Voxzogo saves the day for Biomarin

Yesterday Biomarin confirmed what many had suspected: the company has yet to treat any patients with its haemophilia A gene therapy Roctavian. In the US, this is understandable, since the product only received an FDA nod in June. But it has been approved in Europe for almost a year. Biomarin did stick to its full-year Roctavian sales guidance of $50-150m, but if it is relying on the US market time could be tight – the group said it could take two to five months from initial interest to treatment. Biomarin added that it would only need to treat 50 US patients this year to hit the mid-point of its guidance. Meanwhile, the first German patient could be treated in late August, and negotiations are ongoing with payers in France and Italy. With scepticism around the Roctavian launch, at least the group’s achondroplasia therapy Voxzogo is picking up the slack, with second-quarter sales of $113m beating consensus of $96m. However, during a conference call yesterday Biomarin flagged supply constraints with this product. With these issues vexing investors, the group’s stock fell 3% in early trade.