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.

The Supreme Court has ruled 5-4 to allow the Trump administration’s NIH funding cuts to continue, impacting more than 1,700 medical research grants in areas including heart disease, HIV/AIDS, Alzheimer’s disease, and mental health.

The agreement values Mural Oncology at up to $36.2 million, with shareholders set to receive a base cash payment and potential additional consideration tied to closing net cash.

Regeneron Pharmaceuticals announced FDA extended review timelines to Q4 2025 for two regulatory submissions related to Eylea HD due to a major amendment stemming from a third-party manufacturing inspection. Despite the delay, Eylea HD remains available in the U.S., and Regeneron anticipates a swift resolution as they address manufacturing concerns and continue expanding treatment options.

The collaboration will leverage Boehringer Ingelheim’s manufacturing expertise to accelerate AnGes’ HGF gene therapy toward regulatory approval and patient access.

A joint statement lays out the framework for a potential trade agreement.

The company will be heavily investing over the next decade to build a new biopharmaceutical plant in Holly Springs, NC, creating 120 jobs and reinforcing its $55 billion pledge to strengthen US manufacturing, R&D, and innovation.

Johnson & Johnson assures no impact on current treatments for multiple myeloma.

Staff members published an open letter to the HHS Secretary following a deadly shooting earlier this month.

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 company is downsizing its gene therapy operations following reduced demand from a major client, even as industry reliance on CDMOs and CMOs for capacity and expertise continues to grow.

Jazz Pharmaceuticals partners with Saniona to develop SAN2355, a promising epilepsy treatment, enhancing options for neurological disorders.

Pharm Exec unveils its new slate of rising leaders in the biopharma industry—profiling the stories of five executives steering innovative scientific and strategic pursuits in R&D, market access, and patient support.

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.

The regulatory body determined that PTC Therapeutics’ New Drug Application for vatiquinone did not provide sufficient evidence of efficacy in patients with Friedreich’s ataxia.

The comments came after the AAP announced its vaccine recommendations, which go against those of the CDC.

The move is likely part of the company’s goal to reduce costs as it faces increased competition in the GLP-1 space.

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.

The EMD Serono president discusses his new role and goals for the company.

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.

Rob Abbott, CEO, ISPOR, highlights how AI is streamlining research and improving patient care while stressing the importance of human oversight.

The AAP diverges from CDC guidelines, advocating for Covid-19 vaccines in young children, igniting debate over vaccine policy and public health integrity.

Why pharma and all stakeholders in the healthcare ecosystem should undertake a fundamental shift in how evidence to support new treatments is generated and assessed.

The disease doesn’t have any FDA approved treatments, leaving families desperate for FDA to approve the treatment.