Regulatory

FDA has approved BeOne Medicines’ Beqalzi for relapsed or refractory mantle cell lymphoma and Taiho Oncology’s Inqovi plus venetoclax for newly diagnosed acute myeloid leukemia, expanding treatment options for patients with difficult-to-treat blood cancers.

Reports also claim that Kyle Diamantas will serve as acting head of FDA.

FDA has approved Partner Therapeutics’ Bizengri for NRG1 fusion-positive cholangiocarcinoma, expanding precision oncology options for a rare subset of gastrointestinal cancer patients with limited targeted treatment alternatives.

FDA extended its review of Leqembi’s once-weekly subcutaneous starting dose for early Alzheimer’s disease by three months after requesting additional information from Eisai and Biogen.

Zentails announced the first dosage of Azenosertib administered in its phase III Aspenova trial to test azenosertib vs chemotherapy in Cyclin E1-positive platinum-resistant ovarian cancer.

FDA clears Cellenkos’ IND for CK0802, enabling a mid-stage trial in steroid-refractory GVHD.

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.