New Drug Approvals in 2017 Outpacing 2016

By Patricia Van Arnum - DCAT Editorial Director

September 27, 2017

The US Food and Drug Administration (FDA) has approved 33 new molecular entities (NMEs) thus far in 2017, already surpassing the 2016 total of 22. So which drugs are making the mark?

Following a downturn in 2016, new drug approvals are on the uptick in 2017 and are on pace to meet or exceed a recent high in NME approvals in 2015 when 45 NMEs were approved. DCAT Value Chain Insights examines the blockbuster potential and the mix between small molecules and biologics.

NME approvals on the rise

The number of approvals for new molecular entities (NMEs) by the US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) is on a strong pace in 2017 after a drop-off in NME approvals in 2016. As of September 21, 2017, the FDA had approved 33 NMEs (see Table I at end of article) compared to 22 NMEs (20 new drugs and two new diagnostic/imaging agents) in all of 2016. The number of NME approvals thus far in 2017 resumes an upward trajectory beginning in 2011 (with the exception of 2013) for NME approvals with 30 NMEs approved in 2011 and 39 in 2012. The exception was in 2013, which had a decline to 27 NMEs, but levels jumped again to 41 NMEs approved in 2014 and peaked at a recent high of 45 NME approvals in 2015.

Small molecules versus biologics

In looking at the 33 NME approvals by FDA’s CDER (see Table I at end of article), 25 or 76% were small molecules and eight or 24% were biologics. The nearly quarter of NME approvals that were biologics are slightly down from recent product mix for NME approvals. In 2016, 32% of the NME approvals were biologics, and in 2015 and 2014, 27% of NME approvals were biologics (see Table II at end of article).

Also in 2017, FDA’s Center for Biologics Evaluation and Research approved a cell-based gene therapy, Novartis' Kymriah (tisagenlecleucel), a chimeric antigen receptor T-cell (CAR T) therapy, marking the first CAR T therapy approved by the FDA. The therapy was approved in August 2017 for treating certain pediatric and young adult patients with a form of acute lymphoblastic leukemia. Each dose of Kymriah is a customized treatment created using an individual patient’s own T-cells, a type of white blood cell known as a lymphocyte. The patient’s T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein (a chimeric antigen receptor or CAR) that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.

Blockbuster potential

Several NME approved thus far in 2017 are projected by some analysts as future blockbusters (defined as drugs with sales of $1 billion) by 2021. They include: AstraZeneca’s Imfinzi (durvalumab) for treating bladder cancer; Merck Serono's and Pfizer’s Bavencio (avelumab) for treating a rare form of skin cancer; Novartis’ Kasqali (ribociclib) for treating breast cancer; Roche’s Ocrevus (ocrelizumab) for treating multiple sclerosis (MS); Sanofi’s and Regeneron Pharmaceuticals’ Dupixent (dupilumab) for treating a common form of eczema;and Tesaro’s Zejula (niraparib) for treating ovarian cancer.

AstraZeneca’s Imfinzi (durvalumab). AstraZeneca’s PD-L1 antibody, durvalumab, was approved by the FDA for treating bladder cancer. A recent analysis by Clarivate Analytics points out that while the drug will provide a meaningful treatment option in the bladder cancer setting, it is in lung cancer where the most notable sales are expected. Additional clinical data in non-small-cell lung cancer is expected, which could set the stage for a filing in that indication and potentially offering a competitive alternative to other immunotherapies: Bristol-Myers Squibb’s Opdivo (nivolumab) and Merck & Co.’s Keytruda (pembrolizumab). Projected 2021 sales are $2.0 billion.

Merck Serono's and Pfizer’s Bavencio (avelumab). Merck KGaA’s and Pfizer’s Bavencio (avelumab) is a fully human anti-PDL1 antibody expected to enter the market in the third quarter of 2017 in its first cancer setting of second-line metastatic Merkel cell carcinoma, a rare form of skin cancer, for which it received FDA approval in March 2017. Avelumab is part of the immunotherapy alliance that Pfizer and Merck KGaA formed in November 2014 under which the companies are collaborating on up to 20 high priority immuno-oncology clinical development programs, including combination trials. The clinical development program for avelumab involves study of more than 15 tumor types, including breast cancer, gastric/gastroesophageal cancer, head and neck cancer, Merkel cell carcinoma (an aggressive form of skin cancer), mesothelioma, melanoma, non-small cell lung cancer, ovarian cancer, renal cell carcinoma, and urothelial (e.g. bladder) cancer. Phase III cinical trials for ovarian cancer are ongoing with data expected in 2018 for 2019. Forecast 2021 sales are $1.2 billion, according to Clarivate.

Novartis’ Kisqali (ribociclib). Novartis’ Kisqali (ribociclib) is another drug approved in 2017 with potential blockbuster status. It is a highly selective CDK4/CDK6 inhibitor, which was approved by the FDA for treating HR-positive, HER2-negative first-line breast cancer. The drug will compete with Pfizer’s CDK4/6 inhibitor, Ibrance (palbociclib), a strong competitor and earlier market entry that was launched in early 2015. Forecasts for 2021 for the drug are nearly $1.3 billion, according to the Clarivate analysis.

Roche’s Ocrevus (ocrelizumab). Roche’s Ocrevus (ocrelizumab) is expected to materially disrupt the MS market, according to a recent analysis by Clarivate Analytics in analyzing future blockbusters. Ocrevus is a first-in-class anti-CD20 antibody, and it showed positive results in reducing the annualized relapse rate in MS and is the first drug to prove effective in primary progressive MS. The Clarivate report points out that initial market share gains are likely in the second line behind oral options and in severe patients on Biogen’s Tysabri (natalizumab) who are at risk of progressive multifocal leukoencephaolopathy, but adoption in naive patients may be expected in the medium term as prescriber familiarity builds. Clarivate projects 2021 sales of $3.3 billion.

Sanofi’s and Regeneron Pharmaceuticals’ Dupixent (dupilumab). Sanofi’s and Regeneron Pharmaceuticals’ Dupixent (dupilumab) is a human monoclonal antibody that is designed to specifically inhibit overactive signaling of two key proteins, IL-4 and IL-13, which are believed to be major drivers of the persistent underlying inflammation in atopic dermatitis (AD), a common form of eczema. The drug was approved by the FDA in March 2017 for treating moderate-to-severe AD whose disease is not adequately controlled with topical prescription therapies, or when those therapies are not advisable. Clarivate projects 2021 sales of $2.8 billion.

Tesaro’s Zejula (niraparib). Tesaro’s Zejula (niraparib), an oral, once-daily poly(ADP-ribose) polymerase (PARP) inhibitor, was approved in 2017 for the maintenance treatment of women with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy. Projected 2021 sales are nearly $1.1 billion.

Table I (see below) outlined the 33 NME approvals thus far in 2017, and Table II (see below) shows the product mix of NME approvals between small molecules and biologics from 2010 to 2017 thus far. 

Table I: Approvals of New Molecular Entities (New Drug Applications (NDAs) and Original Biologics License Applications (BLAs) in 2017 by the US Food and Drug Administration’s Center for Drug Evaluation and Research (as of September 21, 2017).
Company Brand name (active ingredient); application type Indication
AbbVie Mavyret (glecaprevir and pibrentasvir); NDA Chronic hepatitis C virus genotypes 1-6 without cirrhosis or with mild cirrhosis
Amgen Parsabiv (etelcalcetide); NDA Secondary hyperparathyroidism in adult patients with chronic kidney disease on hemodialysis
AstraZeneca Imfinzi (durvalumab); BLA Locally advanced or metastatic urothelial carcinoma (bladder cancer)
Bayer Aliqopa (copanlisib); NDA Relapsed follicular lymphoma
BioMarin Pharmaceutical Brineura (cerliponase alfa); BLA A specific form of Batten disease
Celgene Idhifa (enasidenib); NDA Relapsed or refractory acute myeloid leukemia in patients with a specific genetic mutation
Chemo Research benznidazole; NDA Chagas disease
Gilead Sciences Vosevi (sofosbuvir, velpatasvir, and voxilaprevir); NDA Chronic hepatitis C virus, genotypes 1-6 without cirrhosis or with mild cirrhosis
Johnson & Johnson (Janssen Biotech) Tremfya (guselkumab); BLA Moderate-to-severe plaque psoriasis
Lexicon Pharmaceuticals Xermelo (telotristat ethyl); NDA Carcinoid syndrome diarrhea
Melinta Therapeutics Baxdela (delafloxacin); NDA Acute bacterial skin infections
Merck KGaA and Pfizer Bavencio (avelumab); BLA Metastatic Merkel cell carcinoma (a rare form of skin cancer)
Mitsubishi Tanabe Pharma America Radicava (edaravone); NDA Amyotrophic lateral sclerosis (Lou Gehrig’s disease)
Neurocrine Biosciences Ingrezza (valbenazine); NDA Tardive dyskinesia
Novartis Kisqali (ribociclib); NDA Hormone receptor-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer
Novartis Rydapt (midostaurin); NDA Acute myeloid leukemia in patients with a specific genetic mutation, FLT3, in combination with chemotherapy
Pfizer Besponsa (inotuzumab ozogamicin); BLA Relapsed or refractory B-cell precursor acute lymphoblastic leukemia
Portola Pharmaceuticals Bevyxxa (betrixaban); NDA Prophylaxis of venous thromboembolism
PTC Therapeutics Emflaza (deflazacort); NDA Duchenne muscular dystrophy
Puma Biotechnology Nerlynx (neratinib); NDA Extended adjuvant treatment of early-stage, HER2-positive breast cancer
Radius Health Tymlos (abaloparatide); NDA Osteoporosis
Roche Ocrevus (ocrelizumab); BLA Relapsing forms of multiple sclerosis and primary progressive multiple sclerosis
Sanofi and Regeneron Pharmaceuticals Dupixent (dupilumab); BLA Moderate-to-severe eczema (atopic dermatitis)
Sanofi Kevzara (sarilumab); BLA Rheumatoid arthritis
Shionogi Symproic (naldemedine); NDA Opioid-induced constipation in adult patients with chronic non-cancer pain
Symbiomix Therapeutics Solosec (secnidazole); NDA Bacterial vaginosis
Synergy Pharmaceuticals Trulance (plecanatide); NDA Chronic idiopathic constipation
Takeda Pharmaceutical Alunbrig (brigatinib); NDA Anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer
Tesaro Zejula (niraparib); NDA Epithelial ovarian, fallopian tube or primary peritoneal cancer
Teva Pharmaceuticals Austedo (deutetrabenazine); NDA Chorea associated with Huntington’s disease
The Medicines Company Vabomere (meropenem and vaborbactam); NDA Complicated urinary tract infections
US World Meds Xadago (safinamide); NDA An add-on treatment for patients with Parkinson’s disease
Valeant Pharmaceuticals Siliq (brodalumab); BLA Moderate-to-severe plaque psoriasis

PTC Therapeutic acquired rights to Emflaza (deflazacort) for the treatment of Duchenne muscular dystrophy in the US from Marathon Pharmaceuticals in April 2017.

Takeda acquired Ariad Pharmaceuticals in February 2017.

The Medicines Company acquired Rempex Pharmaceuticals in 2013.

US WorldMed is partnered with Zambon, which is partnered with Newron Pharmaceuticals by which Zambon has the rights to develop and commercialize Xadago (safinamide) globally, excluding Japan and other key territories where Meiji Seika has the rights to develop and commercialize the compound. The rights to develop and commercialize Xadago in the US have been granted to US WorldMeds by Zambon.

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 (as of September 21, 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 (as of September 25, 2017) 33 NMEs approved 76% small molecules (25 NMEs)
24% biologics (8NMEs)

*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
*****In 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.