eChapter 2017 Part 2: The Goodman & Gilman Year in Review: 2017 New and Noteworthy FDA Approvals

PART 2: BIOLOGIC APPROVALS

In 2017 the U.S. Food and Drug Administration (FDA) approved 40 new drugs and granted 48 noteworthy biologic licenses. The 40 new drugs are cataloged in Part 1. Among them are:

• Seven pharmacological “firsts;”

• Twenty-eight new molecular entities that are pharmacologically similar to previously approved small molecule drugs;

• Three “follow-on” products (see Part 3); and

• Two previously unapproved marketed drugs (see Part 3).

Part 1 looked back at first-in-class and “me-too” small molecule drug approvals, breakthrough therapies, and new companion diagnostic tests for therapies targeting cancer. Other 2017 noteworthy FDA accomplishments, including previously marketed drugs licensed for expanded indications, novel dosage forms, and new fixed-dose combination products are summarized in Part 3. Throughout the series orphan (†), breakthrough ($), cancer therapy (#), and biologic (¶) designations are notated by the symbols †, $, #, and ¶, respectively. Biologic notations (¶) are omitted from this part of the series.

The 48 noteworthy new biologic licenses approved in 2017 include:

• Ten first-in-class market entrants: (Table P2-1)

  • Three gene therapies (axicabtagene ciloleucel [Yescarta]¹ for lymphoma^†$#, tisagenlecleucel [Kymriah]² for leukemia^†$#, and voretigene neparvovec-rzyl [Luxturna]³ for retinal dystrophy^†$);

  • Three monoclonal antibodies (dupilumab^† [Dupixent]⁴ for atopic dermatitis^$, emicizumab-kxwh [Hemlibra]⁵ for hemophilia^†$, and guselkumab [Tremfya]⁶ for psoriasis);

  • Two antibody-drug conjugates⁷ for leukemia^# (gemtuzumab ozogamicin [Mylotarg]⁸ and inotuzumab ozogamicin [Besponsa]⁹); and

  • Two enzyme therapies for inborn errors of metabolism (cerliponase alfa [Brineura]¹⁰ for tripeptidyl peptidase 1 deficiency^†$ and vestronidase alfa-vjbk [Mepsevii]¹¹ for beta-glucuronidase deficiency^†)

• Six monoclonal antibodies functionally similar to others (Table P2-2)

• Six “me-too” biologics (Table P2-3)

• 5 biosimilars (none are “interchangeable”)¹² (adalimumab-adbm,¹³ bevacizumab-awwb^#,14 infliximab-abda,¹⁵ infliximab-qbtx,¹⁶ and trastuzumab-dkst^#17) (Table P2-2)

• Two improved-efficacy vaccines formulated with Toll-like receptor (TLR) agonist adjuvants: (see below and Table P2-3)

  • Recombinant hepatitis B vaccine formulated with the TLR-9 agonist adjuvant CpG 1018 ISS (Heplisav-B);¹⁸

  • Recombinant zoster vaccine formulated with the TLR-4 agonist monophosphoryl lipid A (Shingrix)¹⁹

• Four new formulations:

  • α₁-proteinase inhibitor (Prolastin-c)²⁰ formulated as a liquid; (Table P2-3)

  • Belimumab (Benlysta)²¹ formulated for weekly subcutaneous self-administration for the management of lupus;²² (Table P2-2)

  • Dust mite allergen extract formulated as a sublingual tablet (Odactra);²³ (Table P2-3)

  • Rituximab (Rituxan Hycela)²⁴ formulated with hyaluronidase (Table P2-2)

• Fifteen marketed biologics with expanded indications, including a new formulation of C1 esterase inhibitor (Haegarda)²⁵ enabling twice-weekly subcutaneous self-administration as routine prophylaxis against attacks of hereditary angioedema (Table P2-3).