Veralox Therapeutics Inc., a Maryland-based startup developing a small molecule for halting aberrant platelet activation and thrombosis, said the FDA has granted the candidate, VLX-1005, orphan drug status for the treatment of heparin-induced thrombocytopenia (HIT).
At the recent 39th J.P. Morgan Healthcare Conference, Biomarin Pharmaceutical Inc. popped the lid off top-line results from its ongoing phase III GENEr8-1 study with valoctocogene roxaparvovec – also known as valrox, now commonly called Roctavian. Data, though encouraging, may not have quelled controversy around the prospect.
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