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SpringWorks Therapeutics sprung out of Pfizer’s storeroom, when a rare disease advocacy group pushed to keep a program for neurofibromatosis alive. This method could work for “every rare disease under the sun,” advocates say.

SpringWorks Therapeutics is the perfect case study for rescuing a discontinued assets. It’s time to repeat the process for every rare disease, experts say.

The industry remains unwavering in the commitment to increased clinical trial accessibility and representation.

Price-negotiation provisions that are out of step with reality are discouraging funders and Big Pharma partners from investing in potentially transformative therapies. Fixing some of the unintended consequences of the IRA will clear the way for innovative medicines to reach patients in need.

Non-opioid pain therapies are entering an unprecedented era, marked by the landmark FDA approval of Vertex’s Journavx and a growing number of alternative approaches. Their ultimate uptake, however, remains to be seen.

With the modality now in early clinical trials, experts say more efficiency, broader editing capabilities and delivery breakthroughs are needed to propel RNA editing to the next stage.

Morale is low at the FDA, which was hit with layoffs this week following RFK Jr.’s confirmation. Biopharma leaders and agency insiders fear further workforce cuts could delay new medicines.

2023 marked the most bankruptcies in biopharma in more than a decade, with 14 companies filing for Chapter 11 protection. The number remained high in 2024.

A cautionary tale illustrates how forging a deal with a Big Pharma can have unexpected and far-reaching tax consequences.

Elisa Cascade, CPO at Advarra explains why addressing the challenges faced by clinical research sites is vital for improving patient trust and the overall viability of clinical trials.