BioMarin Heads to FDA With Mixed Data For Rare Disease Drug
Summary
San Rafael, CA-based BioMarin (NASDAQ: BMRN) said in a statement that longer-term evaluations it has conducted of patients in the Phase 3 program provided “supportive evidence” of a link between reduced phenylalanine levels and an improvement in attention span.In a research note, RBC Capital Markets analyst Michael Yee called its approval prospects “murky and uncertain” given the drug didn’t hit its secondary goals and the cognitive improvement trend was exploratory only.He added that the path forward in Europe is “not completely clear yet as well and needs better visibility.” One in every 14,000 babies born in the U.S. is diagnosed with PKU every year, and the available treatment options are a strict diet and BioMarin’s own sapropterin (Kuvan), a pill that only works in about half of patients.Earlier this month, BioMarin released positive data from study of a drug for a form of the genetic condition Batten disease, and aims to apply for approval.The study is a small, open-label trial, and while RBC’s Yee wrote in a previous note that the drug appeared to produce a “major treatment effect,” he also added “whether FDA will approve this is not clear.” BioMarin shares dipped just over 5 percent in pre-market trading on Monday.