Also known as: CBER
The FDA reversed its initial rejection of Moderna's mRNA flu vaccine, agreeing to a normal review after public backlash, while a federal judge invalidated the ACIP advisory committee, leaving the US without functioning vaccine oversight. Moderna's vaccine showed 26.6% greater efficacy than standard flu shots in clinical trials.
The FDA has granted accelerated approval to Denali Therapeutics' Avlayah (tividenofusp alfa-eknm), the first therapy targeting neurological symptoms of Hunter syndrome. The approval was based on a surrogate endpoint measuring heparan sulfate reduction in cerebrospinal fluid, with confirmatory study results required for full approval. The global Hunter syndrome treatment market is projected to reach $2.6 billion by 2033.
Disc Medicine has agreed with the FDA to resubmit bitopertin for rare blood disorder erythropoietic protoporphyria, with the agency allowing the Phase 3 APOLLO trial to support a new filing. The agreement follows a February rejection and could lead to a mid-2027 decision. Disc has implemented workforce cuts and its CFO sold shares amid the regulatory setback.
The FDA reversed its refusal to review Moderna’s mRNA flu vaccine application and accepted a revised filing with split age cohorts and a post-market study for adults over 65. The reversal came amid broader vaccine regulatory uncertainty and industry concern about unpredictable review timelines.
The global antibody drug conjugate market is calculated at USD 14.76 billion in 2026 and is expected to reach USD 32.66 billion by 2035. North America held a 53% share in 2025, while Asia Pacific is projected to grow fastest.
FDA released its 2026 Rare Disease Hub Strategic Agenda and a draft guidance on 3-year New Clinical Investigation Exclusivity. The agency also outlined new pathways including the plausible mechanism pathway and Rare Disease Evidence Principles to accelerate treatments for rare diseases.
The Consolidated Appropriations Act, 2026 extends the FDA’s rare pediatric disease priority review voucher authority through Sept. 30, 2029. The law also requires a GAO report on the program’s effectiveness.
The FDA will now allow drug approval based on a single pivotal trial instead of two, a policy change confirmed in a New England Journal of Medicine paper. The new guidance maintains requirements for confirmatory evidence and focuses on trial design quality, aiming to reduce development costs and speed drugs to market. FDA officials argue this approach may actually improve standards by concentrating scrutiny on a single well-designed study.
The FDA issued reforms on January 11, 2026, loosening chemistry, manufacturing, and control requirements for cell and gene therapy products to advance innovation and accommodate the unique complexity of these treatments.
The FDA has established a "plausible mechanism pathway" to approve personalized genome editing and RNA-based therapies for rare and ultra-rare diseases without requiring large randomized controlled trials, streamlining access to individualized treatments.
