New Drugs: The FDA Enters the New

The FDA Enters the New Year with “2020” Vision

This past quarter was another banner period during which 24 unique drugs or biologics were approved, along with approval for several new formulations as well as significant new indications which are beyond the scope of this column.

Biologicals: Ervebo® (Ebola Zaire, live), through a tropical disease priority review and breakthrough therapy designation, is the first vaccine for the prevention of Ebola virus disease. Jynneos™ (smallpox and monkeypox, live, non-replicating) received priority review as the first vaccine to prevent monkeypox and the first of its type indicated to prevent both infections, and has been included in the Strategic National Stockpile. Beovu® (brolucizumab) is a vascular endothelial growth factor inhibitor indicated to treat wet age-related macular degeneration. Givlaari™ (givosiran), through priority review with orphan drug status, was approved as a once-monthly SQ injection to treat acute hepatic porphyria as an interfering RNA. Vyondys 53™ (golodirsen) is the first treatment for Duchenne muscular dystrophy and uses exon-skipping technology in patients with a confirmed mutation amenable to exon 53 skipping.

Hematology/Oncology: Two drugs were approved to treat sickle cell disease. Adakveo® (crizanlizumab-tmca) was approved as an orphan drug through priority review by inhibiting selectin which contributes to RBC stickiness leading to vasoocclusive crisis. Oxbryta™ (voxelotor) received accelerated approval as a deoxygenated sickle hemoglobin polymerization which is the central abnormality in sickle cell disease. Reblozyl® (luspatercept-aamt) received fast-track approval as an orphan drug to treat beta thalassemia by reducing the number of blood transfusions that poses a risk of iron overload. Brukinsa™ (zanubrutinib) is a kinase inhibitor receiving accelerated approval as an orphan drug for second-line treatment of mantle cell lymphoma. Enhertu® (fam-trastuzumab deruxtecan-nxki) was granted a breakthrough therapy designation through fasttrack review as a HER2-directed antibody and topoisomerase inhibitor conjugate indicated to treat unresectable or metastatic HER2+ breast cancer. Padcev™ (enfortumab vedotin-ejfv) was granted priority review with breakthrough therapy designation as a first-in-class agent directed against cell adhesion nectin-4 and indicated to treat advanced or metastatic urothelial cancer.

Chronic Care: Aklief® (trifarotene) is the first new retinoid cream approved in 20-years to treat acne vulgaris. Ibsrela® (tenapanor) is a minimally-absorbed NHE3 inhibitor approved for irritable bowel syndrome with constipation. Trikafta™ (elexacaftor, tezacaftor and ivacaftor) received fast-track, breakthrough, and orphan drug designation as the first triple combination therapy approved to treat the most common F508del mutation in cystic fibrosis. Vumerity™ (diroximel fumarate), similar to dimethyl fumarate (Tecfidera™) was approved for relapsing forms of multiple sclerosis. Xcopri® (cenobamate) received approval to treat partial-onset seizures.

Acute Care: Fetroja® (cefiderocol) is the only antibiotic approved this quarter and is a cephalosporin indicated to treat gram-negative complicated UTIs. Two oral therapies were also approved to treat acute migraines with/without aura. Reyvow™ (lasmiditan) works as a serotonin 1F receptor agonist, while Ubrelvy™ (ubrogepant) acts as a calcitonin gene-related peptide receptor antagonist. Finally, Scenesse® (afamelanotide) is a melanocortin-1 receptor agonist and the first treatment to ease phototoxic reactions in patients with erythropoietic protoporphyria.

Diagnostic Aids: ExEm® Foam (air polymer-type A) is an ultrasound contrast agent indicated to help assess fallopian tube patency. Fluorodopa F 18™ (xxx) is a radioactive diagnostic agent for use with PET indicated to assess Parkinsonian syndromes. Ga 68 DOTATOC™ (xxx) is a radioactive diagnostic agent for use with PET indicated to assess somatostatin-positive neuroendocrine tumors. TissueBlue (Brilliant Blue G ophthalmic solution) is a disclosing agent indicated to selectively stain the internal limiting membrane on the surface of the retina.

New Dosage Forms: Significant new dosage forms approved this quarter include: Abrilada™ (adalimumab-afzb, SQ injection) a biosimilar to Humira®; Absorica LD™ (isotretinoin, capsules) for severe recalcitrant nodular acne; Amzeeq™ (minocycline, topical foam) for acne vulgaris; Aralzo™ (tazarotene, lotion) for acne vulgaris; Biorphen™ (phenylephrine, ready-to-use) for hypotension in acute care; Bonsity™ (teriparatide, SQ injection) for osteoporosis; Exservan™ (riluzole, oral film) for amyotrophic lateral sclerosis (Lou Gehrig’s disease); Gvoke™ (glucagon, auto-injector) for hypoglycemia; Hemady™ (dexamethasone, 20mg tablet) for multiple myeloma; Katerzia™ (amlodipine, oral suspension) for hypertension and CAD; Nouress™ (cysteine HCl, injection) for neonatal TPN; Ozobax™ (baclofen, oral solution) for spasticity; Quzyttir™ (cetirizine, injection) for acute urticaria; Reditrex™ (methotrexate, SQ injection) for rheumatoid arthritis and psoriasis; Riomet ER™ (metformin, extendedrelease oral suspension) for type 2 diabetes; Rybelsus® (semaglutide, tablet) for type 2 diabetes; Secuado® (asenapine, transdermal patch) for schizophrenia; Slynd® (drospirenone, tablet) to prevent pregnancy; Talicia™ (omeprazole, amoxicillin, and rifabutin, delayed-release capsule) for Helicobacter pylori infection; and Ziextenzo™ (pegfilgrastim-bmez, SQ injection) a biosimilar to Neulasta®. §