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Release date:2025/1/7 1:14:59

In 2024, the U.S. Food and Drug Administration's (FDA) Center for Drug Evaluation and Research (CDER) approved 50 new molecular entities (NMEs), consisting of 32 new chemical entities (NCEs) and 18 biologic entities (NBEs) (see Figure 1). Of the NCEs, small molecules dominated, making up approximately 91% (31 drugs), with the remaining drugs comprising nucleic acids and peptides. For the NBEs, 81% were antibody-based, including 10 monoclonal antibodies and 3 bispecific antibodies, while the rest included fusion proteins and botulinum toxin type A.

CDER-approvals-by-modality

Figure 1. CDER approvals by modality [1]

FDA Approved First-in-class Therapies in 2024

Overall, the number of new drugs approved by CDER in 2024 is similar to that of 2023, with the number of "first-in-class" (FIC) drugs remaining roughly the same. In 2023, CDER approved 20 FIC drugs, accounting for 36% of all new drug approvals. In 2024, 22 of the approved new drugs were FIC drugs, making up 44% of the total. Among these FIC drugs, small molecules represented nearly half (10 drugs), while antibody-based drugs formed another major group of innovative treatments (5 monoclonal antibodies and 3 bispecific antibodies).

Drug (brand name) Sponsor Properties Indication
Resmetirom (Rezdiffra) Madrigal Thyroid hormone receptor-β agonist Noncirrhotic NASH with liver scarring
Danicopan (Voydeya) AstraZeneca/Alexion Complement factor D inhibitor Extravascular haemolysis with paroxysmal nocturnal haemoglobinuria
Elafibranor (Iqirvo) Ipsen PPAR agonist PBC
Vorasidenib (Voranigo) Servier IDH1 and IDH2 inhibitor Astrocytoma or oligodendroglioma
Seladelpar (Livdelzi) Gilead PPARδ agonist PBC
Arimoclomol (Miplyffa) Zevra Denmark MOA unknown Niemann-Pick disease type C
Revumenib (Revuforj) Syndax Menin inhibitor Acute leukaemia
Crinecerfont (Crenessity) Neurocrine CRF type 1 receptor antagonist Classic congenital adrenal hyperplasia

Table 1. FDA Approved First-in-class small molecule drugs in 2024

Rezdiffra (Resmetirom)

On March 14, 2024, Madrigal Pharmaceuticals announced the approval of Rezdiffra™, a new, first in class oral therapy for nonalcoholic steatohepatitis (NASH). Rezdiffra is a thyroid hormone receptor beta (THR-β) agonist that works directly in the liver. 

Rezdiffra

Figure 2. Mechanism of Rezdiffra

THR-β, primarily found in the liver, plays a key role in regulating lipid metabolism by lowering LDL-C, triglycerides, and atherogenic lipoproteins; it also contributes to improved liver function by enhancing fatty acid breakdown and mitochondrial activity, ultimately reducing liver fat accumulation and mitigating lipotoxicity.

Vorasidenib (Voranigo)

The U.S. FDA has approved Vorasidenib (Voranigo; Servier Pharmaceuticals LLC) for adult and pediatric patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation, following surgery including biopsy, sub-total resection, or gross total resection. Voranigo is a first-in-class IDH1/2 dual inhibitor. This approval marks the first systemic therapy approved by the FDA for the treatment of these patients.

FDA Approved Nucleic Acids and Peptides in 2024

As the development of peptide and nucleic acid-based drugs advances, these therapies now account for about 10% of all new FDA-approved drugs in recent years. In 2024, the FDA approved several nucleic acid-based treatments, including Rytelo (the first-ever oligonucleotide telomerase inhibitor), Yorvipath (the long-acting parathyroid hormone prodrug), and Tryngolza (a new treatment for familial chylomicronemia syndrome).

Drug (brand name) Sponsor Properties Indication
Pegulicianine (Lumisight) Lumicell Optical imaging agent Imaging agent for cancerous tissue
Imetelstat (Rytelo) Geron Telomerase inhibitor, oligonucleotide Myelodysplastic syndromes
Olezarsen (Tryngolza) Olezarsen APOC-III-directed ASO Familial chylomicronemia syndrome

Table 2. FDA Approved Nucleic Acids and Peptides in 2024

Rytelo (imetelstat) works by targeting and binding to the RNA template of telomerase, inhibiting its activity. It was approved in June for the treatment of adult patients with myelodysplastic syndromes (MDS).

Peptide-based drugs face a significant challenge in that peptides are easily degraded in the body, making it difficult to maintain their efficacy. However, recent advances in peptide modification have led to longer-lasting drugs, offering patients more convenient treatments. This year, the FDA approved Yorvipath, a long-acting parathyroid hormone prodrug. By attaching the hormone to a polyethylene glycol (PEG) carrier, it controls the release of the hormone in the body. Yorvipath was approved in August as the first treatment for adult hypoparathyroidism.

FDA Blockbuster Drug Approvals

Of the new drug approvals that the FDA handed out in 2024, two of the most important came in the third quarter and both were for neurological conditions. In July, the FDA approved Eli Lilly's Alzheimer's treatment, Kisunla. Then, in September, it approved Bristol Myers Squibb's schizophrenia drug, Cobenfy.

Both drugs address conditions where patients have long been waiting for new treatment options. Millions suffer from these disorders, but only a small percentage are currently treated.

Kisunla is especially exciting because it's the first anti-amyloid drug that allows treatment to stop once amyloid plaques are cleared, reducing the number of infusions and lowering costs.

Cobenfy is generating buzz as the first schizophrenia drug in 70 years with a new mechanism of action. Since the approval of chlorpromazine in 1954, all other schizophrenia treatments have been based on the dopamine receptor class.

FDA Approved Cellular & Gene Therapies in 2024

In 2024, the FDA approved 8 cell and gene therapies, setting a new record. Over the past three years, a total of 20 cell and gene therapies have been approved, highlighting the rapid growth of this treatment approach.

Biologic name (brand name) Sponsor Properties Indication
Lifileucel (Amtagvi) Iovance Tumour-infiltrating lymphocyte therapy Melanoma
Atidarsagene autotemcel (Lenmeldy) Orchard Ex vivo gene therapy, lentiviral vector with an ARSA transgene Metachromatic leukodystrophy
Fidanacogene elaparvovec (Beqvez) Pfizer In vivo gene therapy, AAV vector with a FIX transgene Haemophilia B
Afamitresgene autoleucel (Tecelra) Adaptimmune MAGE-A4-directed T cell therapy, using a TCR Synovial sarcoma
Obecabtagene autoleucel (Aucatzyl) Autolus CD19-targeted CAR T cells ALL
Eladocagene exuparvovec (Kebilidi) PTC Therapeutics In vivo gene therapy, AAV vector with a DDC transgene AADC deficiency
Remestemcel (Ryoncil) Mesoblast Bone marrow-derived mesenchymal stromal cell Steroid-refractory acute GvHD
Acellular tissue engineered vessel (Symvess) Humacyte Global Vascular conduit, containing human ECM proteins Extremity arterial injury when urgent revascularization is needed

Table 3. FDA Approved Cellular & Gene Therapies in 2024

These therapies not only represent technological innovation but also bring the first approved treatments for patients with various types of diseases.

References:
[1] 2024 FDA approvals https://www.nature.com/articles/d41573-025-00001-5

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