Regulatory

Policy changes like Most-Favored Nation (MFN) Pricing are creating new operational requirements for pharmaceutical companies to prove every dollar delivers value.

FDA approves Auvelity for Alzheimer’s-related agitation, marking the first therapy targeting NMDA and sigma-1 receptors for this high-burden neuropsychiatric symptom and expanding treatment options for millions of patients.

Kaida Biopharma’s chair and acting CEO discusses how President Trump’s MFN policies and other regulatory actions are impacting pharma’s ability to invest in new treatments and adjusting international launch strategies.

Rocket Pharmaceuticals sells it's priority review voucher for $180M, securing non-dilutive funding to extend runway and advance gene therapy pipeline.

FDA approves Breztri Aerosphere for asthma, introducing the first single-inhaler triple-combination maintenance therapy for patients aged 12 and older.

Ireland has built a track record of regulatory excellence that few regions have matched.

Experts convened for a panel discussing how tariffs and MFN policies are impacting drug pricing and what regulatory hurdles they face ahead of the midterms later this year.

FDA approves Caplyta for relapse prevention in schizophrenia, adding long-term Phase III data showing a 63% reduction in relapse risk and reinforcing its role in sustained disease management.

Dr. Stella Vnook discusses how pricing transparency and VBC might have a more positive impact than MFN and other pricing regulations.

FDA granted Compass Pathways with a rolling review for COMP360, a proprietary formulation of synthetic psilocybin, in treatment-resistant depression.

FDA issues Complete Response Letter to AbbVie’s trenibotulinumtoxinE, delaying a potential first-in-class aesthetic neurotoxin over manufacturing questions despite no identified safety or efficacy concerns.

Traditional randomized trials are often impractical for treatments in the rare and ultra rare disease space.

Early clinical data from the CHORD trial supported accelerated approval of the first gene therapy targeting OTOF-related hearing loss under the FDA Commissioner’s National Priority Voucher program.

FDA expands its approval of Tzield to children as young as one with stage 2 type 1 diabetes, extending the first disease-modifying therapy into an earlier, high-risk population to delay progression to insulin-dependent disease.

FDA expands Dupixent approval to children aged 2–11 with chronic spontaneous urticaria, marking the first biologic option for this population and extending its reach across type 2 inflammatory diseases.

The Trump administration’s new executive order aims to accelerate FDA review of psychedelic therapies for mental health conditions, highlighting a potential shift in regulatory timelines and expanded access pathways,

Samsung Bioepis’ SVP and head of US commercial discusses the pricing, regulatory, and broader issues impacting the biosimilars market.

The President also announced a number of other key leadership appointments at federal health agencies.

A new ICER white paper calls for reforms to the FDA’s accelerated approval pathway, citing ongoing challenges with regulatory consistency, confirmatory trial delays, and transparency.

Eli Lilly releases new safety data after FDA requested additional data to conduct extensive post-approval safety studies for Foundayo, including long-term monitoring of cardiovascular, liver, and thyroid cancer risks.

Elevating real-world data (RWD) to the status of regulatory-grade evidence will bring both opportunity and challenges to the industry.

Replimune received a second FDA complete response letter for RP1 in advanced melanoma, raising questions about the viability of its oncolytic immunotherapy program.

China approved Amgen’s tarlatamab for previously treated small cell lung cancer, expanding access to the bispecific immunotherapy.

Dan Bell, chief strategy officer at Marken, discusses how biopharma companies and logistics teams are responding to the dual disruptors to global supply chain operations.

Dismissing the role of independent, outside experts in FDA decision-making weakens the drug review process.