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Keeping up With Drug Approvals at the ASH-FDA Joint Symposium

December 11, 2022

This year, the Joint Symposium of ASH and the U.S. Food and Drug Administration (FDA) is a two-part series to be held on Monday, December 12. The session will showcase new drug and cellular therapy approvals in malignant(10:30 a.m.-12:00 noon, Ernest N. Morial Convention Center, Great Hall AD) and classical hematology (4:30 p.m.-6:00 p.m., Ernest N. Morial Convention Center, Great Hall AD) settings. The first session, titled Something Old, Something New: Recent Immunotherapy and Targeted Agent Approvals, will focus on malignant indications, and the second session, “Three Steps Forward: Approvals in Non-Malignant Hematology” will address classical hematology.

The first session on malignant hematology will start with updates on cellular therapy for multiple myeloma. Drs. Andrea Baines and Dan Vogl will discuss B-cell maturation antigen (BCMA) –targeted chimeric antigen receptor T-cell (CAR-T) therapies, including idecabtagene vicleucel (Abecma), which was approved in March 2021. The session will also spotlight the novel bispecific BCMA-directed CD3 T-cell engager teclistamab, which was recently approved for patients with relapsed/refractory multiple myeloma who have received four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

This will be followed by updates in diffuse large B cell lymphoma (DLBCL) by Drs. Monica Elmacken and Elizabeth Budde. The ZUMA-7 clinical trial focused on promoting axicabtagene-ciloleucel (Yescarta) to second-line therapy for relapsed DLBCL. Similarly, the BELINDA and TRANSFORM trials also evaluated tisagenlecleucel (Kymriah) and lisocabtagene maraleucel (Breyanzi) in the second-line setting for DLBCL, albeit with important differences. Accordingly, the session will address the FDA approval of lisocabtagene maraleucel as second-line therapy for patients with large B-cell lymphoma who are refractory to or relapse within 12 months of first-line chemoimmunotherapy, or for patients with relapsed/refractory disease who are ineligible for stem cell transplantation.

FDA approvals for targeted therapies in newly diagnosed acute myeloid leukemia (AML) will be reviewed by Drs. Ashley Woods and Ehab Atallah. In May 2022, the FDA approved ivosidenib with azacitidine in older/unfit patients with IDH1-mutated newly diagnosed AML based on results from the phase III AG120-C-009, placebo-controlled trial. One criticism of the aformentioned study is that the comparison arm was hypomethylating agent (HMA) therapy alone, instead of combination of HMA with venetoclax, which is standard of care for older/unfit patients with AML.

Drs. Dianne Pulte and Raajit Rampal will then highlight precision medicine in leukemia, discussing the recent approval of pemigatinib in patients with relapsed/refractory myeloid or lymphoid neoplasms (MLN) with fibroblast growth factor receptor 1 (FGFR1) rearrangement. Last, but certainly not the least, Dr. Cara Rabik from the FDA will lead a much-anticipated discussion on a diversity initiative to enroll representative patient populations on clinical trials.

After digesting the malignant approvals, be sure to attend the second ASH-FDA Joint Symposium session on non-malignant (classical) approvals. There will be a focus on approvals in hemolytic anemia, including sutimlimab-jome for cold agglutinin disease (always a hot topic) presented by Drs. Carrie Diamond and David Kuter, and mitapivat for pyruvate kinase deficiency presented by Drs. Tanya Wroblewski and Josef Prchal. Considering the rarity of these disorders, which can be incredibly challenging to treat, we are looking forward to expanding our toolbox. In a similar fashion, the approval of cobimetinib in patients with BRAF V600 mutations associated with histiocytic disorders such as Erdheim-Chester disease, Rosai-Dorfman disease, and Langerhans cell histiocytosis will be presented by Drs. Carrie Diamond and Gaurav Goyal, following impressive results from a phase II trial conducted at Memorial Sloan Kettering Cancer Center.

Lastly, be sure to review the Classification Clarification: Consensus and Compromise session held on Saturday, December 10, for clarifications on the new World Health Organization (WHO), International Consensus (ICC), and European Leukemia Net (ELN) classifications for diagnosis and management of lymphoid and myeloid disorders that are likely to impact clinical trials.

Be sure to bring something along to take notes for these high-impact, heavily sought out sessions! It will make you nostalgic for medical school!

Dr. Blackmon and Dr. Amanam indicated no relevant conflicts of interest.

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