FDA approves gene-editing therapy for sickle cell disease

The FDA approved gene-editing therapy Casgevy to treat sickle cell disease. Developed by Vertex Pharmaceuticals (VRTX) and CRISPR Therapeutics (CRSP), the one-time treatment harnesses gene editing advancements.

Vertex CEO Dr. Reshma Kewalramani says the approach involves extracting a patient's stem cells, correcting the defect in those cells using CRISPR gene editing tools, then re-infusing the edited cells into the patient - relieving painful symptoms.

Patients who undergo this $2.2 million procedure may eliminate painful sickle cell crises called VOC's. Kewalramani contrasts the cost against the $4-6 million lifetime price tag for conventional sickle cell management.

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Video transcript

ANJALEE KHEMLANI: Of course, we know that the drug is or the cure, rather, the treatment is the first to be approved. The FDA did approve both the Bluebirds as well as Vertex and CRISPR's gene-editing for sickle cell today. And we do have, of course, Dr. Reshma Kewalramani with us today.

Dr. Kewalramani, thank you for joining us. And I want to just ask you-- first of all, a momentous occasion we know so much has been built up to this point. The accolades have already been won. Talk to me about now this next chapter for the treatment and the long-term sort of follow-up that is needed to just see how it works.

RESHMA KEWALRAMANI: Well, thanks so much for having me on Anjalee. It is a huge day in science and medicine. And it's a particularly important day for patients with sickle cell disease. So what this medicine is Casgevy is the first CRISPR Cas9 gene-editing based therapy for sickle cell disease.

And what we do here is take the patient's own cells, their own stem cells, to our labs. We edit the cells. And we give them their cells back with the defect corrected. And what the studies have shown is that patients who undergo this therapy have no more of these painful crises called VOCs, veno-occlusive crises. Patients describe it as a Mack truck going up and down and up and down on their bodies. Patients describe this as worse than childbirth. And the studies show us that patients undergoing this therapy, it's a one-time therapy, no longer have these veno-occlusive crises to deal with.

It is an amazing technology. The seminal papers were written just in 2012. The Nobel Prize was awarded for this discovery in 2020. And here we are in 2023 with an FDA-approved product for sickle cell disease patients.

ANJALEE KHEMLANI: Absolutely. And we know, of course, this one time treatment requires multiple steps to get to the finish line, but the reward, of course, being not to have to worry about this disease. We know that the short-term look at it has brought on the approval for the FDA with, of course, on the label a look to the future. As we get more information on this new technology, how confident are you that the future holds no concerns of off gene-editing or any other concerns that you might have?

RESHMA KEWALRAMANI: As a medical doctor and as a physician scientist, we always have to be humble. We have to be aware. And we have to follow our patients over the long term. That being said, and we will be doing that, we will be following our patients over a many year period, this is a very precise gene edit. It's a durable gene edit.

And all of the data that we've seen to date, including long-term follow-up, as long as up to four years, tells us that this is a therapy that is incredibly effective. And we have seen no safety concerns in terms of this concept of off-target editing. We simply haven't seen any of that.

ANJALEE KHEMLANI: Well, that's wonderful. I know that you've already set the price tag for that a little bit lower than the other gene therapy that was out there. You've had these conversations with the government and payers. Talk to me about the value that you see there and why this price tag and what you see moving forward if any potential changes.

RESHMA KEWALRAMANI: The price tag for Casgevy is 2.2 million. And remember, that is for this one-time therapy. As we think about the price, it's always about the value that our medicines bring for patients. And in this disease, it is a devastating disease. It is a life-shortening disease. And when you think about the cost to the healthcare system over the lifetime of a patient with severe sickle cell disease, the kind of patient who would be eligible for our therapy, that number is between $4 and $6 million, not including the humanistic burden to the patient or their families.

And we've priced the medicine to be able to bring this incredible value, this one-time therapy, for potentially a lifetime of cure. And we want our medicine to be accessible to as many eligible patients as possible.