January 2026 FDA Decisions: Five Critical Approvals That Could Transform Patient Care

As the pharmaceutical industry enters 2026, January promises to be a pivotal month for drug approvals, with the FDA set to make decisions on five potentially transformative therapies. These Prescription Drug User Fee Act (PDUFA) dates represent critical milestones for patients facing conditions ranging from rare cancers to common age-related vision problems.

Atara's Tabelecleucel: Breaking New Ground in Cell Therapy

The month's most significant decision arrives January 10, when the FDA will rule on Atara Biotherapeutics' tabelecleucel for EBV-positive post-transplant lymphoproliferative disease (EBV+ PTLD). This allogeneic T-cell immunotherapy represents a potential breakthrough for transplant recipients facing this rare but deadly complication.

EBV+ PTLD affects patients who have undergone solid organ or hematopoietic cell transplantation, with limited treatment options currently available. Tabelecleucel's pivotal ALLELE study demonstrated a remarkable 48.8% objective response rate, offering hope for patients who have exhausted conventional therapies including rituximab and chemotherapy.

If approved, tabelecleucel would become the first allogeneic T-cell therapy available in the United States for this indication, potentially establishing a new treatment paradigm for post-transplant complications. The therapy's "off-the-shelf" nature could provide rapid access for patients facing aggressive disease progression.

Addressing Unmet Needs Across Multiple Therapeutic Areas

January 23 brings two significant decisions that could reshape treatment landscapes in nephrology and oncology. Travere Therapeutics awaits FDA approval for sparsentan in focal segmental glomerulosclerosis (FSGS), a rare kidney disease that represents a leading cause of end-stage renal disease.

Sparsentan, an oral endothelin and angiotensin II receptor antagonist, demonstrated significant proteinuria reduction compared to standard care in the phase 3 DUPLEX study. Approval would mark the first targeted treatment for FSGS, offering hope to patients who currently face limited therapeutic options beyond supportive care.

The same date marks another potential milestone in breast cancer treatment, as the FDA reviews the combination of trastuzumab deruxtecan (Enhertu) and pertuzumab for first-line HER2-positive metastatic breast cancer. The DESTINY-Breast09 trial showed impressive results, with median progression-free survival reaching 40.7 months compared to 26.9 months for standard care.

This combination could establish a new standard of care for newly diagnosed HER2-positive metastatic breast cancer, potentially extending survival outcomes significantly beyond current expectations. The 85.1% objective response rate demonstrated in clinical trials suggests substantial clinical benefit for this patient population.

Innovation in Ophthalmology and Emergency Medicine

January 28 features Tenpoint Therapeutics' Brimochol PF, a preservative-free combination of carbachol and brimonidine tartrate for presbyopia treatment. This age-related vision condition affects virtually everyone over 45, yet treatment options remain limited.

The phase 3 BRIO studies demonstrated significant improvements in near and distance visual acuity, with effects lasting up to 10 hours. Approval would provide the first pharmacological approach to presbyopia management, potentially transforming how clinicians address this common condition affecting millions of aging patients.

The month concludes with Aquestive Therapeutics' Anaphylm decision on January 31. This sublingual epinephrine film represents a paradigm shift in anaphylaxis treatment, offering the first needle-free alternative to traditional auto-injectors.

Anaphylm's rapid dissolution and systemic delivery could address significant barriers to epinephrine administration, particularly in pediatric populations and patients with needle phobia. Clinical studies demonstrated symptom resolution beginning just two minutes after administration, potentially improving emergency response outcomes.

Broader Implications for Healthcare Innovation

These January decisions reflect broader trends in pharmaceutical innovation, including the advancement of cell therapies, precision medicine approaches, and patient-centric drug delivery systems. The diversity of therapeutic areas represented underscores the industry's expanding ability to address previously intractable medical conditions.

For investors and industry observers, these approvals could validate emerging therapeutic modalities and influence future development strategies. Success in rare diseases like EBV+ PTLD and FSGS demonstrates the viability of orphan drug development, while innovations in presbyopia and anaphylaxis treatment highlight opportunities in large, underserved markets.

The outcomes of these January decisions will likely influence treatment guidelines, healthcare economics, and patient outcomes across multiple therapeutic areas. As the pharmaceutical industry continues evolving toward more targeted and personalized therapies, these approvals represent critical steps in expanding the therapeutic arsenal available to clinicians and patients.

With each decision carrying the potential to transform patient care in its respective therapeutic area, January 2026 may well be remembered as a watershed month in pharmaceutical innovation and regulatory approval.