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BioMarin Stock Downgraded After FDA Rejects Hemophilia Drug

BioMarin shares were downgraded by analysts after the FDA failed to clear the biotech's gene therapy for hemophilia.
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BioMarin Pharmaceutical  (BMRN) - Get BioMarin Pharmaceutical Inc. Report shares on Thursday slipped as analysts downgraded them after the Food and Drug Administration failed to clear the biotech's gene therapy for hemophilia.

The San Rafael, Calif., company's stock recently traded at $75.42, down 1.7%. It had slumped 35% Wednesday and is down 11% year to date.

Citi analyst Mohit Bansal cut his rating on the stock to neutral from buy and slashed his share-price target to $86 from $148. 

The "surprising" FDA decision on Valrox means no approval is likely until late 2022, he wrote in a commentary, according to The Fly.

The FDA wants two-year follow-up data from the drug’s Phase III trial, which raises uncertainty and gives competitors an opportunity to catch up, he said.

RBC Capital analyst Kennen Mackay downgraded BioMarin to sector perform from outperform, cutting his share-price target to $92 from $123.

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The FDA’s action is "thesis-changing,” he wrote in a commentary cited by The Fly. It reduces management credibility, threatens revenue ahead of Kuvan's 2021 patent cliff and also increases worries over the drug itself.

On Wednesday, Stifel analyst Paul Matteis downgraded BioMarin stock to hold from buy and slashed his price target to $100 from $127

The FDA decision puts a major dent into BioMarin’s effort to come up with one-time gene therapies that can ameliorate severe conditions stemming from flaws in DNA, Bloomberg reports.

BioMarin is apparently unbowed by the setback.

"We remain committed to the hemophilia community and to leading the way to the first ever gene therapy in hemophilia A," Chief Executive Jean-Jacques Bienaimé said in a statement.

Meanwhile, BioMarin said on Thursday that it submitted a new-drug application to the FDA for vosoritide, an investigational once-daily injection for children with achondroplasia. 

The company called the disorder the “most common form of disproportionate short stature in humans.”