMEs in 2018.

In addition, continuing a recent trend, orphan drugs accounted for a large percentage of NME approvals. More than half (58%), or 34, of the NME approvals in 2018 were for orphan drugs, defined as drugs that treat 200,000 or fewer people in the US. In 2017, 39%, or 18 of the 46 NMEs approved by the FDA’s CDER were orphan drugs, which continued a recent trend of approximately 40% of NME approvals being orphan drugs. In 2016, 9 of the 22 NMEs approved by the FDA were orphan drugs. In 2015, 47%, or 21 of the 45 NMEs approved by the FDA’s CDER were orphan drugs, and in 2014, 41%, or 17 of the 41 NMEs approved were orphan drugs.

Inside NME approvals in 2018

Table I at the end of article outlines the NMEs approved in 2018  which was second only to the 53 NMEs approved in 1996. Small molecules dominated with 42 small molecule NME approvals (72% of NME approvals) and 17 biologic-based NME approvals (28% of NME approvals), which includes one antibody drug conjugate (ADC). This product mix in 2018 is consistent with NME approvals in 2017. In 2017, small-molecule NME approvals accounted for 76% of new drug approvals (see Table II at end of article) and biologics 24%.

The large pharmaceutical companies with biologic-based drug approvals in 2018 include: Amgen’s Aimovig (erenumab-aooe), a drug to prevent migraines in adults; AstraZeneca’s Lumoxiti (moxetumomab pasudotox-tdfk), an ADC for treating hairy cell leukemia; Eli Lilly and Company’s Emgality (galcanezumab-gnlm) for the preventive treatment of migraine in adults; Merck & Co.’s and Sun Pharmaceutical's Ilumya (tildrakizumab) for treating moderate-to-severe plaque psoriasis; and Teva Pharmaceutical Industries’ Ajovy (fremanezumab-vfrm) for the preventive treatment of migraine in adults. Table I at the end of the article summarizes approvals for biologics [denoted by BLA for biologics license applications (BLA) and small-molecules (denoted by new drug application (NDA)] in 2018.

Big Pharma and NME approvals

Table I: Approvals of New Molecular Entities, New Drug Applications (NDAs) and Original Biologics License Applications (BLAs) by the US Food and Drug Administration’s Center in 2018.
Company Brand name (active ingredient); application type Indication
AbbVie Orilissa (elagolix sodium); NDA Moderate-to-severe pain associated with endometriosis
Advanced Accelerator Applications (Novartis company) Lutathera (lutetium Lu 177 dotatate); NDA Gastroenteropancreatic neuroendocrine tumors, a type of cancer that affects the pancreas or gastrointestinal tract
Achaogen Zemdri (plazomicin); NDA Adults with complicated urinary tract infections
Agios Pharmaceuticals Tibsovo (ivosidenib); NDA Relapsed or refractory acute myeloid leukemia
AkaRx Doptelet (avatrombopag); NDA Low blood platelet count (thrombocytopenia) in adults with chronic liver disease who are scheduled to undergo a medical or dental procedure
Alexion Pharmaceuticalstd> Ultomiris (ravulizumab); BLA Paroxysmal nocturnal hemoglobinuria
Allergan Seysara (sarecycline); NDA Inflammatory lesions of non-nodular moderate to severe acne vulgaris in patients 9 years of age and older
Alnylam Pharmaceuticals Onpattro (patisiran); NDA Polyneuropathy of hereditary transthyretin-mediated amyloidosis in adult patients
Amgen Aimovig (erenumab-aooe); BLA Preventive treatment of migraines in adults
Amicus Therapeutics Galafold (migalastat); NDA Adults with Fabry disease
Array BioPharma Braftovi (encorafenib); NDA Unresectable or metastatic melanoma
Array BioPharma Mektovi (binimetinib); NDA Unresectable or metastatic melanoma
Astellas Xospata (gilteritinib); NDA Lapsed or refractory acute myeloid leukemia (AML)
AstraZeneca Lokelma (sodium zirconium cyclosilicate); NDA Hyperkalemia (high potassium levels)
Astellas Lokelma (sodium zirconium cyclosilicate); NDA Hyperkalemia (high potassium levels)
Biocodex SA Diacomit (stiripentol); NDA Seizures associated with Dravet syndrome in patients 2 years of age and older taking clobazam
BioMarin Pharmaceutical Palynziq (pegvaliase-pqpz); BLA Phenylketonuria (inherited disorder that increases the levels of phenylalanine in the blood)
Cosmo Technologies Firdapse (amifampridine); NDA Travelers’ diarrhea
Dompe Farmaceutici Oxervate (cenegermin-bkbj); BLA Neurotrophic keratitis
Dyax Corp. Takhzyro (lanadelumab); BLA Types I and II hereditary angioedema
Eli Lilly and Company Emgality (galcanezumab-gnlm); BLA For the preventive treatment of migraines in adults
Eli Lilly and Company Olumiant (baricitinib); NDA Moderately to severely active rheumatoid arthritis
Fresenius Kabi Omegaven (fish oil triglycerides); NDA A source of calories and fatty acids in pediatric patients with parenteral nutrition-associated cholestasis
Gilead Sciences Biktarvy (bictegravir, embitcitabine, and tenofovir alafenamide); NDA HIV-1 infection in adults who have no antiretroviral treatment history or to replace the current antiretroviral regimen
GlaxoSmithKline Krintafel (tafenoquine); NDA Radical cure (prevention of relapse) of Plasmodium vivax malaria
GW Research Epidiolex (cannabidiol); NDA Seizures associated with two rare and severe forms of epilepsy, Lennox-Gastaut syndrome and Dravet syndrome
Helsinn Healthcare Akynzeo (fosnetupitant and palonosetron); NDA To prevent acute and delayed nausea and vomiting associated with initial and repeat courses of highly emetogenic cancer chemotherapy
Ionis Pharmaceuticals Tegsedi (inotersen); NDA Polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
Janssen Pharmaceutical (Johnson & Johnson) Erleada (apalutamid); NDA Non-metastatic, castration-resistant prostate cancer
Kyowa Kirin Poteligeo (mogamulizumab-kpkc); BLA Two rare types of non-Hodgkin lymphoma
Leadiant Biosciences Revcovi (elapegademase-lvlr); BLA Adenosine deaminase-severe combined immunodeficiency (ADA-SCID)
Loxo Oncology Vitrakvi (larotrectinib); NDA Patients whose cancers have a specific genetic feature (biomarker)
Medicines Development for Global Health Moxidectin (moxidectin); NDA Onchocerciasis due to Onchocerca volvulus in patients aged 12 years and older
Merck & Co. Pifeltro (doravirine); NDA HIV-1 infection in adult patients
Merck & Co. and Sun Pharmaceutical Ilumya (tildrakizumab); BLA Adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy
Mylan Yupelri (revefenacin); NDA Chronic obstructive pulmonary disease (COPD)
Novoimmune Gamifant (emapalumab-lzsg) BLA Primary hemophagocytic lymphohistiocytosis
Paratek Pharmaceuticals Nuzyra (omadacycline); NDA Community-acquired bacterial pneumonia and acute bacterial skin and skin structure infections
Pfizer Daurismo (glasdegib); NDA Newly-diagnosed acute myeloid leukemia (AML) in adult patient
Pfizer Lorbrena (loratinib); NDA Patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer
Pfizer Talzenna (talazoparib); NDA Locally advanced or metastatic breast cancer patients with a germline BRCA mutation
Pfizer Vizimpro (dacomitinib); NDA Metastatic non-small-cell lung cancer
Regeneron Pharmaceuticals Libtayo (cemiplimab-rwlc); BLA Cutaneous squamous cell carcinoma
Rigel Pharmaceuticals Tavalisse (fostamatinib); NDA Thrombocytopenia (low blood platelet count) in adult patients with persistent or chronic immune thrombocytopenia
Servier Asparlas (calaspargase pegol-mknl); BLA Acute lymphoblastic leukemia (ALL) in pediatric and young adult patients age 1 month to 21
Shionogi Mulpleta (lusutrombopag); NDA Thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure
Shionogi Xofluza (baloxavir marboxil); NDA Idiopathic constipation
Siga Technologies Tpoxx(tecovirimat); NDA Smallpox
Stemline Therapeutics Elzonris (tagraxofusp-erzs); BLA Blastic plasmacytoid dendritic cell neoplasm (BPDCN)
TaiMed Biologics USA Trogarzo (ibalizumab-uiyk); BLA HIV
Tetraphase Pharmaceuticals Xerava (eravacycline); NDA Complicated intra-abdominal infections in patients 18 years of age and older
Teva Pharmaceutical Industries Ajovy (fremanezumab-vfrm); BLA For the preventive treatment of migraines in adults
TherapeuticsMD Annovera (segesterone acetate and ethinyl estradiol vaginal system); NDA New vaginal ring used to prevent pregnancy for an entire year
Verastem Copiktra (duvelisib); NDA Relapsed or refractory chronic lymphocytic leukemia, small lymphocytic lymphoma and follicular lymphoma
Ultragenyx Pharmaceutical Crysvita (burosumab-twza); BLA To treat adults and children ages 1 year and older with x-linked hypophosphatemia, a rare, inherited form of rickets
US WorldMeds Lucemyra (lofexidine); NDA Non-opioid treatment for management of opioid withdrawal symptoms in adults
Vertex Pharmaceuticals Symdeko (tezacaftor and ivacaftor); NDA Cystic fibrosis in patients age 12 years and older

Novartis acquired Advanced Accelerator Applications in January 2018.

In August 2015, Amgen entered into a global collaboration with Novartis to jointly develop and commercialize treatments in the fields of migraine and Alzheimer's disease. The collaboration focuses on investigational Amgen drugs in the migraine field, including Aimovig. In April 2017, the collaboration was expanded to include co-commercialization of Aimovig in the US. For the migraine program, Amgen retains exclusive rights in Japan, and Novartis has exclusive rights in Europe, Canada, and rest of the world.

Array BioPharma has exclusive rights to Braftovi (encorafenib) and Mektovi (binimetinib) in the US and Canada. Array has granted Ono Pharmaceutical exclusive rights to commercialize both products in Japan and South Korea and Pierre Fabre exclusive rights to commercialize both products in all other countries, including in Europe, Asia, and Latin America.

Eli Lilly and Company agreed to acquire Loxo Oncology in 2018.

Shire was acquired by Takeda in January 2018.

Sun Pharmaceutical Industries Ltd.'s wholly owned subsidiary licensed worldwide rights to Ilumya (tildrakizumab) from a subsidiary of Merck & Co., Inc. in 2014. Funded by a Sun Pharma subsidiary, Merck & Co., Inc. was responsible for the completion of Phase-III trials and submission of a biologics license application to the US Food and Drug Administration (FDA) as well as manufacturing finished goods to support Sun Pharma's initial product launch. Sun Pharma is responsible for all post-approval regulatory activities, including subsequent submissions, pharmacovigilance, post approval studies, manufacturing, and commercialization of the approved product. Sun Pharma is also responsible for all regulatory, pharmacovigilance, post approval studies, manufacturing, and commercialization of approved products for all non-U.S. markets. Merck & Co is eligible to receive milestone payments and royalties on sales of Ilumya.

Source: US Food and Drug Administration's Center for Drug Evaluation and Research and company information.

 

Table II: Small Molecule and Biologics New Molecular Entities Approved by the US Food and Drug Administration’s Center for Drug Evaluation and Research, 2010 to 2017.
Year Number of New Molecular Entities (NMEs) Approved Percentage relative to total NME approvals and number of small molecules and biologics approved as NMEs
2010 21 NMEs approved 71% small molecules (15 NMEs)
29% biologics (6 NMEs)
2011 30 NMEs approved 77% small molecules (23 NMEs)
20% biologics (6 NMEs)
Plus 1 NME radioactive diagnostic imaging agent*
2012 39 NMEs approved

79% small molecules (31 NMEs)
15% biologics (6 NMEs)
Plus 2 NME radioactive diagnostic imaging agents**

2013 27 NMEs approved 81% small molecules (22 NMEs)
11% biologics (3 NMEs)
Plus 2 NME radioactive imaging agents***
2014 41 NMEs approved 71% small molecules (29 NMEs)
27% biologics (11 NMEs)
Plus 1 NME radioactive diagnostic imaging agent****
2015 45 NMEs approved 71% small molecules (32 NMEs) and 1 insulin analog NME approved as a new drug application*****
27% biologics (12 NMEs)
2016 22 NMEs approved 59% small molecules (13 NMEs)
32% biologics (7 NMEs)
Plus 2 NME radioactive diagnostic imaging agents******
2017 46 NMEs approved 74% small molecules (34 NMEs)
26% biologics (12 NMEs)

In 2011, 23 small-molecule drugs and 1 radioactive diagnostic imaging were approved as new drug applications (NDAs).

In 2012, 31 small-molecule drugs and 2 radioactive diagnostic imaging agents were approved as NDAs.

In 2013, 22 small-molecule drugs and 2 radioactive diagnostic imaging agents were approved as NDAs.

In 2014, 29 small-molecule drugs and 1 radioactive diagnostic imaging agent were approved as NDAs .

n 2015, 32 small-molecule drugs were approved as NDAs and one insulin analog, Novo Nordisk's Tresiba (insulin degludec injection), a long-acting basal human insulin analog produced by a process that includes expression of recombinant DNA in Saccharomyces cerevisiae followed by chemical modification was approved as a NDA, not as a biologics license application.

In 2016, 13 small-molecule drugs and 2 diagnostic imaging agents were approved as NDAs.

Source: US Food and Drug Administration's Center for Drug Evaluation and Research and company information.