[maxander]

The inherent cost of manufacturing the drug is relatively negligible. The problem is the cost of development and deployment, and the tiny customer (patient) population to spread that cost around to.

A gene therapy for a more prevalent disorder would not have the same problems, or at least not as badly.

[ekianjo]

No, its only negligible for small molecules. Making proteins/biologics is much more expensive, and this is where many new drugs are headed. Plus the investment required to design the process and build facilities is far from negligible either.

[maxander]

Relatively negligible. I don’t know what the precise price point for biologics are, but equivalent techniques are routinely used in research; a bit of Googling found a lab services page pricing a an AAV (the kind of vector used in Glybera) batch at the neighborhood of $1k [1]. Would be much more for a medical-grade preparation, I’m sure, but not an appreciable fraction of a million.

[1] https://sites.duke.edu/dvvc/services/adeno-associated-vector...