NMEs, notable line extensions and worldwide approvals: A comprehensive look at the year’s new drugs & biologics, 2017

NMEs, notable line extensions and worldwide approvals:  A comprehensive look at the year’s new drugs & biologics, 2017
by
Senior Content Manager, Clarivate Analytics
NMEs, notable line extensions and worldwide approvals:  A comprehensive look at the year’s new drugs & biologics, 2017
Ann Graul
Senior Content Manager, Clarivate Analytics
Ann Graul is Senior Content Manager of the Cortellis/Integrity Disease Briefings. She has over 20 years of experience writing about the pharmaceutical industry, and 12 years as lead scientific writer and content manager of the Disease Briefings. The series of comprehensive, continuously updated reviews, covering close to 150 human diseases and conditions and their treatments, was first introduced in 2000 as part of Integrity and has since been incorporated in the Cortellis platform. Ann is also the lead author and creative force behind the annual two-part review article, The Year's New Drugs & Biologics, which is published each year in the Thomson Reuters journal Drugs of Today and reports on major trends in the pharma industry over the year just past. She has also authored and edited drug monographs for the journals Drugs of Today and Drugs of the Future, and has written for Thomson Reuters Drug News. Ann was born and educated in the U.S. and lives in Barcelona, Spain. She holds a degree from Lawrence University and certification from the American Medical Writers Association.
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“The year’s new drugs & biologics, 2017” presents an in-depth look at 113 of the new drugs and biologics that were approved or launched for the first time globally in 2017. Fifty-two of the new arrivals reached their first markets worldwide in 2017. Seven of the new launches were first-in-class agents, meaning the first drug with a novel mechanism of action to be approved and launched anywhere in the world.

In addition, 36 notable line extensions (i.e., new indications, new combinations and new formulations of previously marketed products) were introduced last year. Twenty-five products discussed in the review were approved for the first time during the year just passed, but had not yet been launched as of December 31, 2017.

“The year’s new drugs & biologics,” a comprehensive annual two-part review, was compiled from Cortellis and Integrity, the Clarivate Analytics databases, along with Clarivate’s Drug News and company communications.

Notable developments during 2017 included the launch of the world’s first CAR-T therapies for cancer (see Figure I for a graphic depiction of this novel treatment approach) and approval of the first digital pill, as well as the approval of the first gene therapy in the U.S. for an inherited disease caused by mutations in a specific gene.

Tisagenlecleucel and axicabtagene ciloleucel are new gene therapies consisting of adaptive, autologous T lymphocytes transduced with a replication-deficient lentiviral vector. They are designed for use in the therapeutic intervention of relapsed or refractory (r/r) B-cell precursor acute lymphoblastic leukemia (ALL) in pediatric and young adult patients, as well as r/r diffuse large B-cell lymphoma in individuals ineligible for autologous stem cell transplantation (ASCT). These agents are comprised of genetically modified antigen-specific autologous T cells that have been reprogrammed to target cells that express CD19, an antigen expressed on the surface of B cells and tumors derived from B cells. The chimeric antigen receptor (CAR) protein harbors an extracellular segment that has a murine anti-CD19 single chain antibody fragment (scFv) and an intracellular segment that contains T-cell signaling (CD3-ζ) and costimulatory (4-1BB) domains. These intracellular domains are essential for each drug’s activity, including the induction and persistence of T cells and their antitumor effects, including cytotoxicity, degranulation and granzyme-mediated tumor lysis.

The seven first-in-class agents included the CAR-T therapies as well as the tryptophan hydroxylase 1 (TPH1) inhibitor telotristat ethyl, the anti-interleukin-4 receptor α (IL-4Rα) monoclonal antibody (MAb) dupilumab, and the purine nucleoside phosphorylase (PNP) inhibitor forodesine.

Regarding newly launched NMEs and biologics, oncology was by far the most active therapeutic group in 2017, with 17 new products introduced last year; this was the highest number in the last decade (see Table I). Orphan drugs continue to be a focus of new drug R&D, and 31 of last year’s newly launched drugs and biologics (including line extensions) had orphan drug status, some for more than one indication.

Table I. New drugs & biologics by therapeutic category, launched in 2007-2017*

THERAPEUTICS20072008200920102011201220132014201520162017
Central nervous system34745243476
Respiratory22211215311
Cardiovascular22111121120
Renal-urologic12202102110
Hematologic26313217741
Gastrointestinal13110141414
Endocrine drugs20321446313
Dermatologic02101211243
Anti-infective531260511554
Musculoskeletal01301201112
Immunologic43175451125105
Cancer5167710121014517
Ophthalmic01112012012
Metabolic drugs22342273543
Poisoning & drug abuse00000110010
Dental00000010000
Diagnostic agents11000113021
Total3033512936365658555052

*Does not include line extensions.

Sources: Cortellis, Integrity and Drug News, from Clarivate Analytics; company reports.

The most active market for new drugs in 2017 was again the U.S., which welcomed 35 of the 52 new products (67%). Other markets lagged much further behind, with eight new launches in the European Union, three in Japan and just one apiece in other countries (Canada, Korea, Russia, Argentina, Taiwan and India).

Part I of the annual review looks at drug development advances across multiple therapeutic areas, from agents for analgesia and anesthesia to ophthalmic drugs to treatments for cancer. It was written by Clarivate’s Ann Graul, Patricia Pina and Mark Stringer and includes six additional drug diagrams.

Part II provides insight into some of last year’s top news and trends, including the growing opioid crisis affecting the U.S. and other developed countries and the 2017-2018 influenza epidemic, with a spotlight on the need for a universal flu vaccine.

As in previous years, we also review orphan drug development, new agency-supported programs such as PRIME and RMAT, pipeline attrition and drug pricing, as well as pharma/biotech mergers and acquisitions of note. Finally, we take a glimpse into the crystal ball to anticipate the new drugs that will be approved in 2018. Part II was prepared by A. Graul, P. Pina, Coia Dulsat, Michelle Tracy and Paul D’Souza.

Both parts of “The year’s new drugs & biologics” first appeared in Drugs of Today, the Clarivate journal. They may be downloaded for free here:

Part I

Part II

For additional analysis, learn about the drugs expected to launch in 2018 and forecast to reach blockbuster status in the next five years. Know the Drugs to Watch in 2018 now.