Keyword: BioMarin Pharmaceutical
11 would-be blockbusters span several therapeutic areas, including CNS, oncology, hematology, inflammatory diseases and metabolic disorders.
Five more pharma companies have joined founders BioMarin and Invitae in sponsoring free genetic testing for children who've had an unprovoked seizure.
Drug pricing remains the pharma industry's top concern in 2020, execs said in a survey, as political threats continue from Washington, D.C.
While BioMarin has filed its hem A gene therapy valrox for approvals in the U.S. and EU, Roche is only running a phase 3 lead-in study for SPK-8011.
8. Valoctocogene roxaparvovec
We've assembled a list of the top 10 most-anticipated new drug launches of 2020 based on estimated global sales in 2024. Of those 10, eight could break the blockbuster barrier in four short years—putting a new batch of heavy hitters in pharma's ranks.
Gene and cell therapies will make strides in 2020, but their developers will have to address challenges in manufacturing, reimbursement and more.
Previously, despite repeated delays, tendered Spark shares stayed relatively stable at around 20%—until now.
BioMarin is collaborating again with Believe Limited for an arts program to support people with hemophilia.