Dr. RAS Naidu, PhDIntroduction:
Healthcare is rapidly shifting from traditional, generalized treatments to personalized medicine, where each patient’s unique molecular and clinical profile guides therapy decisions. This transformation is driven by advances in diagnostics and targeted therapies, with the FDA approving a growing number of personalized medicines—especially for rare genetic disorders, cancers, and select common diseases.
Personalized Medicine: A New Paradigm
Personalized medicine, also called precision or individualized medicine, uses diagnostic testing and patient-specific data to select the most effective treatments. This approach replaces the historical trial-and-error model, integrating molecular diagnostics with a patient’s medical history to develop tailored care plans. In 2024, the FDA approved 18 new personalized medicines and six gene/cell therapies, continuing a trend where such therapies account for at least 25% of new drug approvals annually—a significant rise from less than 10% a decade ago. In 2024, 38% of new molecular entities approved by the FDA were personalized medicines, underscoring their growing clinical impact.
2024 FDA-Approved Personalized Medicines and Gene/Cell Therapies:
Table 1: 2024 FDA-Approved Personalized Medicines
| Product Name (Generic) | Formulation Design Strategy | Diagnostic Data Guiding Use | Treatment / Indication / Disease | Molecule Type |
| Tevimbra (tislelizumab-jsgr) | IV monoclonal antibody; stabilized | PD-L1 expression (IHC assay) | Unresectable or metastatic esophageal squamous cell carcinoma | Large |
| Ojemda (tovorafenib) | IR oral tablet for suspension | BRAF fusion/V600 mutation (molecular testing) | Pediatric low-grade glioma with BRAF alteration | Small |
| Kisunla (donanemab-azbt) | IV monoclonal antibody; lyophilized or liquid | ApoE ε4 genotype, amyloid PET/CSF | Early symptomatic Alzheimer’s disease | Large |
| Leqselvi (deuruxolitinib) | IR oral tablet | CYP2C9 genotype (pharmacogenomics) | Moderate to severe alopecia areata | Small |
| Voranigo (vorasidenib) | IR oral tablet | IDH1/ID2 mutation (molecular testing) | IDH-mutant diffuse glioma | Small |
| Livdelzi (seladelpar) | Capsule | CYP2C9 genotype (pharmacogenomics) | Primary biliary cholangitis | Small |
| Lazcluze (lazertinib) | Film-coated oral tablet | EGFR exon 19/21 mutation (molecular testing) | EGFR-mutated non-small cell lung cancer | Small |
| Miplyffa (arimocomol) | Oral capsule | NPC1/NPC2 gene mutation (genetic testing) | Niemann-Pick disease type C | Small |
| Aqneursa (levacetylleucine) | Granules for oral suspension | NPC1/NPC2 gene mutation (genetic testing) | Niemann-Pick disease type C | Small |
| Itovebi (inavolisib) | IR oral tablet | PIK3CA mutation, HR, HER2 status | HR-positive, HER2-negative, PIK3CA-mutated breast cancer | Small |
| Vyloy (zolbetuximab-clzb) | IV monoclonal antibody; stabilized | HER2, CLDN18.2 expression (IHC/FISH) | CLDN18.2-positive, HER2-negative gastric or GEJ cancer | Large |
| Revufori (revumenib) | IR oral tablet | KMT2A (MLL) rearrangement | KMT2A-rearranged acute leukemia | Small |
| Zilhera (zanidatamab-hrii) | High-concentration IV bispecific antibody | HER2 overexpression (IHC/FISH) | HER2-positive biliary tract cancer | Large |
| Bizengri (zenocutuzumab-zbco) | IV bispecific antibody; stabilized | NRG1 gene fusion | NRG1 fusion-positive solid tumors | Large |
| Unloxcyt (cosibelimab-ipdl) | IV monoclonal antibody; stabilized | PD-L1 expression (IHC assay) | Cutaneous squamous cell carcinoma | Large |
| Ensacove (ensartinib) | Capsule | ALK rearrangement | ALK-positive non-small cell lung cancer | Small |
| Tryngolza (olezarsen) | SC antisense oligonucleotide; lipid-stabilized | LPL gene mutation | Familial chylomicronemia syndrome | Small (oligonucleotide) |
| Alyftrek (vanzacaftor, tezacaftor, deutivacaftor) | Fixed-dose combo oral tablet; layered design | CFTR F508del/other responsive mutations | Cystic fibrosis with responsive CFTR mutations | Small |
Table 2: 2024 FDA-Approved Gene/Cell Therapies
| Product Name (Generic) | Formulation Design Strategy | Diagnostic Data Guiding Use | Treatment / Indication / Disease | Molecule Type |
| Amtagvi (lifileucel) | Autologous TILs, cryopreserved, IV infusion | Tumor biopsy for TIL expansion; prior therapy status | Advanced melanoma (after anti-PD-1 and targeted therapy) | Large (cell therapy) |
| Lenmeldy (atidarsagene autotemcel) | Autologous CD34+ HSCs, lentiviral transduction, cryopreserved, IV infusion | ARSA gene mutation, MLD diagnosis | Metachromatic leukodystrophy (MLD) | Large (cell/gene therapy) |
| Beqvez (fidanacogene elaparvovec-dzkt) | AAV vector gene therapy, single IV infusion | Factor IX deficiency, AAV antibody status | Hemophilia B (congenital factor IX deficiency) | Large (gene therapy) |
| Tecelra (afamitresgene autoleucel) | Autologous T cells, lentiviral transduction, cryopreserved, IV infusion | HLA typing, MAGE-A4 expression | Synovial sarcoma, myxoid/round cell liposarcoma | Large (cell/gene therapy) |
| Aucatzyl (obecabtagene autoleucel) | Autologous anti-CD19 CAR-T cells, cryopreserved, IV infusion | CD19 expression in B-ALL | B-cell acute lymphoblastic leukemia (B-ALL) | Large (cell/gene therapy) |
| Kebilidi (eladocagene exuparvovec-tneq) | rAAV2 vector gene therapy, single IV infusion | AADC deficiency (genetic testing) | Aromatic L-amino acid decarboxylase (AADC) deficiency | Large (gene therapy) |
Implications:
The diversity of formulation strategies in 2024’s personalized medicines highlights the need for close collaboration among formulation scientists, clinicians, and regulatory experts. Understanding the delivery system is crucial for healthcare professionals making treatment choices and for scientists developing innovative, patient-friendly therapies. As personalized medicine evolves, formulation technologies will remain central to translating scientific advances into real-world patient benefits. This collaborative and multidisciplinary approach is essential to overcome implementation barriers, optimize patient outcomes, and ensure that advances in diagnostics and therapeutics are effectively integrated into clinical practice.
Conclusion:
The 2024 data highlight a dynamic and complementary relationship between small molecules and biologics in personalized medicine. Both classes are essential: small molecules for their accessibility and versatility, and biologics for their specificity and transformative potential in complex and rare diseases. The future will see continued innovation in both areas, with small molecules remaining highly relevant alongside the expanding role of biologics. Personalized medicine is poised to further enhance patient care by moving beyond the “one-size-fits-all” approach, offering more precise, effective, and patient-centered therapies.
The 2024 approvals show that personalized medicine is most heavily applied in oncology and rare genetic diseases, with a growing but still smaller presence in severe autoimmune and metabolic disorders. This trend is driven by the availability of precise molecular diagnostics, the urgent need for effective therapies in these high-burden areas, and the ability of personalized approaches to deliver significant clinical benefit where traditional “one-size-fits-all” treatments fall short.
Disclaimer:
This blog is for informational purposes only and is not a substitute for professional medical or regulatory advice. For detailed product information, consult official FDA resources and product labels.
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