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 FDA approval is contingent on demonstration of a clinical benefit in a confirmatory trial)
¶ biologic license designation
First-In-Class and Pharmacological Similars
In the first half of 2020, the U.S. Food and Drug Administration (FDA) granted 23 noteworthy new drug application (NDA) licenses and approved 13 noteworthy new biologic license applications (BLAs). Among the 23 NDA approvals are:
Five pharmacological “firsts” (avapritinib$# and ripretinib$# for gastrointestinal stromal tumors†; bempedoic acid [alone and in combination with ezetimibe] for hypercholesterolemia; selpercatinib$# for non-small cell lung cancer!† and thyroid cancers!†; and tazemetostat# for epithelioid sarcoma!† and follicular lymphoma!†) (Table P1-1);
Thirteen new molecular entities that are pharmacologically similar to previously approved drugs (amisulpride for postoperative nausea and vomiting, artesunate$ for parenteral therapy of malaria†; capmatinib$# for non-small cell lung cancer†;1 lactitol for chronic idiopathic constipation, lurbinectedin# for small cell lung cancer!†; monomethyl fumarate for multiple sclerosis, opicapone for Parkinson disease, osilodrostat for Cushing disease†, ozanimod for multiple sclerosis, pemigatinib$# for cholangiocarcinoma†, rimegepant for migraine, selumetinib$# for neurofibromatosis†, and tucatinib$ for metastatic breast cancer†) (Table P1-2);
Two radioactive positron emission tomography (PET) imaging agents (flortaucipir f-18 [TAUVID]2,3 first in class for the detection of aggregated tau neurofibrillary tangles in adults being evaluated for Alzheimer disease; and fluoroestradiol f-18 [CERIANNA]4 for the detection of estrogen receptor–positive lesions associated with breast cancer);
Citric acid, lactic acid, and potassium bitartrate (PHEXXI),5 a first-in-class vaginal pH regulator gel for nonhormonal on-demand contraception;
Footnote 1Fenfluramine [C-IV] (FINTEPLA)11,12 for seizures associated with Dravet syndrome† in children >2 years of age); and
Triheptanoin (DOJOLVI),13 a medium-chain triglyceride, used as a triglyceride regulator and source of fat and calories for patients with long-chain fatty acid oxidation disorders†.
The 13 noteworthy new biologic licenses (¶) approved in the first half of 2020 include: