Elrexfio (BLA)
(elranatamab-bcmm; Pfizer Inc.)
Class/route: Bispecific B-cell maturation antigen-directed CD3 T-cell engager; injection
Indication: Treatment of adults with relapsed or refractory multiple myeloma who have received ≥4 prior lines of therapy
Approval considerations: Accelerated, orphan status
Approval date: August 14, 2023
Sohonos (NME)
(palovarotene; IPSEN Biopharmaceuticals, Inc)
Class/route: Retinoic acid receptor agonist; capsules
Indication: To reduce the volume of new heterotopic ossification in adults and pediatric patients (aged ≥8 years for females and ≥10 years for males) with fibrodysplasia ossificans progressive
Approval considerations: Priority, orphan status
Approval date: August 16, 2023
Veopoz (BLA)
(pozelimab-bbfg; Regeneron Pharmaceuticals Inc)
Class/route: Complement inhibitor; injection
Indication: Treatment of patients ≥1 year old with CD55-deficient protein-losing enteropathy, also known as CHAPLE disease
Approval considerations: Fast track, orphan status
Approval date: August 18, 2023
Aphexda (NME)
(motixafortide; BioLineRx, Ltd)
Class/route: Hematopoietic stem cell mobilizer; injection
Indication: In combination with filgrastim (a granulocyte colony-stimulating factor) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma
Approval considerations: Orphan status
Approval date: September 8, 2023
Ojjaara (NME)
(momelotinib; GlaxoSmithKline LLC)
Class/route: Janus kinase inhibitor; tablets
Indication: Treatment of intermediate or high-risk myelofibrosis (MF), including primary MF or secondary MF (postpolycythemia vera and post-essential thrombocythemia), in adults with anemia
Approval considerations: Orphan status
Approval date: September 15, 2023
Exxua (NME)
(gepirone; Fabre-Kramer Pharmaceuticals, Inc)
Class/route: Selective serotonin 1a receptor agonist; tablets
Indication: Treatment of major depressive disorder in adults
Approval date: September 22, 2023
Pombiliti (BLA)
(cipaglucosidase alfa-atga; Amicus Therapeutics, Inc.)
Class/route: A hydrolytic lysosomal glycogen-specific enzyme; injection
Indication: In combination with Opfolda, an enzyme stabilizer, for the treatment of adults with late-onset Pompe disease who weigh ≥40 kg and who are not improving on their current enzyme replacement therapy
Approval considerations: Orphan status
Approval date: September 28, 2023
Rivfloza (NME)
(nedosiran; Novo Nordisk Inc)
Class/route: LDHA-directed small interfering RNA; injection
Indication: To lower urinary oxalate levels in children ≥9 years of age and adults with primary hyperoxaluria type 1 and relatively preserved kidney function
Approval considerations: Orphan status
Approval date: September 29, 2023
Velsipity (NME)
(etrasimod; Pfizer Inc.)
Class/route: Sphingosine 1-phosphate receptor modulator; tablets
Indication: Treatment of moderately to severely active ulcerative colitis in adults
Approval date: October 12, 2023
Zilbrysq (NME)
(zilucoplan; UCB, Inc.)
Class/route: Complement inhibitor; injection
Indication: Treatment of generalized myasthenia gravis in adults who are antiacetylcholine-receptor–antibody positive
Approval considerations: Orphan status
Approval date: October 17, 2023
Bimzelx (BLA)
(bimekizumab; UCB, Inc.)
Class/route: Humanized interleukin-17A and F antagonist; injection
Indication: Treatment of moderate to severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy
Approval date: October 17, 2023
Agamree (NME)
(vamorolone; Santhera Pharmaceuticals (Switzerland) Inc.)
Class/route: Corticosteroid; oral suspension
Indication: Treatment of Duchenne muscular dystrophy in patients ≥2 years of age
Approval considerations: Orphan status
Approval date: October 26, 2023
Omvoh (BLA)
(mirikizumab-mrkz; Eli Lilly and Company)
Class/route: Interleukin-23 antagonist; injection
Indication: Treatment of moderately to severely active ulcerative colitis in adults
Approval considerations: Orphan status
Approval date: October 26, 2023
Loqtorzi (BLA)
(toripalimab-tpzi; Coherus BioSciences, Inc.)
Class/route: PD-1-blocking antibody; injection
Indication: In combination with cisplatin and gemcitabine, for first-line treatment of adults with metastatic or with recurrent locally advanced nasopharyngeal carcinoma (NPC); as a single agent for the treatment of adults with recurrent unresectable or metastatic NPC with disease progression on or after a platinum-containing chemotherapy
Approval considerations: Orphan status
Approval date: October 27, 2023
Fruzaqla (NME)
(fruquintinib; Takeda Pharmaceuticals America, Inc.)
Class/route: Kinase inhibitor; capsules
Indication: Treatment of adults with metastatic colorectal cancer who have been previously treated with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, an anti-VEGF therapy, and, if RAS wild-type and medically appropriate, an anti-EGFR therapy
Approval considerations: Priority
Approval date: November 8, 2023
Defencath (NME)
(taurolidine, heparin; CorMedix Inc)
Class/route: A combination of taurolidine, a thiadiazinane antimicrobial, and heparin, an anti-coagulant; sterile catheter lock solution
Indication: To reduce the incidence of catheter-related bloodstream infections in adults with kidney failure receiving chronic hemodialysis through a central venous catheter
Approval considerations: Priority
Approval date: November 15, 2023
Augtyro (NME)
(repotrectinib; Bristol-Myers Squibb Company)
Class/route: Kinase inhibitor; capsules
Indication: Treatment of adults with locally advanced or metastatic ROS1-positive non–small cell lung cancer
Approval considerations: Priority, orphan status
Approval date: November 15, 2023
Ryzneuta (BLA)
(efbemalenograstim alfa-vuxw; Evive Biotechnology Singapore Pte. Ltd)
Class/route: Leukocyte growth factor; injection
Indication: To decrease the incidence of infection, as manifested by febrile neutropenia, in adult patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with a clinically significant incidence of febrile neutropenia
Approval date: November 16, 2023
Truqap (NME)
(capivasertib; AstraZeneca Pharmaceuticals LP)
Class/route: Kinase inhibitor; tablets
Indication: Treatment of adult patients with hormone receptor–positive, HER2-negative, locally advanced or metastatic breast cancer with one or more PIK3CA/AKT1/PTEN-alterations
Approval considerations: Priority
Approval date: November 16, 2023
Ogsiveo (NME)
(nirogacestat hydrobromide; SpringWorks Therapeutics Inc.)
Class/route: Gamma secretase inhibitor; tablets
Indication: Treatment of adult patients with progressing desmoid tumors who require systemic treatment
Approval considerations: Priority, orphan status
Approval date: November 27, 2023
Fabhalta (NME)
(iptacopan hydrochloride; Novartis Pharmaceuticals Corporation)
Class/route: Complement factor B inhibitor; capsules
Indication: Treatment of adults with paroxysmal nocturnal hemoglobinuria
Approval considerations: Priority, orphan status
Approval date: December 5, 2023
Casgevy (BLA)
(exagamglogene autotemcel; Vertex Pharmaceuticals Inc.)
Class/route: Autologous genome edited hematopoietic stem cell-based gene therapy; suspension for intravenous infusion
Indication: Treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events
Approval considerations: Priority review, orphan drug, fast track
Approval date: December 8, 2023
Lyfgenia (BLA)
(lovotibeglogene autotemcel; Bluebird Bio Inc.)
Class/route: Autologous hematopoietic stem cell-based gene therapy; suspension for intravenous infusion
Indication: Treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events
Approval considerations: Priority review, orphan drug, fast track
Approval date: December 8, 2023
Filsuvez (NME)
(birch triterpenes; Amryt Pharmaceuticals DAC)
Class/route: Botanical drug substance; topical gel
Indication: Treatment of wounds associated with dystrophic and junctional epidermolysis bullosa in adult and pediatric patients ≥6 months of age
Approval considerations: Priority, orphan status
Approval date: December 18, 2023
Wainua (NME)
(eplontersen sodium; AstraZeneca Pharmaceuticals LP)
Class/route: Transthyretin-directed antisense oligonucleotide; injection
Indication: Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
Approval considerations: Orphan status
Approval date: December 21, 2023