Valrox (valoctocogene roxaparvovec) from San Rafael Calif-based Biomarin Pharmaceutical Inc. moved one step closer to entering the U.S. market, with the company reporting that that the FDA had accepted for priority review the BLA for its investigational AAV5 gene therapy for adults with hemophilia A.
“Lack of knowledge is the true bottleneck to clinical translation. We need to stop telling basic scientists, especially trainees, that their work’s value lies in its translatability.” That is the unexpected advice of none other than William Kaelin Jr., whose scientific discoveries have proved to be both top-rate science and very translatable indeed. His work, for which Kaelin has won the 2019 Nobel Prize in Physiology or Medicine and a host of other awards, has enabled the development of multiple therapies targeting anemia and cancer, including vadadustat.
Millions of people are affected by blood disorders, and the prevalence is expected to grow as our population ages. It is not surprising that, according to the American Society of Hematology, the FDA approved several new therapies – or new indications for previously approved therapies – in 2019 for people living with non-malignant blood disorders. Those included two disease-modifying treatments for sickle cell disease and the first anticoagulant for venous thromboembolism management in children.
No matter how effective it is, a drug is worthless if the people who need it can’t afford it. That’s been almost an anthem for patients and policy wonks testifying before U.S. Congress on drug prices.
New analysis from Clarivate Analytics' Cortellis Forecast Team predicts 11 medicines set to enter the market in 2020 will reach more than $1 billion in sales by 2024.
Data for this report were compiled from Cortellis, the suite of life sciences intelligence solutions from Clarivate Analytics. Cortellis includes the broadest and deepest range of sources of intelligence across the R&D lifecycle, including annual filings, drug pipelines, clinical trials, patents, chemistry, deals, conferences and company announcements.
Crowned by a potential cure for severe hemophilia A, that could become the most expensive drug ever, a new list of 11 medicines expected to generate $1 billion-plus in annual sales by the end of 2024 or earlier throws into stark relief the growing tension between medical innovation and society's ability to pay for it. The 2020 Cortellis Drugs to Watch list, including medicines both approved and likely to be, points to a future of ongoing conflict between payers and industry spurred by fundamental disagreements.
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