The FDA 2024 Drug Approval Landscape
The FDA’s Center for Drug Evaluation and Research (CDER) lists 50 new drug approvals listed for 2024. This number includes 50 novel molecular entities (NMEs), including both small molecule drugs and biologics. Some external sources have categorized these numbers slightly differently, reporting that there were 32 NCEs + 18 biologics, while others have stated that there were 34 NCEs + 16 biologics. The difference in opinion over whether certain pegylated peptides/oligonucleotides and protein derived drugs should be considered “chemical” entities vs. biologics is why some are splitting up the categories.
Of the newly approved therapies, small molecules continued their historical dominance of new drug approvals, accounting for 64% of all approvals (32 of 50 NMEs). Despite the recent surge in biologics approvals, small molecule drugs remain therapeutically relevant and synthetically accessible therapeutic modalities for modern medicinal chemists.
Key Trends of 2024 FDA NCEs
A deeper analysis reveals several noteworthy trends in the 2024 approval cohort:
Oncology Leadership
Cancer therapeutics continued to be the largest therapeutic category, with 14 (28%) of all NCEs approved in 2024 for oncology indications. This trend underscores the high level of unmet need in oncology drug discovery and the success of new drug entities to address this need. For instance, the highly selective RAF kinase inhibitor, Ojemda (tovorafenib), is approved for pediatric relapsed or refractory low-grade glioma. This targeted therapy represents an important advancement in pediatric oncology, where treatment options have historically been limited.
The August 2024 approval of Lazcluze for use in combination with amivantamab for first-line treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) with specific EGFR mutations demonstrates the continuing evolution of precision medicine in oncology. Similarly, Tecelra’s approval for unresectable or metastatic synovial sarcoma represents a novel immunotherapeutic approach for a challenging malignancy.
Rare Disease Emphasis
Therapies for orphan diseases represented 20% of approvals, highlighting the industry’s growing commitment to addressing specialized patient populations with limited treatment options. For example, Aqneursa (levacetylleucine) is approved for homozygous NPC disease and Miplyffa is approved for late infantile or juvenile NPC disease. These both target the same orphan indication for Niemann-Pick disease type C, a rare and currently fatal lysosomal storage disease. These approvals highlight the pharmaceutical industry’s growing capacity to develop targeted interventions for complex genetic disorders that were previously considered untreatable. Similarly, Duvyzat is a new PBA designed for Duchenne muscular dystrophy (DMD) approved in March 2024. DMD is a rare, X-linked disease which mostly affects young boys and for which there has been a paucity of treatment options.
Antibiotic Renewal
Three new antibiotics were approved in 2024, an impressive figure given that the previous year, 2023, only saw one new antibiotic approved. The antibiotics approved in 2024 are Exblifep (cefepime and enmetazobactam), Zevtera (ceftobiprole medocaril sodium), and Orlynvah (sulopenem etzadroxil and probenecid). All three represent new and exciting new additions to the antibiotic arsenal. Exblifep is a combination of the old cephalosporin antibiotic, cefepime, and a novel β-lactamase inhibitor enmetazobactam. Enmetazobactam is a new chemical entity approved as part of this combination drug to help protect cefepime against β-lactamase resistance mechanisms in Gram-negative bacteria. This combination approach exemplifies how medicinal chemistry can extend the utility of existing antibiotic classes through strategic partnership with new resistance-modifying agents.
Imaging & Contrast Agents:
Approvals such as flurpiridaz F-18 and iomeprol create demand for radiochemistry precursors, contrast excipients and stability/formulation services.
List of NCEs
The following table presents a detailed inventory of the NCEs approved by the FDA in 2024, highlighting their therapeutic applications and molecule type.
| Trade Name | Active Ingredient | Approval Date | Indication | Molecule Type |
| Alyftrek | vanzacaftor, tezacaftor, deutivacaftor | 12/20/2024 | Cystic fibrosis (combo) | Small molecule (combination) |
| Aqneursa | levacetylleucine | 9/24/2024 | Niemann-Pick disease type C (rare) | Small molecule |
| Attruby | acoramidis | 11/22/2024 | Transthyretin-mediated cardiomyopathy (ATTR-CM) | Small molecule |
| Cobenfy | xanomeline + trospium chloride | 9/26/2024 | Schizophrenia (combination muscarinic approach) | Small molecule (combination) |
| Crenessity | crinecerfont | 12/13/2024 | Classic congenital adrenal hyperplasia | Small molecule |
| Duvyzat | givinostat | 3/21/2024 | Indicated uses noted in FDA summary (oncology/rare) | Small molecule (HDAC inhibitor) |
| Ensacove | ensartinib | 12/18/2024 | Non-small cell lung cancer (NSCLC) | Small molecule (TKI) |
| Exblifep | cefepime + enmetazobactam | 2/22/2024 | Serious bacterial infections (antibiotic combo) | Antibiotic combination (beta-lactam + inhibitor) |
| Flyrcado | flurpiridaz F-18 | 9/27/2024 | Myocardial perfusion imaging (radiopharmaceutical) | Radiopharmaceutical (imaging agent) |
| Iomervu | iomeprol | 11/27/2024 | Radiographic contrast agent | Contrast agent |
| Iqirvo | elafibranor | 6/10/2024 | Primary biliary cholangitis (PBC) (in combination) | Small molecule (PPAR modulator) |
| Itovebi | inavolisib | 10/10/2024 | Advanced/metastatic breast cancer (PI3K inhibitor) | Small molecule (PI3K inhibitor) |
| Lazcluze | lazertinib | 8/19/2024 | NSCLC (EGFR mutant) | Small molecule (EGFR TKI) |
| Leqselvi | deuruxolitinib | 7/25/2024 | Severe alopecia areata (JAK inhibitor) | Small molecule (JAK inhibitor) |
| Livdelzi | seladelpar | 8/14/2024 | Primary biliary cholangitis (PBC) | Small molecule (PPAR agonist) |
| Lumisight | pegulicianine | 4/17/2024 | Optical imaging agent to detect cancerous tissue (pegylated peptide) | Pegylated peptide (imaging) |
| Miplyffa | arimoclomol | 9/20/2024 | Rare disease (chaperone-modulating therapy) | Small molecule |
| Ohtuvayre | ensifentrine | 6/26/2024 | Chronic obstructive pulmonary disease (COPD) (inhaled) | Small molecule (inhaled) |
| Ojemda | tovorafenib | 4/23/2024 | Pediatric low-grade glioma with RAF alteration | Small molecule (RAF inhibitor) |
| Orlynvah | sulopenem etzadroxil + probenecid | 10/25/2024 | Uncomplicated urinary tract infections (uUTI) (oral antibiotic combination) | Antibiotic combination (beta-lactam prodrug + enhancer) |
| Rapiblyk | landiolol hydrochloride | 11/22/2024 | Supraventricular tachycardia (IV beta-blocker) | Small molecule (beta blocker) |
| Revuforj | revumenib | 11/15/2024 | Relapsed/refractory acute leukemia with KMT2A translocation (menin inhibitor) | Small molecule (menin inhibitor) |
| Rezdiffra | resmetirom | 3/14/2024 | Non-cirrhotic MASH/NASH with liver fibrosis | Small molecule (thyroid receptor agonist) |
| Rytelo | imetelstat | 6/6/2024 | Myelodysplastic syndromes (MDS) (telomerase inhibitor; oligonucleotide class) | Oligonucleotide (TIDE class) |
| Sofdra | sofpironium bromide | 6/18/2024 | Primary axillary hyperhidrosis (topical gel) | Small molecule (topical) |
| Tryngolza | olezarsen | 12/19/2024 | Familial chylomicronemia syndrome (antisense oligonucleotide) | Antisense oligonucleotide |
| Tryvio | aprocitentan | 3/19/2024 | Resistant/difficult-to-control hypertension (add-on) | Small molecule (endothelin receptor antagonist) |
| Vafseo | vadadustat | 3/27/2024 | Anemia due to chronic kidney disease (CKD) | Small molecule (HIF stabilizer) |
| Voranigo | vorasidenib | 8/6/2024 | Grade 2 astrocytoma/oligodendroglioma with IDH1/2 mutation | Small molecule (IDH inhibitor) |
| Voydeya | danicopan | 3/29/2024 | Extravascular hemolysis in paroxysmal nocturnal hemoglobinuria (PNH) | Small molecule (complement inhibitor) |
| Xolremdi | mavorixafor | 4/26/2024 | WHIM syndrome (rare immunodeficiency) | Small molecule (CXCR4 antagonist) |
| Yorvipath | palopegteriparatide | 8/9/2024 | Hypoparathyroidism (pegylated PTH analogue) | Pegylated peptide (TIDE) |
| Zelsuvmi | berdazimer | 1/5/2024 | Molluscum contagiosum (topical gel) | Small molecule (topical) |
| Zevtera | ceftobiprole medocaril sodium | 4/3/2024 | Antibacterial (bloodstream infections, skin, pneumonia) – cephalosporin | Antibiotic (cephalosporin) |
Selected NCEs of Interest from 2024
Alfa Chemistry, as a premier API supplier, is ready to facilitate this innovation surge by providing pharma quality intermediates and APIs for these newly approved entities.
Resmetirom
- Indication: Noncirrhotic nonalcoholic steatohepatitis (NASH) with fibrosis.
- MOA: Selective thyroid hormone receptor-β (THR-β) agonist.
- Why it matters for APIs: This first-in-class metabolic agent highlights the potential and market demand for selective THR-β modulators. Alfa Chemistry can provide custom synthesis for resmetirom analogues and intermediates, which can be leveraged for both generic or next-generation development.
Danicopan (Voydeya)
- Indication: Paroxysmal nocturnal hemoglobinuria (PNH), as add-on therapy to ravulizumab or eculizumab.
- MOA: Complement factor D inhibitor.
- API Implication: Complement pathway inhibitors are a growing class in immunology and hematology. Alfa Chemistry can accelerate supply of danicopan API (or intermediates) to support commercial scale or future pipeline combinations.
Inavolisib (Itovebi)
- Indication: Advanced HR-positive, HER2-negative, PIK3CA-mutated breast cancer (in combination with palbociclib + fulvestrant).
- MOA: PI3K-α (p110α) inhibitor.
- Regulatory Highlights: Received Priority Review and Breakthrough Therapy Designation.
- Why It’s Strategically Important: PI3K inhibitors continue to be refined for safer and more selective profiles. We offer inavolisib or its analogues/intermediates to support your high-value opportunity in oncology-focused API markets.
