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FDA Annual Review | Record-breaking number of new drug approvals in 2023!

The curtain has fallen on 2023, with 2024 ushering in a new chapter! Looking back at 2023, the momentum of FDA new drug approvals was as hot as ever. The total number of new drugs approved by the FDA in 2023 hit a five-year high! The Center for Drug Evaluation and Research (CDER) approved 55 innovative drugs. The Center for Biologics Evaluation and Research (CBER) approved 7 cell and gene therapy products, which included the world's first approved CRISPR gene editing therapy, Casgevy, two cell therapy products, and the first oral fecal microbiota product, Vowst.

With the development of science and technology, the demand for precision in disease treatment is increasing. Cell and Gene Therapy (CGT) drugs refer to the use of biotechnology methods to modify individual gene expression or repair abnormal genes through gene addition, gene modification, gene silencing, etc., in order to achieve precise cure of diseases. Specific drug forms include gene editing technology, stem cell therapy, and CAR-T cell therapy.

Since the emergence of CGT drugs, encouraging clinical trial results have been created, including improving the complete remission rate of cancer and achieving blindness reversal caused by specific gene mutations, bringing new hope to patients. According to authoritative predictions, Frost&Sullivan predicts that the global market size in the CGT field is expected to reach 30.54 billion US dollars by 2025, corresponding to a CAGR of 71.2% from 2020 to 2025.

Figure 1. mIHC technique to detect multispectral images

In recent years, the number of approvals for CGT new drugs has been increasing year by year. In the past 2023, the FDA has successively approved 11 CGT drugs, of which 7 were first approved, accounting for 11.3% of the new drugs approved in 2023. What specific CGT products are worth paying attention to? The editor has compiled relevant information and will take you through a brief review.

Product name
（Trade name）		 Type Description
Talimogene
laherparepvec
(IMLYGIC)
2023/2/10		 Gene therapy Talimogene laherparepvec (TVEC) is a genetically modified type I herpes simplex virus. It can preferentially replicate in tumor cells, enhance the antigen loading of MHC class I molecules. It also expresses granulocytes, macrophages, and colony stimulating factors to increase tumor antigen presentation in dendritic cells[1]. It is a "first in class" oncolytic virus therapy. This therapy has been approved for local treatment of unresectable skin, subcutaneous, and lymph node lesions in patients with recurrent melanoma after initial surgery.
Omidubicel-onlv
(OMISIRGE)
2023/4/17 *		 Cell therapy Omidubicel-onlv originates from umbilical cord blood (UCB) and is a donor source for nicotinamide modified allogeneic hematopoietic progenitor cell therapy[2].This therapy is approved for adults and children aged 12 and above with hematological malignancies, who will undergo umbilical cord blood transplantation after myeloablative preconditioning to shorten the time for neutrophil recovery and recovery.
Beremagene
geperpavec-svdt
(VYJUVEK)
2023/5/19 *		 Gene therapy VYJUVEK aims to address the genetic root of dystrophic epidermolysis bullosa (DEB) by providing functional copies of the human COL7A1 gene for wound healing, and to achieve sustained expression of the functional COL7 protein through re administration[3].This therapy has been approved for patients with nutritional dysplastic epidermolysis bullosa (DEB) at 6 months and above. It is the first replicable gene therapy in history and the first and only FDA approved drug for the treatment of DEB (implicit and explicit).
Delandistrogene
moxeparvovec-rokl
(ELEVIDYS)
2023/6/22 *		 Gene therapy This is a gene therapy that utilizes an adeno-associated virus (AAV) vector to deliver genes. These genes encode microvascular dystrophy proteins and target all muscles affected in the pathology of Duchenne muscular dystrophy (DMD)[4].This therapy is the first gene therapy approved in the United States for the treatment of DMD, suitable for outpatient children aged 4-5 who have DMD and have confirmed DMD gene mutations.
Donislecel
(LANTIDRA)
2023/6/28 *		 Cell therapy Type 1 diabetes (T1D) is an autoimmune disease. It's characterized by T cell-mediated damage to the pancreas' β cells, resulting in insulin deficiency and hyperglycemia. In patients with brittle T1D, exogenous insulin therapy may not suffice to maintain blood sugar levels[5]. Hence, endogenous insulin production via pancreatic islet cell therapy has drawn considerable research interest. Donislecel holds the distinction of being the first FDA-approved cell therapy drug for brittle T1D. This marks a significant milestone in the field of autoimmune diseases.
Valoctocogene
roxaparvovec-rvox
(ROCTAVIAN)
2023/6/30*		 Gene therapy A gene therapy product based on adeno-associated virus (AAV) 5, which is based on FVIII cDNA with B-domain deletion. This therapy is suitable for adult patients without a history of FVIII neutralizing antibodies and without detectable AAV5 carrier capsid antibodies[6].This therapy has been approved for the treatment of severe type A hemophilia, becoming the first gene therapy to treat severe type A hemophilia.
Onasemnogene
abeparvovec-xioi
(ZOLGENSMA)
2023/10/17		 Gene therapy This therapy is a gene treatment that uses an adenovirus vector. This therapy involves a gene treatment that utilizes an adenovirus vector. Its aim is to deliver functional copies of the human motor neuron survival (SMN) genes to patients' motor neuron cells, offering a potential treatment for spinal muscular atrophy (SMA)[7].This therapy has been approved for the treatment of pediatric patients under 2 years old who have SMA and have biallelic mutations in the SMN1 gene.
Exagamglogene
autotemcel
(Casgevy)
2023/12/8 *		 Cell therapy A cell therapy aimed at non-viral in vitro CRISPR/Cas9 gene editing.It reactivates fetal hemoglobin (HbF) in the red enhancer region of BCL11A in autologous CD34+ hematopoietic stem cells and progenitor cells (HSPCs)[8].This therapy has been approved for the treatment of sickle cell disease patients aged 12 years and above with recurrent vascular occlusion crisis. Casgevy is the first FDA approved therapy to utilize CRISPR/Cas9 (a genome editing technique).
Lovotibeglogene
autotemcel
(LYFGENIA)
2023/12/8 *		 Cell therapy After autologous transplantation of hematopoietic stem cells (HSCs) from patients, this therapy uses BB305 lentivirus vector（encoding the βA-T87Q-globin gene） for transduction, resulting in the production of anti sickle hemoglobin (HbAT87Q) in patients[9].This therapy has been approved for the treatment of sickle cell disease (SCD) in patients aged 12 years and above.
Brexucabtagene
autoleucel
(TECARTUS)
2023/12/11		 CAR-T Cell therapy The treatment options for mantle cell lymphoma (MCL) include traditional chemotherapy immunotherapy and targeted therapy for relapsed/refractory (R/R) diseases (such as BTK inhibitors). Brexucabtagene autoleucel is the first approved anti-CD19 CAR-T cell therapy for patients with R/R type MCL[10].This therapy has been approved for the treatment of adult patients with recurrent or refractory (r/r) B-cell precursor acute lymphoblastic leukemia (B-ALL).
Axicabtagene
ciloleucel
(YESCARTA)
2023/12/21		 CAR-T Cell therapy A first in class CAR-T cell therapy against CD19. This therapy is made from the patient's own peripheral blood mononuclear cells (PBMCs), during which T cells are modified and guided to recognize CD19 expressing cells[11].This therapy is approved for the treatment of adult patients with recurrent or refractory large B-cell lymphoma after two or more systemic treatments, including diffuse large B-cell lymphoma (DLBCL), primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and DLBCL caused by follicular lymphoma, unless otherwise specified.
* Representative First Approval

With the increasing attention to CGT therapy, in recent years, the proportion of CGT therapy in innovative drugs has been increasing. The successful approval of each CGT therapy carries the efforts of every researcher and the expectations of patients behind it. In the future, it is hoped that more and more CGT therapies will be approved to create higher clinical treatment levels and meet the needs of patients.

MCE provides GMP small molecules in the field of CGT, which can be used for stem cell reprogramming, induction of differentiation, proliferation and expansion, stemness maintenance, and transdifferentiation. Its functions include activating and alleviating CAR-T depletion, helping you develop CGT therapies.

Figure 1. mIHC technique to detect multispectral images

The GMP small molecule of MCE has the following characteristics:
（1）High purity, good stability between batches;
（2）Strong research and development capabilities, equipped with kilogram level production workshops;
（3）Multiple spot products with short lead times, reducing time costs;
（4）Provide additional services, materials for each stage of drug declaration, and on-site audit support;
（5）High consistency of sources, scale and standardization, reducing clinical late stage conversion costs.

Each batch of MCE's GMP small molecules undergoes strict quality control, and the system has passed the EU QP on-site audit. The release testing items formulated include but are not limited to purity, moisture, microbiology, internal toxicity, and residual solvents.

The partial GMP small molecule list is as follows:
Catalog_No & Name Description
HY-10182G
Laduviglusib		 GSK-3 inhibitor. It induces the transformation of fibroblasts into neurons; It induces fibroblasts and renal derived organs to transform into pluripotent stem cells; It induces the differentiation of pluripotent stem cells into myoblasts and pancreatic islet cells; It maintains the stemness and proliferation ability of pluripotent stem cells cultured in vitro.
HY-10583G
Y-27632 dihydrochloride		 ATP competitive ROCK inhibitors. It enhances the differentiation ability of human pluripotent stem cells into myogenic cells.
HY-15147G
XAV-939		 Tankyrase inhibitors. It induces human pluripotent stem cells to differentiate into functional neurons; It promotes the ability of pluripotent stem cells to differentiate into osteoblasts.
HY-10431G
SB-431542		 TGFR kinase inhibitor. It induces human pluripotent stem cells to differentiate into pancreatic islet cells and functional nerve cells.
HY-13012G
RepSox		 TGF-I receptor inhibitors. It induces the transformation of human adult cells into pluripotent stem cells; It induces the differentiation of human pluripotent stem cells into pancreatic islet cells and skeletal muscle cells.
HY-10475G
AM580		 RARα agonist. It induces the transformation of mouse embryonic fibroblasts into chemically induced pluripotent stem cells (CiPSCs) in phase I and II media; It induces the development of SH-SY5Y cells into neuron like cells; It induces the development of myeloid progenitor cells into neutrophils.
HY-10432G
A 83-01		 ALK 4/5/7 inhibitors. It induces human fibroblasts to differentiate into cardiomyocytes.
HY-12071G
LDN193189		 BMP type I receptor inhibitor. Inducing the differentiation of human pluripotent stem cells into pancreatic islet cells and hematopoietic progenitor cells.
HY-12318G
IBMX		 PDE inhibitors. Inducing neural like cells for neural differentiation of umbilical cord blood derived mesenchymal stem cells; Inducing neuronal differentiation in small cell lung cancer; Induce adipose tissue derived stem cells (ADSCs) to differentiate into neuronal like cells.
HY-13257G
Thiazovivin		 ROCK inhibitors. Promote the rapid and effective reprogramming of mouse tail tips and embryonic fibroblasts into iPSCs.
HY-15764G
A 419259		 Src, Lck, and Lyn inhibitors inhibit endogenous SFK (c-Src and Lck) activity, thereby inhibiting the differentiation of Src driven mES cells into primitive ectodermal like cells.

In addition, MCE also provides customized services for research grade and GMP grade small molecule products. Please feel free to contact us if you have any needs.