Regulatory

In order to enforce MFN, regulatory agencies must navigate the complex process of determining a drug’s true value.

The President announced FDA will no longer recommend the use of the common painkiller during pregnancy, despite a casual link between the medication and autism not being established.

Alongside regulations from the IRA, oncology R&D is set to face unique challenges when MFN is enforced.

Peter Rubin discusses how the industry is an ecosystem and each of the parts will feel the impact differently.

The FDA has expanded the indication of Incyte’s Opzelura (ruxolitinib) cream to include children aged two to 11 years with mild to moderate atopic dermatitis, making it the first steroid-free topical treatment option for this age group.

FDA warns major pharmaceutical companies to improve transparency in drug advertising, addressing misleading claims and enforcing stricter regulations.

R-DXd has previously shown promising clinical results in platinum-resistant ovarian, peritoneal, and fallopian tube cancers.

The new administration has a different perspective of vaccines than the previous administration, causing confusion with the public.

The pause follows a patient death in the SYNRGY trial evaluating the novel therapy for STXBP1-related disorders, as the company prioritizes safety and transparency with the community.

The designation supports expedited development of SAR402663, Sanofi’s one-time gene therapy designed to reduce treatment burden and slow vision loss in patients with wet age-related macular degeneration.

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.