Regulatory Roundup 1Q 2024

April 4, 2024
Regulatory Roundup Q1


The first quarter of 2024 has brought approvals for a novel cell therapy—the first TIL for a solid tumor—a novel gene therapy for a rare disease, and additional indications for 2 other CGT products.  

The first cell therapy for a solid tumor, Iovance Biotherapeutics’ AmtagviTM (lifileucel) received accelerated approval on February 15, 2024. Amtagvi is for treatment of adult patients with unresectable or metastatic melanoma previously treated with a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitor.

Orchard Therapeutics’ LenmeldyTM (atidarsagene autotemcel) was approved on March 18, 2024. Lenmeldy is indicated for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD). 

On January 16, 2024 , Vertex’s CasgevyTM (exagamglogene autotemcel) received approval for a second indication on the treatment of patients aged 12 years and older with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) and transfusion-dependent ß-thalassemia (TDT).

A notable additional indication for Juno Therapeutics/BMS’ Breyanzi® (lisocabtagene maraleucel) was granted accelerated approval on March 14, 2024: relapsed/ refractory chronic lymphocytic leukemia or small lymphocytic lymphoma in patients who have received at least two prior lines of therapy including a Bruton’s tyrosine kinase (BTK) inhibitor and a B-cell lymphoma-2 (BCL-2) inhibitor.

In a press release issued February 13, 2024, Rocket Pharmaceuticals announced that for KRESLADI™ for severe Leukocyte Adhesion Deficiency-I (LAD-I), “FDA extended the review period by three months, to June 30, 2024, to allow additional time to review clarifying Chemistry, Manufacturing, and Controls (CMC) information submitted by Rocket in response to FDA information requests.”

II. CGT BLAs under US FDA Review 

The following information on BLAs under US FDA review was obtained from company press releases.


No upcoming meetings of the Cellular, Tissue, and Gene Therapies Advisory committee have been announced.


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