As updated Phase I/II results for BioMarin's hemophilia A and Spark's hemophilia B gene therapies were presented on May 22, it was revealed that FDA plans to make hemophilia the first gene therapy indication eligible for accelerated approval based on a surrogate endpoint.

There's a lot of finger-pointing in the industry when it comes to why pharma has a bad reputation. Maybe it's time everyone who works in pharmaceuticals takes responsibility for it.

Wells Fargo analyst David Maris warned biopharma investors that the US drug market is on a long-term trajectory toward price controls, which he characterized as an under-appreciated risk for the sector, ahead of President Trump's speech on drug pricing.

Express Scripts has agreed to give Praluent preferred access over Amgen's Repatha on its national formulary and is removing restrictions that have been a significant barrier to market access for the PCSK9 inhibitors.

Price transparency reports from the big pharmas also show continued increases in price concessions in 2017, but a slow-down in list price increases.

Roche used its ASCO investor briefing to highlight its digital and personalized healthcare strategy, including examples of how it’s facilitating R&D and reimbursement – and how that should position it to be an oncology leader in the future. 

Alliance for Regenerative Medicine initiative seeks to explain gene and cell therapy to the general public. Also, EY's Giovannetti says strategic decisions, not tax reform, should drive deal-making; FDA's Woodcock touts benefits of new drug review office overhaul; and Siga Technologies eyes priority review voucher upon approval of Tpoxx for smallpox.

Are drug firms being lax when it comes to Phase IV studies in markets like India or are certain arbitrarily determined regulatory requirements largely to blame? Scrip delves into the issue.

New FDA labeling brings Tafinlar/Mekinist into adjuvant melanoma setting, but Bristol's competing Opdivo already has captured nearly 50% of the market and is approved regardless of genetic mutation.

Company believes there is a path forward for developing ribaxamase in Phase III, based on discussions with the FDA, but deaths in Phase II meant loss of breakthrough designation.

The US FDA gives Sweden's privately held Modus Therapeutics rare pediatric disease designation for sickle cell disease.