All Specialty, All Impact—Are You Ready to Manage Spring 2025’s FDA Approvals?
In Q2 2025, the FDA approved nine new molecular entities—all (100%) qualifying as specialty therapies. These are not your typical low-cost maintenance medications; they represent a transformative shift in the landscape of pharmaceutical costs and treatment options. This is a wake-up call for self-funded employers, as these therapies bring significant challenges and risks.
Every drug approved this quarter has inherent complexities: rare-disease treatments, precision-targeted genetic therapies, bundled regimens, and a price range of $140,000 to $800,000+ annually. This isn’t speculation; these therapies could hit your plan’s claims starting tomorrow.
At Axum Rx, we’ve closely examined each Q2 approval. Three standout therapies exemplify this shift—and call for a comprehensive strategy from employers.
Three Q2 Therapies Redefining Employer Strategy
Tryptyr (acoltremon)
This daily eye-drop for chronic dry-eye disease costs $12,000 per year. It’s indicated when standard lubricants fail, offering patients relief from persistent irritation and vision disturbance. For plans, it signals the rising cost of treatment for common conditions—where specialty pricing is becoming the norm.
Avmapki + Fakzynja
This co-packaged combination targets KRAS G12C-mutated ovarian cancer at an eye-popping $700,000 annually per patient. For those facing this devastating diagnosis, it offers a targeted therapy option. For plans, it highlights the risks of bundled therapies that lock in high costs, with little flexibility for substitutions or step-therapy adjustments.
Andembry (garadacimab-gxii)
A monthly injection that prevents hereditary angioedema attacks—potentially life-threatening swelling episodes—costs $822,000 annually. While it offers patients life-changing freedom from constant anxiety over airway closures, for plans, it demonstrates how rare-disease treatments can exceed entire stop-loss thresholds.
Across these approvals, three themes demand employer attention:
Specialty Pricing in Common Conditions highlights that everyday therapies—from chronic dry-eye drops to dermatology treatments and preventive antibodies—can carry six-figure price tags, meaning governance must extend beyond oncology and ultra-rare diseases.
Fixed-Format Bundles underscore how co-packaged regimens lock in high costs with no room for step-therapy edits or ingredient substitutions, so benefit policies must be retooled to anticipate and manage bundled therapies.
Prophylactic Treatments as Financial Outliers reveal that preventive therapies for rare diseases can generate single-claim liabilities dwarfing traditional stop-loss models, making it critical for employers to assess the financial impact of prophylaxis alongside acute treatment.
Specialty Takeover: Q2 Edition
In Q2 2025, 100% of FDA approvals were specialty therapies—44% oncology, 22% rare-disease, and 11% each in nephrology, ophthalmology, and pediatric prophylaxis. Here’s the breakdown by clinical area and indication:
Four oncology agents, targeting:
• A rare form of lung cancer driven by ROS1 gene alterations (Ibtrozi)
• Ovarian cancer with KRAS G12C mutation (Avmapki + Fakzynja)
• Recurrent or metastatic throat cancer (penpulimab-kcqx)
• Lung cancer that overproduces the MET protein (Emrelis)
Two rare-disease treatments:
• Monthly prophylaxis for hereditary angioedema (Andembry)
• An antibody therapy for generalized myasthenia gravis (Nipocalimab)
One nephrology therapy: Vanrafia (atrasentan) for proteinuria reduction in IgA nephropathy
A dry-eye specialty drop: Tryptyr (acoltremon)
A pediatric RSV prophylactic antibody: Enflonsia (clesrovimab-cfor)
From Containing Costs to Governing Complexity
Each therapy spans different channels and demands—some draw on the medical benefit (clinic infusions), others on pharmacy budgets (self-administered injectables or eye drops); adherence support ranges from simple reminders to full nursing coordination; prior-authorization can be a single checklist or a layered genetic/biomarker review; patient pools swing from ultra-rare cancer subtypes to broad infant prophylaxis cohorts. Yet most vendors still lean on one-size-fits-all PA templates and static stop-loss designs that ignore these critical distinctions.
That misalignment isn’t just operational—it’s a strategic vulnerability. Rigid protocols stall legitimate approvals and inflate appeal backlogs, frustrate members into abandoning therapy, and allow six-figure claims to sail past outdated risk models. This isn’t a pharmacy story. It’s a leadership one. Employers must demand proactive governance—tailored coverage rules, bespoke support programs, and dynamic actuarial frameworks that reflect each therapy’s unique clinical and financial footprint—before unchecked spend erodes plan stability.
Specialty Savvy: Your Proactive Precision Action Blueprint
This moment isn’t just about containing costs; it’s about managing complexity. Here’s what you need to do now:
Require Genetic and Biomarker Testing
Ensure precision diagnostics are a prerequisite for high-cost therapies. Embed flexible, clinically reviewed exception processes to maintain patient trust and cost control.
Tie Reimbursement to Real-World Outcomes
For therapies exceeding a threshold annually, release reimbursement based on documented effectiveness, not just claim submissions. Align payment with measurable, real-world outcomes, rather than blind reimbursement for utilization.
Integrate Medical and Pharmacy Data
Data silos lead to duplicate billing, missed opportunities for monitoring, and uncontrolled spending. Employers must ensure near-real-time integration of medical and pharmacy data.
Recalibrate Stop-Loss Models
Today’s six-figure claims don’t fit into traditional stop-loss paradigms. Update your layers, triggers, and exclusions to reflect emerging risks from rare-disease, ophthalmology, immunology, and genetic therapies.
Prioritize Human-Centered Onboarding
The first fill is a clinical event, not just a transaction. Provide members with clear education, transparent appeals processes, and dedicated case management to improve adherence and health outcomes.
Partner with an Independent Specialty Drug Advisor
PBMs and health plans profit when spend increases. Employers need a partner who is solely focused on protecting the plan and empowering patients. That’s where Axum Rx can help.
Final Employer Takeaway:
This is no longer about containing cost—it’s about governing complexity. If your plan still assumes specialty equals oncology or rare disease, it’s already exposed.
If it relies on PBM templated protocols, it’s already vulnerable.
And if it waits until claims hit to respond, it’s already too late.
It's time for employers to shift from reactive cost containment to proactive complexity governance. This means embedding precision diagnostics, outcome-linked payment structures, integrated data oversight, and recalibrated risk models before six-figure claims overwhelm your plan. Don’t wait for the storm to hit—take action now.
Strategic Consulting by AxumRx.com
Axum Rx empowers employers to harness the power of Spring 2025’s specialty drug surge—turning complex FDA approvals into clear, actionable strategies. From precision diagnostics and outcome-driven contracts to seamless medical-pharmacy data flow and patient-centered support, we help you govern complexity, control costs, and protect your plan’s future.
Contact us today to learn how we can help: Learn more.
References
U.S. Food & Drug Administration. Spring 2025 New Molecular Entity Approval Summaries.
IQVIA Institute. Medicine Use and Spending in the U.S. 2024. www.iqvia.com
U.S. Food & Drug Administration. Tryptyr (acoltremon) Label. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/217370s000lbl.pdf
U.S. Food & Drug Administration. Avmapki + Fakzynja Co-Pack Label. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/219616s000lbl.pdf
CSL Behring. Andembry (garadacimab-gxii) Prescribing Information. https://labeling.cslbehring.com/PI/US/Andembry/EN/Andembry-Prescribing-Information.pdf
The American Journal of Managed Care. "FDA Approves Garadacimab in Hereditary Angioedema."
Pharmaceutical Executive. "FDA Grants Accelerated Approval to Avmapki + Fakzynja."
Axum Rx. About Us. www.axumrx.com
U.S. Food & Drug Administration. Ibtrozi (taletrectinib) Label.
U.S. Food & Drug Administration. Penpulimab-kcqx Approval.
U.S. Food & Drug Administration. Emrelis (telisotuzumab vedotin-tllv) Label.
CheckRare. Nipocalimab Approval for Myasthenia Gravis.
Novartis. Vanrafia (atrasentan) Prescribing Information.