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PART 1: FIRST-IN-CLASS DRUGS, PHARMACOLOGICAL SIMILARS, BREAKTHROUGH THERAPIES, ORPHAN INDICATIONS, CANCER APPROVALS, AND COMPANION DIAGNOSTIC TESTS

Note: The following symbols are used throughout.

! Accelerated approval (final approval contingent on demonstration of a clinical benefit in a confirmatory trial)

Biologic license designation

$Breakthrough therapy

#Cancer drug

Orphan indication

A Note About Melphalan Flufenamide

Melphalan flufenamide!# (PEPAXTO)1 is a peptide-conjugated prodrug of melphalan, an alkylating drug. Accelerated approval for relapsed or refractory multiple myeloma was granted by the U.S. Food and Drug Administration (FDA) on February 26, 2021. A safety signal related to survival emerged prior to widespread marketing, and FDA approval was withdrawn on October 22, 2021.1–4 Melphalan flufenamide is excluded from the FDA approvals compiled below.

First-In-Class and Pharmacological Similars

In the first half of 2021, the FDA granted 21 noteworthy new drug application (NDA) licenses and approved 10 noteworthy new biologic license applications (BLAs). Among the 21 NDA approvals are:

  • Three pharmacological “firsts”: (Table P1-1)

    • Fosdenopterin$ for molybdenum cofactor deficiency,5

    • Ibrexafungerp for vulvovaginal candidiasis, and

    • Sotorasib!$# for KRAS-G12C-mutated non-small cell lung cancer6–8

  • Fourteen new molecular entities that are pharmacologically similar to previously approved drugs: (Table P1-2)

    • Brincidofovir, a lipid conjugate of cidofovir for smallpox;9

    • (Footnote 1a) Cabotegravir, an integrase strand transfer inhibitor for HIV-1 infection, presented in both extended-release injectable suspension for intramuscular (IM) administration and oral tablet formulations;10

    • Casimersen!, an antisense oligonucleotide indicated for Duchenne muscular dystrophy;13

    • Dasiglucagon, a glucagon analog for hypoglycemia;

    • Infigratinib#, a kinase inhibitor for cholangiocarcinoma;14

    • Pegcetacoplan, a complement inhibitor for paroxysmal nocturnal hemoglobinuria;

    • Ponesimod, a sphingosine 1-phosphate receptor modulator for multiple sclerosis;

    • Tepotinib!#, a kinase inhibitor for non-small cell lung cancer harboring mesenchymal-epithelial transition (MET) exon 14-skipping alterations;15

    • Tivozanib#, a kinase inhibitor for renal cell carcinoma;16

    • Trilaciclib$#, a kinase inhibitor for adjunctive therapy of small cell lung cancer;17

    • Umbralisib#, a kinase inhibitor for follicular!, splenic marginal zone!$, extranodal marginal zone, and nodal marginal zone lymphomas;18

    • Vericiguat, a soluble guanylate cyclase stimulator for heart failure;

    • Viloxazine, a norepinephrine reuptake inhibitor for attention deficit hyperactivity disorder; and

    • Voclosporin, a calcineurin inhibitor immunosuppressant for lupus nephritis.

  • Three combination drug products containing new molecular entities that are pharmacologically similar to previously approved drugs:

    • AZDTARYS [C-II]:19 serdexmethylphenidate (C-IV),20 a prodrug of dexmethylphenidate (C-II), formulated in combination with dexmethylphenidate for attention deficit disorder;

    • LYBALVI:21 samidorphan22, a μ-opioid receptor antagonist ...

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