FDA Activity Recap: May 2024 Features 2 Expanded Indication Approvals, 2 RMAT Designations, and More

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Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

Last month, May 2024, the CGTLive® team was diligently tracking the FDA's activities related to the development of cell and gene therapies for the treatment of rare, complex, and otherwise challenging diseases and disorders.

The agency has continued to ramp up its activities around these therapies as more of them progress through the pipeline in tandem. Last month proved no different, with 2 expanded indication approvals for the chimeric antigen receptor T-cell (CAR-T) therapy lisocabtagene maraleucel (liso-cel, marketed as Breyanzi), the granting of regenerative medicine advanced therapy (RMAT) designation to 2 products, and a few other important actions. Our team has highlighted these below.

Click the read more buttons for more details and information about each update.

Liso-Cel/Breyanzi Gets Accelerated Approval for Relapsed/Refractory Follicular Lymphoma

May 15, 2024 — The FDA has granted accelerated approval to Bristol Myers Squibb’s liso-cel CAR T-cell therapy for treating adults with relapsed or refractory follicular lymphoma (FL) who have received 2 or more prior lines of systemic therapy.

“In the treatment of relapsed or refractory follicular lymphoma, patients often cycle through treatments with typically shorter responses with each new line of therapy. Those who have experienced early disease progression have notably poor prognosis,” study investigator M. Lia Palomba, MD, lymphoma and cell therapy specialist, Memorial Sloan Kettering Cancer Center, said in a statement. “The FDA approval of liso-cel for patients with relapsed or refractory FL is an important advancement in addressing an ongoing unmet need in the FL treatment paradigm, providing patients a new option that has shown remarkably high response rates and an established safety profile.”

The approval is based off of data from the TRANSCEND FL trial (NCT04245839) that Palomba served as an investigator on. The most recent data from TRANSCEND FL were presented at the 2023 American Society of Hematology (ASH) in December by Franck Morschhauser, MD, PhD, professor, hematology, University of Lille and Hospital Claude Huriez, and chair, Lymphoma Study Association (LYSA).

Liso-Cel/Breyanzi Approved for Relapsed/Refractory Mantle Cell Lymphoma

May 30, 2024 — The FDA has approved Bristol Myers Squibb’s liso-cel, an autologous CD19-directed CAR T-cell therapy, for treating adults with relapsed/refractory (r/r) mantle cell lymphoma (MCL) who have been previously treated with at least 2 lines of systemic therapy, including a Bruton tyrosine kinase (BTK) inhibitor. MCL is the fourth subtype of nonHodgkin lymphoma that liso-cel has been approved for use in and the product now constitutes the CAR-T with FDA approval for the broadest range of B-cell malignancies

“With Breyanzi, we are delivering on the promise of cell therapy by offering a definitive treatment option for some of the most difficult-to-treat lymphomas,” Bryan Campbell, PharmD, the senior vice president and head of commercial for Cell Therapy at Bristol Myers Squibb, said in a statement. “We are proud of the advances we are making to bring our differentiated CAR T-cell therapy to the most patients across indications and lines of therapy to ensure treatment options that provide improved outcomes are available when most needed.”

Cartesian Therapeutics’ Myasthenia Gravis mRNA CAR-T Descartes-08 Garners FDA RMAT Designation

May 26, 2024 — Cartesian Therapeutics’ Descartes-08, an investigational autologous mRNA-engineered CAR-T therapy intended for the treatment of autoimmune diseases including myasthenia gravis (MG), has received regenerative medicine advanced therapy (RMAT) designation from the FDA for MG.

Descartes-08 is comprised of patients’ own T-cells that have been modified ex-vivo with RNA to target B-Cell Maturation Antigen (BCMA). The use of RNA engineering is intended to avoid risks of genomic integration associated with DNA-based CAR-T therapies and to allow for control of pharmacokinetics.

The CAR-T product is currently being evaluated in the phase 2b portion of a clinical trial (NCT04146051) for MG. The first patient in this portion was dosed in January 2023.

Cartesian Therapeutics’ Myasthenia Gravis mRNA CAR-T Descartes-08 Garners FDA RMAT Designation

Wugen Garners New FDA and EMA Regulatory Designations for T-Cell Malignancy CAR-T WU-CART-007

May 28, 2024 — Wugen’s WU-CART-007, an investigational allogeneic CD7-directed CAR-T therapy intended to treat r/r T-cell acute lymphoblastic leukemia (T-ALL) and r/r T-cell lymphoblastic lymphoma (T-LBL), has been granted RMAT designation by the FDA and priority medicines (PRIME) designation by the European Medicines Agency (EMA).

Both the RMAT and PRIME designation were granted in relation to both aforementioned indications. The regulatory agencies based their decisions on data packages that included results from a phase 1/2 clinical trial (NCT04984356) evaluating the CAR-T in r/r T-ALL and r/r T-LBL.

Alongside the announcement of the new designations, Wugen also reported that it will be presenting new phase 2 trial data at the European Hematology Association (EHA) 2024 Hybrid Congress, which will be held from June 13 to 16, in Madrid, Spain. The company noted that its 2 presentations related to WU-CART-007 at the conference will cover data demonstrating safety and antileukemic activity along with additional findings.

Wugen Garners New FDA and EMA Regulatory Designations for T-Cell Malignancy CAR-T WU-CART-007

AADC Deficiency Gene Therapy up For FDA Priority Review

May 15, 2024 — The FDA has accepted PTC Therapeutics’ biologics license application for eladocagene exuparvovec gene therapy with priority review for treating patients with aromatic L–amino acid decarboxylase (AADC) deficiency with a PDUFA date of November 13, 2024.

Eladocagene exuparvovec is approved in the United Kingdom and European Union under the name Upstaza for children aged 18 months or older with a severe phenotype of AADC deficiency.

"We are excited to be one step closer to bringing an approved therapy to patients with AADC deficiency in the United States," Matthew B. Klein, MD, Chief Executive Officer, PTC Therapeutics, said in a statement. "The data collected to date continue to support the transformative benefit of Upstaza, this highly innovative gene therapy directly infused into the brain."

AADC Deficiency Gene Therapy up For FDA Priority Review
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