BioMarin Receives FDA Rejection for Hemophilia Gene Therapy

The decision puts a major dent into BioMarin’s effort to come up with one-time gene therapies for the disease.
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Share of BioMarin Pharmaceutical  (BMRN) - Get Report cratered Wednesday after the biotechnology company revealed that the Food and Drug Administration decided against approval of its gene therapy for hemophilia A, a blood-clotting disease.

The company’s shares traded at $80.53, down 32.07%, and have dipped 4% so far this year, including Wednesday’s plunge.

The FDA determined that review of BioMarin’s application for the drug “is complete and that the application is not ready for approval in its present form,” the company said in a statement.

The decision obviously 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. BioMarin is one of several companies taking that route, according to Bloomberg.

BioMarin is apparently unbowed by the failure.

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

"We are surprised and disappointed that the FDA introduced new expectations for the first time in the Complete Response Letter. We are confident in valoctocogene roxaparvovec gene therapy and its potential to redefine the treatment paradigm for people with hemophilia A," the CEO added.

The company said, “The application was based on the Phase 3 study interim analysis of study participants treated with investigational product manufactured by the to-be-commercialized process and three-year data from the Phase 1/2 Study.”

After the news, Stifel analyst Paul Matteis downgraded BioMarin stock to hold from buy and slashed his price target to $100 from $127.