FDA approves two new therapies for sickle cell disease

December 19, 2023

People with sickle cell disease have horrific pain when their abnormal red blood cells deform and block arteries. They have higher risks of stroke and heart, lung and liver disease, and have an average life expectancy of 52.6, compared to life expectancy of 76.2 for all Americans. Sickle cell disease is common (about 100,000 in the US have the disease), and disproportionately affects Black people.

Two new treatments for sickle cell disease were approved by the Food and Drug Administration (FDA) last week. Casgevy, from Vertex and CRISPER, uses CRISPER technology to modify the patient’s own stem cells to produce fetal hemoglobin, which prevents sickle cell crisis. In clinical trials, 29 of 31 had freedom from severe sickle cell crisis for over a year. Casgevy’s price is $2.2 million. Lyfgenia, from Bluebird, uses a viral vector to modify a person’s stem cells to produce a variant of hemoglobin which prevents sickle cell crisis. Twenty-eight of 32 patients treated with Lyfgenia had freedom from severe sickle cell crisis for a year after treatment. The price for this drug is $3.1 million. The drugs are indicated for those over age 12 with moderate to severe sickle cell disease.

These treatments are complicated, and patients are subject to serious risks; stated cost of these drugs does not include associated medical care including long hospitalizations. Stem cells are harvested from the bone marrow, and patients are then treated with high doses of chemotherapy to kill all remaining stem cells in the bone marrow. The stem cells are treated and infused back into the patient, where they take up residence in the bone marrow and produce enough functional hemoglobin to protect the patient from sickle cell complications. This treatment requires a hospitalization that can last a month or more and has the same kind of dangers of infection associated with bone marrow transplants. The treatment likely decreases fertility and could come with risk of future cancers. The treatment is complex enough that it will only be offered in a few centers.

Source: Feurstein, StatNews  December 8, 2023 LINK

Implications for employers;

-  Cost of these treatments is quite high, although it’s projected that very few people will receive this treatment each year.  IPD Analytics projects that neither of these drugs will be a billion-dollar drug before 2027 or 2028.

-  Follow up so far is short, so it is possible that protection from these treatments will not persist for a patient’s lifetime.

- Effective treatment of sickle cell disease is great news for patients and could help prevent many cases of disability.

-  Employers may want to consider adding sickle-cell disease to their travel benefit.

-   Those who are being treated with these genetic therapies may want to freeze eggs or sperm to preserve future fertility. Employers may wish to review their plan documents, as many currently restrict coverage to those receiving therapy for cancer.

-   Many with sickle cell disease are currently undertreated; publicity around the new genetic treatments could decrease barriers to treatment with older therapies.

-    Outcomes based contracts for gene therapies in a commercial population are challenging to implement as employees may change insurers or employment during the monitoring period.

-Employers can review their stop loss policies to be sure that this treatment is not excluded.

Tomorrow: Chronic Fatigue Syndrome more common than we thought

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