BioMarin stock increases; Why its next launch could be among its biggest

BioMarin shares rose Friday after winning Food and Drug Administration approval for its dwarfism treatment in children as young as 5 years old.


The drug called Voxzogo treats achondroplasia, a form of short-limbed dwarfism. With the approval, patients in the United States can begin treatment as young as 5 years old BioMarin Pharmaceutical (BMRN) drug encourages growth before a patient’s growth plates close.

Piper Sandler analyst Christopher Raymond noted that FDA approval “is just down the fairway.” In Europe, the drug is allowed for children from 2 years of age.

“Most importantly, with this approval, BioMarin’s next growth cover is finally unlocked,” he said in a report to customers. “We model Voxzogo revenue of $ 165 million, $ 449 million, $ 558 million and $ 630 million in fiscal years 2022-25.”

In today’s stock market, the BioMarin stock rose 10.3% to close at 91.47. The news sent the BioMarin stock to its highest point since January.

BioMarin shares pop after FDA approval

In children with achondroplasia, the gene that controls bone tissue growth contains a mutation. It is a spontaneous mutation in 80% of patients. Others have a hereditary mutation. Voxzogo bypasses this gene to promote endochondral bone growth.

This is also the first treatment option for children with achondroplasia, BioMarin CEO Jacques Bienaime said in a written statement.

“Voxzogo is a medical first that is rooted in BioMarin’s focus on molecular genetics and addresses the underlying cause of the condition,” he said. “More than a decade of scientific research underpins the medical advances that Voxzogo represents.”

Piper Sandlers Raymond retained its overweight rating and 121 price targets on the BioMarin share after approval.

Why this launch could be great

SVB Leerink analyst Joseph Schwartz expects the Voxzogo launch to be among BioMarin’s largest. He raised his price of the BioMarin share to 112 from 108 days before the approval.

Voxzogo aims to increase the annual growth rate – how much and how fast a child grows over the course of a year. Schwartz recognizes that achondroplasia is a complex disorder. In the test, Voxzogo showed tendencies toward improving body proportionality. But the results were not statistically significant.

“However, according to our study, physicians believe that annual growth rate improvement is meaningful and is likely to translate into additional benefits, including improvements in quality of life,” he said in a note.

Schwartz’s medical study suggests that Voxzogo could capture about 25% of the market at its peak. He has an outperform rating on the BioMarin share.

Follow Allison Gatlin on Twitter at @IBD_AGatlin.


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