Regulatory

The idea of denying people proven medicines in the name of research has a dark legacy in this country.

Approval was based on results from the Phase II TROPION-Lung05 trial, which showed that Datroway demonstrated a strong overall response rate and duration of response in patients with previously treated, locally advanced or metastatic EGFR-mutated non-small cell lung cancer.

Approval was based on results from the Phase III inMIND trial, which demonstrated a statistically significant and clinically meaningful improvement in progression-free survival in patients treated with Monjuvi combined with rituximab and lenalidomide for relapsed or refractory follicular lymphoma.

Dupixent is the first and only targeted therapy to receive FDA approval for bullous pemphigoid.

Harliku becomes the first FDA-approved treatment for alkaptonuria, indicated to reduce homogentisic acid levels in affected adults.

Approval of first twice-yearly HIV pre-exposure prophylaxis was based on results from the Phase III PURPOSE 1 and PURPOSE 2 trials, which show that ≥99.9% of patients treated with Yeztugo remained HIV-negative.

The investigational MRI contrast agent, gadoquatrane, is designed to deliver effective imaging at a significantly reduced gadolinium dose, supporting safer repeat use in adults and pediatric patients.

Special Guest Opinion: A reimagined regulatory framework for the life sciences is not just pro-investor—it is pro-patient.

Under the new initiative, companies may receive a voucher enabling FDA review to be shortened from the standard 10–12 months to just 1–2 months following final application submission if the drug addresses US national health priorities.

Andembry is the first prophylactic therapy for hereditary angioedema to target factor XIIa, inhibiting the top of the inflammatory cascade that drives attacks.

The bill is in line with comments RFK Jr. has made about this form of advertising.

mRESVIA is now authorized for use in adults aged 18–59 years with an increased risk of respiratory syncytial virus-related lower respiratory tract disease.

Keytruda marks the first perioperative anti-PD-1 treatment option for adults with PD-L1–positive resectable locally advanced head and neck squamous cell carcinoma.

Approval of Zusduri was based on data from the Phase III ENVISION trial, which showed a 78% complete response at three months in patients with recurrent low-grade, intermediate-risk non-muscle invasive bladder cancer.

Mavyret marks the first and only direct-acting antiviral approved to treat both acute and chronic hepatitis C virus infections in adults and children aged three years and older, without cirrhosis or with compensated cirrhosis.

Approval was based on results from the Phase II TRUST-I and TRUST-II trials, which demonstrated confirmed overall response rates of up to 90% in patients treated with Ibtrozi for locally advanced or metastatic ROS1-positive non-small cell lung cancer.

Delivered as a once-daily 30 mg/mL IV bolus injection over 15 seconds, Xifyrm can be used alone or alongside non-NSAID analgesics as part of a multimodal pain strategy.

Regulatory action was based on data from the Phase III IMforte trial, which showed that Zepzelca plus Tecentriq significantly extended both progression-free survival and overall survival in patients with extensive-stage small cell lung cancer.

Approval of Enflonsia was based on data from the Phase IIb/III CLEVER trial, which showed a 60.5% reduction in medically attended lower respiratory infections and an 84.3% reduction in respiratory syncytial virus-related hospitalizations.

Widaplik marks the first and only FDA-approved triple combination medication that can be used as an initial therapy for patients with hypertension who require multiple drugs to reach their blood pressure reduction goals.

FDA concluded that AXS-14’s New Drug Application was not sufficiently complete for substantive review, citing concerns with one of two clinical trials in fibromyalgia patients.

Approval was based on results from the Phase III ARANOTE trial, in which Nubeqa demonstrated a 46% reduction in the risk of radiographic progression or death when combined with androgen deprivation therapy, in patients with metastatic castration-sensitive prostate cancer.

New drug application submission was based on positive data from the Phase III GLISTEN trial, which demonstrated significant and sustained reductions in pruritus and related sleep disruption in patients treated with linerixibat.

The industry is facing challenges from the new administration that could have serious impacts on everything from market access, pricing, and supply chains.

Approval was based on data from the Phase III NextCOVE trial, which demonstrated that mNEXSPIKE met non-inferiority criteria compared to the original Spikevax as a vaccine for COVID-19.

The biopharmaceutical sector is underpinned by a unique ecosystem that combines public funding for basic research, university-led translational science, technology transfers, venture capital funding, partnerships with larger companies, and a relatively transparent and responsive regulatory system.

Approval of Nucala marks the first biologic approved for chronic obstructive pulmonary disease patients with blood eosinophil counts as low as ≥150 cells/μL.

Approval of Susvimo marks the first continuous delivery therapy indicated for diabetic retinopathy.

Clearance of ASC50 paves the way for a Phase I trial in patients with mild-to-moderate plaque psoriasis.

In an article recently published by The New England Journal of Medicine, FDA higher-ups Vinay Prasad, MD, MPH; and Martin A. Makary, MD, MPH, wrote that any new COVID-19 vaccine must now be evaluated in placebo-controlled studies.