Regulatory

Key points from our recent webinar on the President’s MFN order.

Approval was based on data from the Phase IIb SunRISe-1 trial (NCT04640623), in which Inlexzo demonstrated an 82% complete response rate in patients with Bacillus Calmette-Guérin-unresponsive non-muscle invasive bladder cancer.

The agency noted ongoing safety violations at the location.

The company says addressing the issues raised in the CRL could take years.

The list prevents the import of ingredients from potentially dangerous facilities.

Wayrilz is the first Bruton’s tyrosine kinase inhibitor to be approved for adults with persistent or chronic immune thrombocytopenia who have not responded adequately to prior therapy.

Lilly has temporarily halted UK shipments of Mounjaro until Sept. 1, when new list prices take effect.

FDA approves COVID-19 vaccines from Moderna, Pfizer-BioNTech, and Novavax targeting the LP.8.1 sublineage of SARS-CoV-2, with eligibility limited to adults aged 65 years and older and those with underlying medical conditions that place them with a high risk for severe disease.

New indication positions Repatha as an option for high-risk adults with elevated LDL cholesterol and no prior cardiovascular events.

Dawnzera is the first and only RNA-targeted prophylactic therapy to be approved for hereditary angioedema in adults and pediatric patients aged 12 years and older.

Rob Abbott, CEO, ISPOR, highlights how uniting regulators, payers, and academic leaders worldwide can accelerate access to innovative treatments and strengthen evidence-based healthcare.

The clinical hold is due to questions about BX004’s nebulizer device, temporarily pausing US enrollment in the cystic fibrosis Phase IIb trial (NCT06998043) while European dosing continues.

Ron Lanton, partner, Lanton Law, warns that ongoing lawsuits targeting vaccine policy could create ripple effects across FDA decision-making and reshape how future emergency use authorizations are granted.

EVERSANA’s Alan Crowther discusses how pharma can prepare for the impact of the order.

Ron Lanton, partner, Lanton Law, warns that recent changes to federal vaccine recommendations could trigger extensive litigation as insurers, providers, and patients face new access and coverage challenges.

Tomnya is the first new fibromyalgia therapy approved by the FDA in over 15 years.

Expanded approval of Wegovy was based on results from the Phase III ESSENCE trial (NCT04822181), which demonstrated significant improvements in patients with metabolic dysfunction-associated steatohepatitis and liver fibrosis compared to placebo at 72 weeks.

The order aims to set US drug prices at the same level as the lowest cost in other markets.

An examination of proactive preparedness and readiness review for future enrollment in the agency’s QMM program—now in the pilot stage as an effort to incentivize pharma manufacturers to achieve a more mature state of quality culture.

Papzimeos is the first-and-only approved therapy for adults with recurrent respiratory papillomatosis.

The federally created panel was previously disbanded in 1998.

Attorney General Paxton argues that the company’s programs and offerings were essentially bribes and resulted in millions in dollars charged to the state’s Medicaid program.

Brinsupri is a first-in-class DPP1 inhibitor that has been found to inhibit activation of neutrophil enzymes that drive chronic airway inflammation in non-cystic fibrosis bronchiectasis.

Accelerated approval was based on results from the Phase Ib Beamion-LUNG 1 trial (NCT04886804), which showed a 75% objective response rate in patients with unresectable or metastatic non-squamous non-small cell lung cancer treated with Hernexeos.

Vinay Prasad returns to lead the FDA's CBER after leaving in late July.