FDA Drug Approvals Q2 2023

Mary L. Windle, PharmD


July 28, 2023

In This Article


Columvi (glofitamab)

Glofitamab is a bispecific CD3-directed and CD20-directed antibody that binds to CD20 expressed on the surface of B cells and to CD3 receptor expressed on the surface of T cells. It causes T-cell activation and proliferation, secretion of cytokines, and lysis of CD20-expressing B cells. Glofitamab is indicated for adults with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) not otherwise specified or large B-cell lymphoma (LBCL) arising from follicular lymphoma, after 2 or more lines of systemic therapy. 

Accelerated approval was granted in June 2023 and supported by the phase I/II NP30179 study. Glofitamab was given as a fixed course with DLBCL in patients who had relapsed or were refractory to prior therapies, including about 30% who had received prior CAR T-cell therapy. Additionally, 83% were refractory to their most recent therapy. At a median follow-up of 12.6 months, 39% had a complete response, according to independent review. Results were consistent among the 52 patients who had previously received chimeric antigen receptor T-cell therapy (35% of whom had a complete response). The majority (78%) of complete responses were ongoing at 12 months. The 12-month progression-free survival was 37%. N Engl J Med. 2022 Dec 15;387(24):2220-2231

Epkinly (epcoritamab)

Epcoritamab is indicated for relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from indolent lymphoma, and high-grade B-cell lymphoma after at least 2 lines of systemic therapy. It is an IgG1-bispecific antibody designed to simultaneously bind to CD3 on T cells and CD20 on B cells and to induce T-cell mediated lysis of CD20+ cells. 

Approval was based on the phase 2 EPCORE NHL-1 trial. The trial's efficacy population included 148 adults with relapsed or refractory CD20+ large B-cell lymphoma who had received at least 2 prior lines of therapy, including anti-CD20 therapies. Almost 40% had undergone CAR T-cell therapy. Epcoritamab was administered initially once weekly, then every 2 weeks, and then every 4 weeks until disease progression or unacceptable toxicity. The trial had no comparator arm. At a median follow-up of 10.7 months, the overall response rate was 61%, and the complete response rate was 38%. At a median follow-up of 9.8 months among responders, the median duration of response was 15.6 months. J Clin Oncol. 2023 Apr 20;41(12):2238-2247

Omisirge (omidubicel)

Omidubicel is a nicotinamide (NAM)-modified allogeneic hematopoietic progenitor cell (HPC) therapy derived from cord blood used as an allogeneic stem cell donor source. Ex-vivo culturing of cord blood–derived HPCs in the presence of NAM leads to preservation of their stemness, homing to the bone marrow (BM) and retained engraftment capacity, as demonstrated by rapid neutrophil engraftment and multilineage immune reconstitution as observed in clinical trials. Omidubicel is indicated for hematologic malignancies in patients aged 12 years and older who are planned for umbilical cord blood transplantation following myeloablative conditioning, to reduce neutrophil recovery time and incidence of infection. 

Evidence in a substudy characterizing immune reconstitution (IR) kinetics following hematopoietic cell transplantation showed omidubicel efficiently promoted IR across multiple immune cells, including CD4+ T cells, B cells, NK cells, and dendritic cell subtypes as early as 7 days post-transplant, potentially endowing recipients of omidubicel with early protective immunity. Transplant Cell Ther. 2023 Apr 27;S2666-6367.

When compared with standard umbilical cord blood transplantation, omidubicel had a median time to neutrophil engraftment of 12 days versus 22 days for the control arm (P<0.001). Blood. 2021 Oct 21;138(16):1429-1440.

Roctavian (valoctocogene roxaparvovec)

The FDA approved the first gene therapy for hemophilia A, valoctocogene roxaparvovec, in June 2023. It is a one-time, single-dose IV adeno-associated virus vector-based gene therapy indicated for severe hemophilia A (congenital factor VIII deficiency with factor VIII activity <1 IU/dL) in adults without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test. 

Approval was based on the phase 3, open-label, single-group GENEr8-1 trial (n=134). Among the 132 HIV-negative participants, the mean factor VIII activity level at Weeks 49 through 52 increased by 41.9 IU/dL (P<0.001; median change, 22.9 IU/dL). The 112 participants enrolled in a prospective, noninterventional study had decreased annualized rates of factor VIII concentrate use and treated bleeding after Week 4 following infusion of 98.6% and 83.8%, respectively (P<0.001 for both comparisons). N Engl J Med. 2022 Mar 17;386(11):1013-1025

Posluma (flotufolastat F 18)

Flotufolastat F 18 is indicated for positron emission tomography (PET) of prostate-specific membrane antigen (PSMA)–positive lesions in men with prostate cancer who have the following:

  • Suspected metastasis who are candidates for initial definitive therapy

  • Suspected recurrence based on elevated serum prostate-specific antigen (PSA) level 

The agent binds to PSMA (IC50 = 4.4 nM) expressed on cells, including prostate cancer cells, and is internalized. Fluorine-18 is a beta-positive–emitting radionuclide that can be detected using positron emission tomography. 

Approval was supported by the SPOTLIGHT and LIGHTHOUSE studies. These studies determined flotufolastat F 18 to be a useful staging tool for distant metastatic lesions.

Other hematology/oncology approvals

Ayvakit (avapritinib) – New indication approved for indolent systemic mastocytosis 

Lynparza (olaparib) – Indicated in combination with abiraterone and prednisone (or prednisolone) for adults with deleterious or suspected deleterious BRCA-mutated (BRCAm) metastatic castration-resistant prostate cancer (mCRPC) 

Talzenna (talazoparib) – New indication for homologous recombination repair (HRR) gene-mutated metastatic castration-resistant prostate cancer (mCRPC) in combination with enzalutamide 

Padcev (enfortumab vedotin) plus pembrolizumab (Keytruda) – New indication for patients with locally advanced or metastatic urothelial carcinoma who are ineligible for cisplatin-containing chemotherapy  

Polivy (polatuzumab vedotin) – New indication for use in combination with rituximab, cyclophosphamide, doxorubicin, and prednisone for treatment of adults who have previously untreated diffuse large B-cell lymphoma 

Injectafer (ferric carboxymaltose) – New indication approved for treatment of iron deficiency in adults with heart failure and New York Heart Association Class II/III to improve exercise capacity 

Coagadex (Factor X, human) – Indication for management of perioperative bleeding expanded to include patients with moderate or severe hereditary factor X deficiency 

Ultravist (iopromide) – New indication for contrast-enhanced mammography (CEM)


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