FAQ: Milestones You May Have Missed in the 2025 Biopharma Pipeline

1. How are FDA and European Commission (EC) accelerated pathways prioritizing cutting-edge chimeric antigen receptor T cell (CAR-T) therapies?

Regulators are accelerating the timeline for next-generation treatments, exemplified by FDA granting priority review and accepting the supplemental biologics license application for lisocabtagene maraleucel (brand name Breyanzi) in relapsed or refractory marginal zone lymphoma (MZL), paving the way for its fifth cancer approval. This strategic priority review status highlights the agency's push for new cancer indications.

2. What major status designations are fast-tracking complex gene therapy development in 2025?

Complex dual-vector gene therapy development, such as AAVantgarde’s AAVB-039, is gaining traction through significant regulatory milestones, including orphan drug designation from FDA and a simultaneous approval in the United Kingdom, emphasizing scalable manufacturing. Additionally, FDA granted regenerative medicine advanced therapy (RMAT) designation to Genascence’s first-in-class gene therapy, GNSC-001, targeting knee osteoarthritis, validating advanced therapy pipelines.

3. Are antibody-drug conjugates (ADCs) replacing traditional biologics in breakthrough status awards?

FDA is heavily prioritizing ADCs, granting breakthrough therapy designation to two separate next-generation ADCs in August 2025: one targeting B7-H3 for small cell lung cancer, ifinatamab deruxtecan, and a bispecific ADC for epidermal growth factor receptor-mutant non-small cell lung cancer. This suggests ADCs are crucial in the next wave of oncology innovation.

4. Why did regulatory bodies prioritize updates to RNA-based vaccines?

Public health mandates drove the need for rapid review, demonstrated when FDA approved Moderna’s updated Spikevax and mNEXSPIKE COVID-19 vaccines in August 2025, specifically targeting the LP.8.1 variant for older adults and high-risk groups. This swift approval process supports the importance of addressing emerging SARS-CoV-2 variants through messenger RNA (mRNA) technology. However, it is important to note that regulatory sentiment for mRNA COVID-19 vaccines may swiftly change in the current environment, as evidenced by a recent internal memo circulated to FDA staff.

5. What market consequence does regulatory rejection carry for high-profile antibody candidates?

Rejection by influential international bodies signals immediate commercial risk; for instance, the Therapeutic Goods Administration (TGA) of Australia confirmed its decision to decline approval for Eisai’s monoclonal antibody lecanemab for early Alzheimer’s disease. This regulatory hurdle underscores the stringent global requirements for novel central nervous system therapies.

6. How does the oligonucleotide and specialty biologic landscape change via expanded labeling?

Expanded labeling significantly increases patient access and commercial reach. FDA approved an expanded label for IZERVAY (avacincaptad pegol intravitreal solution) in February 2025, allowing the oligonucleotide-based solution to be used without a limitation on the duration of dosing for treating geographic atrophy.

7. Which critical regulatory events signal manufacturing or clinical uncertainty for established biologics?

Regulatory extensions and review delays signal potential uncertainty. For instance, FDA extended the review period for GSK’s biologics license application for Blenrep (belantamab mafodotin-blmf), an ADC for relapsed/refractory multiple myeloma. Such extensions require careful planning by drug manufacturers regarding supply chain and launch timelines.

8. How does FDA’s interchangeable designation for high concentration biosimilars radically restructure the specialty drug market?

FDA granted interchangeable designation to Celltrion’s YUFLYMA (adalimumab-aaty) as a biosimilar to Humira (adalimumab) in April 2025. This designation applies to the high-concentration (100mg/mL) and citrate-free formulation of adalimumab, significantly increasing patient access and competition within the specialty biologic market.