BioMarin inches closer to a gene therapy first – BioPharma Dive

Posted: February 22, 2020 at 2:41 pm

Dive Brief:

Though analysts continue to debate the particulars of a valrox launch, they generally expect the therapy to take a significant chunk of the hemophilia market. Cantor Fitzgerald found through a survey that doctors seem quite willing to prescribe valrox within a few years of its launch. SVB Leerink also saw patient and prescriber enthusiasm in its own research.

"We believe the hemophilia market may be less 'sticky' and more poised for disruption by next generation agents than is generally assumed based on historical behavior," SVB Leerink analysts wrote in an investor note last February.

For BioMarin, a market open for disruption could translate to big sales. According to Jefferies, consensus estimates on Wall Street put valrox sales at $44 million this year, growing to $190 million in 2021 and more than $1.3 billion in 2025. Last May, Jefferies said valrox accounted for 16% the value it modeled for BioMarin's stock price.

On the other end, valroxcould threaten leaders in the hemophilia drug space. Roche's blockbuster drug Hemlibra, which comes with convenient dosing and a high list price, looks particularly vulnerable. Survey data from Citi Research cited by BioMarin showed U.S. hematologists said they would switch close to 40% of their Hemlibra-treated patients to a gene therapy within three years of valrox's launch.

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BioMarin inches closer to a gene therapy first - BioPharma Dive

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