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Note: The following symbols are used throughout.

orphan indications

$ breakthrough therapies

# cancer therapies

biologic license designations


In 2018, the U.S. Food and Drug Administration (FDA) granted 46 noteworthy new drug application (NDA) licenses and approved 29 noteworthy new biologic license applications (BLAs). The 46 NDA approvals are cataloged in Part 1. Among them are:

  • Thirty-three new molecular entities that are pharmacologically similar to previously approved drugs;

  • Nine pharmacological “firsts,” including a breakthrough gene silencing therapy employing a small interfering RNA (siRNA) mechanism and a “tissue-agnostic” solid tumor cancer drug;

  • Three ablative agents, including 2 radioablatives for the treatment of cancer; and

  • A reintroduced OTC metered-dose epinephrine inhaler for the self-management of mild asthma.

Part 1 looked back at first-in-class and “me-too” NDA approvals, breakthrough ($) therapies, orphan indications (), cancer drugs (#), and companion diagnostics for precision cancer therapy. Other 2018 noteworthy FDA accomplishments, including previously marketed drugs licensed for expanded indications, novel dosage forms, new fixed-dose combination products, and innovative new devices, are summarized in Part 3.

Beginning in 2015, FDA began appending four-letter suffixes to all FDA-approved biosimilars. These are random, nonmeaningful 4-letter sequences for distinguishing one presentation of an approved biologic from another.1 Late in 2017, FDA extended the requirement for 4-letter generic name suffixes to all new biologic license approvals. The suffixes are used by FDA to facilitate pharmacovigilance activities, including facilitating future clinical differentiation when follow-on biologics or biobetters are approved for different uses or with different safety profiles than the reference (first-to-market) biologics.1 Unless clinically meaningful, biologic nonproprietary name suffixes are omitted from this part of the series.

Biologic First-In-Class and Pharmacological Similars

The 29 noteworthy new biologic licenses approved in 2018 include:

  • Twelve first-in-class licenses (Table P2-1), nine of which were approved with an orphan designation for a rare disease, including three rare forms of cancer:

    • Eight monoclonal antibodies (burosumab-twza$ [Crysvita]2 for X-linked hypophosphatemia; emapalumab-lzsg$ [Gamifant]3 for hemophagocytic lymphohistiocytosis; erenumab-aooe [Aimovig]4, fremanezumab-vfrm [Ajovy]5, and galcanezumab-gnlm [Emgality]6 for migraine; ibalizumab-uiyk$ [Trogarzo]7 for HIV-1 infection; lanadelumab-flyo$ [Takhzyro]8 for hereditary angioedema; and mogamulizumab-kpkc#$ [Poteligeo]9 for mycosis fungoides and Sézary syndrome; and

    • Four products for: neurotrophic keratitis (cenegermin-bkbj$ [Oxervate]),10 reversal of anticoagulation (coagulation factor Xa (recombinant), inactivated-zhzo [Andexxa]),11 phenylketonuria (pegvaliase-pqpz [Palynziq]),12 and dendritic cell cancer (tagraxofusp-erzs$# [Elzonris]).13

  • Seventeen “me-too” products (Table P2-2) that are pharmacologically similar to previously approved drugs:

    • Seven biosimilars, none of which are “interchangeable” with the reference biologic14-16 (adalimumab-adbm, epoetin alfa-epbx, filgrastim-aafi, pegfilgrastim-cbqv, pegfilgrastim-jmdb, ...

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