Casgevy vs. Lyfgenia: The Battle of the Sickle Cell Gene Therapies Has Begun

Boxing Gloves Battle

As a business journalism publication, BioSpace doesn’t get many news days like we had on Friday. Not just one but two major FDA approvals happened simultaneously for the first gene therapies to treat patients with sickle cell disease.

While the FDA’s decision regarding Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy was expected on Friday, the regulator’s verdict on bluebird bio’s Lyfgenia was a bit of a surprise as the PDUFA date was set for Dec. 20. Of course, who can blame the FDA for taking the opportunity to approve two milestone gene therapies for sickle cell disease on the same day. It made for a hell of an announcement from the agency—and early enough in the day to be included in BioSpace’s email newsletter (which I appreciated).

However, at the same time, I felt bad for bluebird having to share the limelight with Vertex and CRISPR, denying the company its own spotlight for an otherwise impressive one-time gene therapy—despite a black-box warning related to certain cases of blood cancer in patients treated with the drug.

Casgevy, on the other hand, got the regulatory go-ahead for what is the first CRISPR-based gene editing therapy for sickle cell disease. It’s a historic, groundbreaking distinction, not to take anything away from Lyfgenia—based on the Nobel Prize-winning CRISPR/Cas9 technology, which has its own potential off-target effects that are a sobering reminder that even good gene edits can go bad.

As I said, initially I felt bad that bluebird stood in the shadow of the day’s other big news—that is until I saw what the company is looking to charge for Lyfgenia. While Vertex and partner CRISPR said on Friday that Casgevy would be available at a U.S. list price of $2.2 million per patient, bluebird set the wholesale acquisition cost of Lyfgenia in the U.S. at $3.1 million. That’s where I believe bluebird made a strategic error—on pricing. It’s a mistake that has already cost the company, whose shares fell 40% Friday. Not surprisingly, in a research note, Evercore analysts gave the advantage to Casgevy due to its lack of a black-box label and lower price.

One company this week that was not afraid to shell out big bucks was AbbVie, which continued its buying spree by purchasing Cerevel Therapeutics for $8.7 billion. That news followed AbbVie’s Nov. 30 announcement that it was acquiring ImmunoGen for $10.1 billion for its antibody-drug conjugate Elahere, which was granted FDA accelerated approval last year, and a pipeline of promising next-generation ADCs—as the industry’s appetite for ADC technology appears to be insatiable.  

Wall Street analysts told Reuters that AbbVie’s bet on Cerevel is risky but smart, raising concerns that Cerevel’s pipeline overlaps too much with AbbVie products and making a Federal Trade Commission approval of the acquisition potentially problematic. We’ll see if the deal gets through the FTC!        

Greg Slabodkin is the News Editor at BioSpace. You can reach him at Follow him on LinkedIn.     

Clarification (December 10): This story has been updated to clarify that Lyfgenia is not a cell therapy but a lentivirus-delivered gene therapy.

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