I think this is a perfectly reasonable price, the article even mentions that it undercuts one of their competitors, and very very few people will actually pay anything close to full price. I think there is a broader conversation to be had though - should the compensation for developing a drug really be tied to patients in this sort of way? There have been some interesting proposals that involve paying the drug company a prize for successfully developing a drug and then regulating the prize to variable cost - especially in a single payer system I could see that being a great way to incentivize drug development for diseases that the nation values the most while removing the burden of paying from individual patients more efficiently than through a complex insurance system.

The pricing of this gene therapy has been much anticipated as it’s expected to set a precedent for future products.

SMA is a terrible disease where babies born with Type 1 are never able to sit independently and typically die before age 2. From my understanding, most of the babies in trial are still alive (after age 2) and most of them can sit independently. Really amazing outcomes.

To get a sense of the the SMA market is like, Biogen’s drug, Spinraza is priced at $750k for the first year and $375k for every year thereafter, for life.

So if this drug works for more than 5 years with a single dose, this $2M product will actually save the healthcare system money.