BioMarin approval ‘should be a win’ despite ValRox label confusion, says Stifel

Stifel analyst Paul Matteis notes the FDA approved BioMarin’s Roctavian and “encouragingly there’s no surprising monitoring requirements or boxed warnings,” adding that steroid use is directed as reactive, which “is a win.” This should represent a credibility boost for management, but “one oddity drove the stock down initially,” namely that the mean ABR reduction looks worse than previously reported on the FDA label since the FDA used different, conservative imputation methodology for 13 patients who went on prophylaxis. While this may make it look like BioMarin “had portrayed an overly-optimistic efficacy profile for Roctavian,” the “median” ABR data didn’t change and ultimately what matters most to prescribers/payors is whether, and for how long, Roctavian can enable HemeA patients to live freely without needing Hemlibra or factor prophylaxis. While BioMarin “encouragingly” reiterated 2023 Roctavian guidance, the Street remains skeptical after Germany and slow initial Hemgenix uptake, added the analyst, who has a Buy rating and $118 price target on BioMarin shares.