Almost A Year Ago -- We Said This Day Was Upon Us... Sickle Cell Is Cured, And FDA Approved -- But At ~$2.2 Million Per Patient? Who Will / How To Pay?!

We first covered this, just about 11 months ago -- and it is excellent, ground-breaking news, for Vertex, CRISPR Therapeutics and blue bird bio. But it ultimately may not mean too terribly much -- for people of limited means, worldwide, with the life-long disease.

Even so, this is a watershed moment, after centuries of early-deaths, and more recently, life-long disability -- largely inside the communities of Black African origin, in the US, UK, Europe and elsewhere. . . in addition to Africa itself. This is truly remarkable news. We could see sickle-cell relegated to the dust bin of history the way polio was.

The real problem though, lies -- as with all genetic therapies -- in the daunting cost to be "cured": best estimates put the cost of cure, including a year or more out of commission. . . at over $2.2 million a million dollars per patient. In the US, that may make marginal sense, because the costs of a life-time dealing with the effects of (uncured) sickle-cell, greatly exceed the therapy's likely costs.

But in the developing world, there is not likely to be any way to incent any provider to deliver it, unlike the very cheap COVID-19 vaccines, by comparison. So it is likely that sickle-cell will continue to be scourge, outside North America and Western Europe. Still -- this is an amazing achievement for biotech / life sciences. Here's the latest:

. . .The U.S. Food and Drug Administration (FDA) on Friday approved two gene therapies for sickle cell disease, making one of them the first treatment in the United States based on the Nobel Prize-winning CRISPR gene editing technology.

Casgevy, developed by partners Vertex Pharmaceuticals, and CRISPR Therapeutics, and bluebird bio's Lyfgenia were approved for people aged 12 years and older. . . .

It is, in fact. . . a functional. . . cure.

Now you know. And as very long term readers recall, we'd covered the Hep C cure wars -- Vertex v. SGP/MRK, for almost 15 years -- right here. [See left-hand legacy graphic.]

Be excellent to one another; for we -- as a society -- now face some very important choices: should a sickle cell sufferer (largely in communities of color) be any less advantaged, in the public health reimbursement scheme. . . than say a sufferer with solid organ tumors / cancers? I submit that we need to confront this, directly -- as Keytruda rumbles ever onward, at ~$22 billion a year. Onward.

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