The FDA approved BioMarin Pharmaceutical Inc's (NASDAQ:BMRN) Roctavian (valoctocogene roxaparvovec-rvox) gene therapy for adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5.

The one-time, single-dose infusion is the first approved gene therapy for severe hemophilia A in the U.S. Roctavian was approved by the European Medicines Agency in August 2022.

In March, the FDA said it needed more time to review a three-year analysis from BioMarin's ongoing Phase 3 GENEr8-1 study of Hemophilia A gene therapy, which BioMarin submitted earlier this year.

Hemophilia A is a genetic condition caused by a mutation in the gene responsible for producing a protein called FVIII, which is necessary for blood clotting.

Roctavian is designed to replace the function of the mutated gene, allowing people with severe hemophilia A to produce their own FVIII and thereby limit bleeding episodes.

Roctavian is manufactured at a BioMarin-owned facility in Novato, California.

BioMarin will continue to monitor the long-term effects of treatment with an extension study that will follow all clinical trial participants for up to 15 years, as well as post-approval studies to follow those dosed in a real-world setting for 15 years or more. 

It is estimated that there are approximately 6,500 adults living with severe hemophilia A in the U.S. BioMarin expects approximately 2,500 of those adults to be eligible to receive Roctavian with this initial approval.

William Blair writes that the impressive early launch of Voxzogo, the company's largest approved product to date, and U.S. and EU approvals of Roctavian represent major additional growth drivers.

The analyst sees strong potential for BioMarin to double its top line in the coming years. 

Noting the company's deep rare-disease pipeline, with a promising growth profile, the analyst views BioMarin as a core holding in the sector and rates BioMarin at Outperform.

Truist Securities analyst Robyn Karnauskas maintained Biomarin Pharmaceutical with a Buy and raised the price target from $125 to $140. 

Wedbush analyst Andreas Argyrides maintained Biomarin with a Neutral and raised the price target from $69 to $73.

Price Action: BMRN shares are up 3.17% at $91.75 premarket on the last check Friday.