A Phase 1/2 study of BioMarin Pharmaceutical’s gene therapy for phenylketonuria has been placed under a clinical hold after interim results from a preclinical study showed that some mice developed liver tumors. Patients did not receive the same high dose that was given to the mice, and no one in the clinical trial has developed cancer.
The biotech company said the FDA had given priority review to valoctocogene roxaparvovec for hemophilia A in adults, with the PDUFA date set at Aug. 21.
Canada has a proud history of achievement in the areas of science and technology, and the field of biomanufacturing and life sciences is no exception.
Should unproven drugs be made available to terminally ill patients? Vice President Mike Pence looks to be preparing that discussion for the national stage, hosting "Right to Try" advocates at the White House.
BioMarin Pharmaceutials is selling the global license for its PARP inhibitor talazoparibin to Medivation in a $570 million deal. Hatteras Venture Partners has raised $90 million as part of its first close for its fifth venture fund.
As a whole host of new Duchenne Muscular Dystrophy therapies mature down the regulatory pathway, the global market for the degenerative disease is expected to expand at an incredible rate – from $8.2 million in 2014 to $990 million in 2019, or so says market research firm GlobalData. The present market’s got only paltry offerings to treat this […]
Life sciences M&A activity – and buzz – has hit a fever pitch in the past few months. As the industry braces itself for biosimilars to hit it big and precision medicine to drive development in targeted therapy, even more acquisitions are on the horizon. BioPharma Dive has compiled a tantalizing list of some potential acquisitions: […]
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By Kurt R. Karst – Last Friday, BioMarin Pharmaceutical Inc. (“BioMarin”) and FDA announced (here and here) the approval of a BLA for a new biological product for patients with Mucopolysaccharidosis type IVA, also known a “Morquio A syndrome”: VIMIZEN (elosulfase alfa) Injection. The approval of VIMIZEN not only marks the first FDA-approved product for Morquio […]