FDA Drug Approvals January-March 2023

Mary L. Windle, PharmD


May 01, 2023

In This Article

Orserdu (elacestrant)

Elacestrant is indicated for postmenopausal women or adult men with estrogen receptor-positive (ER+), HER2-negative, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least 1 line of endocrine therapy. It is the first oral selective ER degrader.

Approval was based on the EMERALD phase 3 trial. Patients were randomly assigned to elacestrant (n = 239) or standard of care (SOC) (n = 238). Progression-free survival (PFS) was prolonged in all patients and those with ESR1 mutation who received elacestrant (P = 0.002 and 0.0005, respectively). J Clin Oncol. 2022 Oct 1;40(28):3246-3256

Jaypirca (pirtobrutinib)

Pirtobrutinib is a non-covalent bruton tyrosine kinase (BTK) inhibitor. It gained accelerated approval for adults with relapsed or refractory mantle cell lymphoma after at least 2 lines of systemic therapy, including a covalent BTK inhibitor (eg, ibrutinib, acalabrutinib, zanubrutinib).

Approval was based on the BRUIN phase I/II trial, which showed objective response rate (ORR) of 50%, complete response (CR) of 13%, and partial response (PR) of 38% in patients treated with pirtobrutinib. Median duration of response (DOR) at 6 months was 65.3%. An open-label phase 3 study is underway to confirm these results. Clin Lymphoma Myeloma Leuk. 2022 Oct;22 Suppl 2:S394-395

Zynyz (retifanlimab)

Retifanlimab is indicated for metastatic or recurrent locally advanced Merkel cell carcinoma (MCC). It is a monoclonal antibody that targets checkpoint inhibitor PD-1 (programmed death 1) and blocks its interaction with the PD-1 ligands PD-L1 and PD-L2 to suppress tumor growth.

Approval was based on data from the phase 2 POD1UM-201 trial, an open-label, multiregional, single-arm study that evaluated retifanlimab in adults with metastatic or recurrent locally advanced MCC who had not received prior systemic therapy for their advanced disease. Among chemotherapy-naive patients (n = 65), retifanlimab monotherapy resulted in an ORR of 52%. Among those who responded, 12 patients (18%) achieved CR, and 22 patients (34%) showed PR. Among the responding patients, the DOR ranged from 1.1 to over 24.9 months, 76% (26/34) experienced a DOR of 6 months or longer, and 62% (21/34) experienced a DOR of 12 months or longer by landmark analysis. Immunother Cancer. 2021;9(suppl 2).

Other hematology/oncology approvals

New indications

Tukysa (tucatinib) – Accelerated approval in combination with trastuzumab (Herceptin) for adults with RAS wild-type, HER2-positive unresectable or metastatic colorectal cancer (CRC) that has progressed after treatment with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy

Brukinsa (zanubrutinib) – Indicated for chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL)

Keytruda (pembrolizumab) – Indicated as a single agent for adjuvant treatment following resection and platinum-based chemotherapy for adults with stage IB (T2a >4 cm), II, or IIIA NSCLC

Trodelvy (sacituzumab govitecan) – Indicated for unresectable locally advanced or metastatic hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative (IHC 0, IHC 1+ or IHC 2+/ISH–) breast cancer

Verzenio (abemaciclib) – Indication for high-risk breast cancer no longer requires Ki-67 testing

Opdivo (nivolumab) – Indicated for melanoma in patients aged 12 years and older with lymph node involvement or metastatic disease who have undergone complete resection, in the adjuvant setting

Tafinlar (dabrafenib) – New indication for children with low-grade glioma in combination with Mekinist (trametinib)

Enjaymo (sutimlimab) – Indicated for treatment of hemolysis in adults with cold agglutinin disease; previously indicated to decrease the need for RBC transfusion due to hemolysis associated with CAD


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