Another drug company explores $2M-$3M price tag for therapy

The world could soon have a new most expensive drug if an experimental gene therapy for hemophilia patients could come in with a price tag between $2 million and $3 million is approved, The Wall Street Journal reported.

The drugmaker, BioMarin Pharmaceutical, is exploring the price range for its drug that has shown to be very effective in cutting bleeding episodes to zero for patients affected by hemophilia, which causes frequent bleeding due to a missing or deficiency blood-clotting protein. The drug would be the first approved gene therapy in the U.S. for an inherited type of hemophilia, according to the WSJ.

And insurers may be receptive to the price, BioMarin chief executive Jean-Jacques Bienamé said at the J.P. Morgan Healthcare Conference in San Francisco this month. The overall cost of care of these patients could drop as a result of the drug.

While the drug could become the most expensive in the world, at least one other, Zolgensma, has a price above $2 million already. Manufactured by Novartis, Zolgensma treats spinal muscular atrophy in children younger than two and is priced at $2.125 million for a five-year course. The treatment is only intended for a small group of patients with the disease, but the drugmaker said it would lower the price if a larger group than expected uses it.

The rollout of new gene therapies with sky-high prices is becoming more common and causing insurers and drugmakers to work to find creative ways to pay for them.

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