Six CGT Therapies Expected to Gain FDA Approval in 2024: Pipeline Highlights and Clinical Progress

Feb 14, 2024 15:52 CST Updated 15:52

Geron

Cancer Treatment Product Developer

Gamida Cell

Cell Therapy Drug Developer

Biogen

New Drug Developer

Krystal Biotech

Gene Therapy Developer

Sarepta

Developer of Therapies for Rare Neuromuscular Diseases

CellTrans

Cell Therapy Developer

BioMarin Pharmaceutical

Gene Therapy Developer

Dicerna

RNAi Therapy Developer

Vertex

Breakthrough Small Molecule Drug Developer

BlueBird Bio

Gene Therapy Developer

AstraZeneca

Biopharmaceutical Manufacturer

Legend Biotech

Tumor Cell Immunotherapy Developer

Bristol-Myers Squibb

Biopharmaceutical and Nutritional Product R&D and Sales

CARsgen Therapeutics

Developer of CAR-T Cell Immunotherapy Drugs

Hrain Biotechnology

Developer of Tumor Immunotherapy Technology R&D

Novartis

Drug Development and Manufacturing

Pfizer

Pharmaceutical R&D Developer

Abeona Therapeutics

Cell and Gene Therapy Developer

Rocket Pharmaceuticals

Gene Therapy Developer

Moderna

mRNA Therapeutics Developer

Iovance Biotherapeutics

Developer of Novel Cancer Immunotherapies

Statement:Due to limited proficiency, errors are inevitable, and some information may not be the most up-to-date. Feel free to leave comments pointing them out.This article only provides an introduction to drugs related to healthcare and is not a recommendation for treatment plans (if involved); this article does not constitute any investment advice.

2023 is a harvest year for cell and gene therapy (CGT). According to incomplete statistics, there are 10 CGT therapies approved by the FDA in 2023. As shown in the figure below:

The U.S. FDA predicted a few years ago that by 2025, it would approve 10 to 20 cell and gene therapies annually.Looking Ahead to 2024, Six CGT Therapies Are Expected to Gain FDA Approval. As shown in the figure below:

SPK-9001

Pfizer's SPK-9001 (fidanacogene elaparvovec) is a novel investigational gene therapy that contains a bioengineered adeno-associated virus (AAV) capsid and a codon-optimized, highly active human factor IX gene. Once infused, it enables the continuous endogenous production of factor IX in patients, thereby eliminating the need for regular factor IX infusions.

RP-L201

Gene therapy RP-L201 uses autologous hematopoietic stem cells genetically modified with a lentiviral vector, enabling normal expression of the ITGB2 gene. After patients receive a chemotherapy drug called busulfan, RP-L201 is reintroduced into the patient’s body to remove existing bone marrow cells and make room for the growth of RP-L201. RP-L201 has received Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease, and Fast Track designations from the FDA, as well as PRIME and Advanced Therapy Medicinal Product (ATMP) designations in the EU, and Orphan Drug designation in both regions.

Rocket Pharmaceuticals R&D Pipeline

EB-101

PZ-cel (prademagene zamikeracel, EB-101) is an investigational autologous cell therapy consisting of epidermal sheets designed to deliver the functional COL7A1 gene into a patient’s own skin cells to achieve normal type VII collagen expression and promote wound healing. PZ-cel has previously been granted Regenerative Medicine Advanced Therapy (RMAT), Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease designations by the U.S. FDA.

Abeona Therapeutics Pipeline

lifileucel

Iifileucel (Other names: LN-144, Contego) is an off-the-shelf autologous cell therapy product containing tumor-infiltrating lymphocytes (TIL). After showing significant results in the pivotal Phase II innovaTIL-01 clinical trial, it is expected to become the first approved TIL therapy and the first approved T-cell therapy for the treatment of solid tumors.

Iovance Biotherapeutics Pipeline

imetelstat

Imetelstat is a "First-in-Class" telomerase inhibitor developed by Geron Corporation for the treatment of transfusion-dependent anemia in patients with lower-risk myelodysplastic syndromes (MDS).

In June 2023, Geron submitted the NDA for Imetelstat to the FDA. On August 21, 2023, the FDA accepted the NDA for Imetelstat, with a PDUFA date of June 16, 2024.

Geron R&D Pipeline

mRNA-1345

mRNA-1345 is an RSV vaccine targeting the pre-fusion F protein, utilizing the same lipid nanoparticles (LNP) as Moderna's COVID-19 vaccine and containing optimized protein and codon sequences.

Phase III clinical data of mRNA-1345 showed that the efficacy of mRNA-1345 in preventing RSV-related lower respiratory tract diseases reached 83.7%. Moderna has submitted marketing applications for mRNA-1345 in multiple countries and is expected to be approved for marketing in 2024.

Moderna Partial R&D Pipeline

Looking at the 2023 CGT therapy revenue, it shows a significant growth trend:

In 2023,Legend BiotechCAR-T Therapy Sales Reach $500 Million, Up 276% Year-on-Year; Expected to Break $1 Billion in 2024.
BlueBird BioTwo gene therapies, ZYNTEGLO and SKYSONA, generated total revenue of $21.66 million in Q1~Q3 2023, marking a year-over-year increase of 681%.
SareptaFirst DMD Gene Therapy Achieves $200.4 Million in Sales in 2023, Exceeding Expectations by a Wide Margin.
BMSTwo CAR-T therapies, Abecma and Breyanzi, generated revenues of $472 million (+22%) and $364 million (100%), respectively.
2023NovartisLeqvio, an siRNA therapy, generated $355 million in revenue, a year-over-year increase of 217%. Kymriah, a CAR-T therapy, saw its sales decline by 5% to $508 million in 2023. Zolgensma, a gene therapy for SMA, recorded $1.214 billion in sales for 2023, representing an 11% decrease compared to the previous year.

In China, from January to September 2023, the total number of CGT therapies accepted and granted clinical trial authorization reached 133.In 2024,CARsgen Therapeutics, LtdZevor-cel,Shanghai Hengrun Dasheng Biotechnology Co., Ltd.Runda Gene Therapy Injection Also Expected to Be ApprovedAccording to Frost & Sullivan statistics, the cell therapy market size in China will grow from 3.3 billion yuan in 2021 to 58.4 billion yuan in 2030, with a CAGR as high as 53%.

In summary, there are substantial unmet clinical needs in the CGT field both in China and abroad.

References:

Official Websites of Various Companies