Don Tracy, Associate Editor

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

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.

The draft assessment finds that the popular weight loss drugs are costly but worth the price tag, while also urging for broader coverage and policy solutions.

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.

Drugmaker aims to simplify operations, speed up decision-making, and reinvest savings into diabetes and obesity growth opportunities in latest round of pharma layoffs.

The new funding is expected to fuel clinical progress for Alchemab’s muscle atrophy candidate and expand its AI-driven pipeline targeting immune and neurological diseases.

The deal gives Avadel global rights to develop and market valiloxybate for narcolepsy and idiopathic hypersomnia, with a once-at-bedtime, sodium-free formulation designed to improve patient convenience and outcomes.

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.

A monthly roundup of business and people news in the pharmaceutical industry.

The collaboration provides BioArctic with an upfront payment of $30 million and potential milestone and royalty payments as the companies explore a novel drug candidate for severe neurological disorders.

The acquisition adds Furoscix, an FDA-approved furosemide infusion therapy for chronic heart failure and kidney disease, while strengthening MannKind’s pipeline and commercial presence in cardiometabolic and orphan lung diseases.

Shiladitya Sengupta, founder, Vyome Therapeutics, explains how the company’s strong ties in India’s biotech and healthcare sectors are helping to create innovative solutions for global markets.

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

Under terms of the deal, Gilgamesh could receive up to $1.2 billion as bretisilocin progresses through clinical development for the treatment of major depressive disorder.

Shiladitya Sengupta, founder, Vyome Therapeutics, explains how the company leveraged a reverse merger with ReShape Lifesciences to streamline its path to going public while minimizing costs and complexity.

The acquisition aims to expand Kite Pharma’s capabilities in in vivo CAR T-cell therapy research and development.

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 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.

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

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.

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.

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.

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 global biotherapeutics company announced that it will seek to streamline operations as part of a broader transformation to boost efficiency, refocus on core businesses, and reinvest in high-priority programs.

The collaboration will explore RNA-based small molecule drug candidates aimed at neurological conditions where existing treatment options remain limited.

Rob Abbott, CEO, ISPOR, highlights that the United States excels in areas like cancer screening and advanced medical technology while facing challenges such as rising costs, lack of universal coverage, and health outcomes that lag behind other high-income nations.

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