Regulatory

Europe’s Joint Clinical Assessment promises harmonization, but it introduces a new layer of strategic complexity that manufacturers must navigate at both EU and national levels.

Ron Wyden led 7 Democrats demanding details from Pfizer, AstraZeneca, Novo Nordisk, and Eli Lilly.

FDA approved GSK's Wellcovorin as the first treatment for cerebral folate deficiency caused by a rare genetic variant.

The application is based on Phase III trial data showing the drug reduced the risk of invasive disease recurrence or death.

The vaccine chief briefly left the role last summer before almost immediately returning to it.

FDA informed uniQure it will not accept external control data as the primary basis for approving AMT-130, instead demanding a prospective, randomized, sham surgery-controlled trial before moving forward.

Alice Valdur Curran discusses how the industry continues to strategize around this complicated order.

Defining the “active ingredient” is the central challenge of biologic PTE, because biologics are large, complex molecules that do not fit neatly into a statutory framework built for small molecule drugs.

FDA approves a supplemental BLA for BioMarin Pharmaceutical Inc.’s Palynziq, expanding its use to adolescents 12 and older with PKU.

FDA expands approval of Novo Nordisk’s once-weekly Sogroya to include three new pediatric growth disorder indications, offering children as young as 2.5 an alternative to daily growth hormone injections backed by Phase III non-inferiority data.

FDA grants accelerated approval to Boehringer Ingelheim’s Hernexeos (zongertinib) for treatment-naïve HER2-mutant advanced NSCLC.

The approval makes Dupixent the first FDA-approved therapy for allergic fungal rhinosinusitis, supported by Phase III data showing significant improvements in sinus opacification, nasal symptoms, and reductions in surgery and systemic corticosteroid use.

The agency converted Braftovi’s accelerated approval to full approval after Phase III BREAKWATER data demonstrated significant improvements in progression-free and overall survival in patients with previously untreated BRAF V600E–mutant metastatic colorectal cancer.

BioNTech filed a patent infringement lawsuit against Moderna alleging unauthorized use of patented mRNA technology.

The judges ruled against tariffs levied using an emergency law.

The vaccine advisory committee’s meeting has yet to be rescheduled.

FDA is shifting its default standard for drug approvals, announcing that one adequate and well-controlled clinical trial will generally be sufficient for approvals.

Following a Type A meeting with the FDA’s Center for Biologics Evaluation and Research, Moderna’s flu vaccine candidate mRNA-1010 has been accepted for formal review after the company revised its regulatory strategy.

HHS Secretary Robert F. Kennedy Jr. reorganized senior leadership at the department to streamline management and advance the administration’s health policy priorities.

The new once-monthly dosing schedule for Rybrevant Faspro in combination with Lazcluze offers a more convenient option and also maintains efficacy and safety comparable to the prior bi-weekly regimen.

Without new legislation, pilot programs, or years of rulemaking, the agency could use enforcement discretion to create a fast, risk-based pathway for low-risk early-stage trials.

The letter follows Phase II data that did not sufficiently link biomarker reductions to clinical benefit.

Eli Lilly's $1.5 billion pre-launch inventory of orforglipron signals an aggressive manufacturing strategy aimed at avoiding supply shortages and capturing early share in the competitive obesity market.

The withdrawal is due to data shared from FDA reflecting that data included in the NDA submission is unlikely to meet the required threshold of evidence.

The approval of Keytruda (pembrolizumab) and Keytruda Qlex was supported by Phase III KEYNOTE-B96 data showing statistically significant improvements in progression-free and overall survival in patients with PD-L1–positive platinum-resistant ovarian, fallopian tube, or primary peritoneal carcinoma.