Impact on BioMarin's Sentiment: Voxzogo's Triumph Clouded by Uncertainty

Raymond James initiated coverage on BioMarin Pharmaceuticals Inc BMRN with a Market Perform rating, noting sparsity in near-term growth drivers. 

Analysts Danielle Brill and Alex Nackenof say the launch of Roctavian and Voxzogo are the company's key growth drivers. 

While Voxzogo's growth is expected to continue over the next few years, they worry about its longer outlook given competitive treatment options that are advancing in the clinic, including BridgeBio Pharma Inc BBIO, Ascendis Pharma A/S ASND. 

The analyst also expects a slow ramp for Roctavian, given 

the hemophilia doc survey work pointing to limited plans for early adoption. 

BioMarin has guided Roctavian sales of $50-150 million in 2023 revenues. While the company may meet the bar for 2023 (with some small pent-up demand), overall appetite from the hemophilia community for the product is low, says the analyst.

Voxzogo launch in achondroplasia has been a success, with ~$380 -430 million in revenues expected this year. 

Though there is ~20% upside potential for achondroplasia alone – given the recent advances from competitor BBIO's much more convenient oral FGFR3-inhibitor, Voxzogo's future is in jeopardy. 

BioMarin's shares will remain under pressure until BBIO reports 12-month data (anticipated early-mid 24). 

Raymond James also questions the potential value of BMRN's two clinical-stage pipeline assets: BMN331 for HAE and BMN255 for hyperoxaluria. 

The HAE market is becoming increasingly competitive, and though BMN255 shows promise as a convenient oral treatment for hyperoxaluria, it is still in its early stages.

Price Action: BMRN shares are down 1.47% at $89.04 on the last check Thursday.