WSJ: Gene Therapy That Causes Hemophilia Remission To Be Priced At $2-$3M

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BioMarin Pharmaceutical Inc. is seeking millions of dollars for its new hemophilia gene therapy treatment.

BioMarin Pharmaceutical Inc. is seeking a price between $2 to $3 million for its new hemophilia gene therapy, according to The Wall Street Journal.

If this price is approved, it would make the drug the most expensive treatment in the world. No price has been set in stone for the treatment, but recent statements by Chief Executive Jean-Jacques Bienaimé at the JPMorgan Healthcare Conference make it seem like the price will end up in that range.

At the conference, Bienaimé stated that the company has spoken with insurers about the price and they have indicated comfortability within that range. The gene therapy would be the first approved in the US for an inherited type of hemophilia.

The treatment has gone through clinical trials where it delivered exceptional results. Patients that were given an injection with the treatment saw bleeding episodes drop from an average of 16.5 to an average of 0. The treatment lasted over three years.

Bienaimé defended the price range further by citing that the cost of treating hemophilia over a lifetime could be as high as $25 million, making the treatment a fraction of that cost and saving patients' lives and money.

BioMarin Pharmaceutical Inc. is seeking millions of dollars for its new hemophilia gene therapy treatment.

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