Oncology Drug Development Pipeline Remains Robust, Driven by Novel Therapies and Cell Therapies

The oncology drug development pipeline continues to be a major area of pharmaceutical innovation, with a strong emphasis on novel therapies and cell therapies. According to a presentation at the 2024 Academy of Managed Care Pharmacy (AMCP) Nexus meeting, cancer drug development remains a dominant force in pharmaceutical innovation, clinical trials, and drug expenditures.

Robust Oncology Pipeline

Approximately 1,600 medicines and vaccines are currently in the oncology pipeline, targeting a wide range of cancers. While a significant percentage of these agents are focused on rare cancers with smaller patient populations, there is also a substantial number of agents in development for common solid tumors. Sita Bhatt, Pharm.D., a clinical pharmacy specialist at Boston Medical Center, noted a growing trend toward cell therapies for solid tumors, with 44% of all trials initiated in 2023 for solid tumor indications.

Rising Costs and Financial Impact

Global spending on cancer drugs is projected to increase to $409 billion by 2028. The high cost of novel agents, averaging over $200,000 per year, is a significant factor. While biosimilars and generic medications may offer some savings, they are unlikely to offset the overall trajectory of increasing expenditures.

Recent FDA Approvals

Several cancer drugs have recently received FDA approval, including:

• Itovebi (inavolisib): A PI3K inhibitor approved for HER2-negative breast cancer with a PIK3CA mutation. It is used in combination with fulvestrant and palbociclib.
• Tecentriq Hybreza (atezolizumab and hyaluronidase-tqjs): A PDL-1 inhibitor approved for multiple indications, including non-small cell lung cancer (NSCLC). This formulation allows for subcutaneous administration.
• Lazcluze (lazertinib) and Rybrevant (amivantamab): A combination approved as a first-line treatment for non-small cell lung cancer with epidermal growth factor receptor (EGFR) mutations.
• Niktimvo (axatilimab): An anti-CSF-1R antibody approved for chronic graft versus host disease (GVHD).
• Lymphir (denileukin diftitox-cxdl): An IL2-receptor-directed cytotoxin approved for relapsed or refractory cutaneous T-cell lymphoma.
• Voranigo (vorasidenib): An IDH1 and IDH2 inhibitor approved for treatment of grade 2 IDH-mutant glioma. The cost per 40 mg tablet is $1,329, and $29,881 per 30 count bottle.
• Tecelra (afamitresgene autoleucel): A MAGE-A4-directed genetically modified autologous T-cell immunotherapy that received accelerated approval as a treatment for unresectable or metastatic synovial sarcoma. The drug was launched with a list price of $727,000.
• Augtyro (repotrectinib): A tyrosine kinase inhibitor granted accelerated approval as a treatment for solid tumors with a neurotrophic tyrosine receptor kinase gene fusion. The estimated cost per 120 count of the drug is $14,500.
• Rytelo (imetelstat): An oligonucleotide telomerase inhibitor approved as a treatment for low- to intermediate-1 risk myelodysplastic syndrome (MDS) with transfusion-dependent anemia. Rytelo is priced to cost approximately $385,000 a year.
• Imdelltra (tarlatamab-dlle): A bispecific T-cell engager that received accelerated approval for extensive-stage small cell lung cancer. The estimated cost of the drug is $15,000 per 10 mg, $31,500 for cycle one and step-up dosing, and $30,000 for cycle 2 and beyond.
• Ojemda (tovorafenib): Accelerated approval as a Pan-RAF kinase inhibitor for relapsed or refractory pediatric low-grade glioma with BRAF fusion or rearrangement, or BRAF V600 mutation. The estimated cost per 16, 20 or 24 count bottle is $33,916 and $8,479 per 12 mL bottle.
• Anktiva (nogapendekin alfa inbakicept-pmln): Received approval as an IL-15 receptor agonist for Bacillus Calmette-Guérin-unresponsive, nonmuscle-invasive bladder cancer. The cost of induction is estimated at $214,800 and patients can receive up to two courses.
• Tevimbra (tislelizumab-jsgr): A PD-1 inhibitor approved for unresectable or metastatic esophageal squamous cell carcinoma. The cost per 10 mL (100 mg) and per dose (200 mg) is approximately $5,204 an $10,408, respectively.
• Amtagvi (lifileucel): Received accelerated approval for unresectable or metastatic melanoma as a tumor-derived autologous T cell immunotherapy.

Upcoming FDA Decisions

Several cancer drugs are awaiting FDA approval decisions, including:

• Zenocutuzumab (NRG1-positive NSCLC and pancreatic cancer)
• Zolbetuximab (CLDN18.2-positive gastric or gastroesophageal junction adenocarcinoma)
• Obecabtegene autoleucel (relapsed or refractory adult B-cell acute lymphoblastic leukemia)
• Zanidatamab (HER2-amplified biliary tract cancer)
• Datopotamab deruxtecan (advanced nonsquamous NSCLC and metastatic HR-positive, HER2-negative breast cancer)
• Revumenib (relapsed or refractory KMT2Ar acute leukemia)
• Cosibelimab (metastatic or locally advanced cutaneous squamous cell carcinoma)
• Ensartinib (ALK-positive NSCLC)
• Subcutaneous formulation of Opdivo (nivolumab)
• Mirdametinib (neurofibromatosis type 1-associated plexiform neurofibromas)
• Remestemcel-L (acute GVHD)
• Tabelecleucel (Epstein-Barr virus-positive post-transplant lymphoproliferative disease)
• Subcutaneous formulation of Rybrevant (amivantamab)