As Vertex preps for exa-cel launch, exec touts 'very large' opportunity but tempers early expectations

After a long development road, the first CRISPR-based gene editing therapy recently cleared a FDA advisory committee and appears to be nearing its U.S. launch. Vertex Pharmaceuticals, awaiting the potential approval, is busy laying the groundwork for the med's debut.

Exa-cel is slated for an FDA decision in sickle cell disease by Dec. 8, and the agency will decide on a second indication in beta thalassemia by March 30, 2024. The drug represents a "multibillion-dollar" opportunity, Vertex Chief Operating Officer Stuart Arbuckle said on the company's third-quarter earnings call.

The company is “on track” with its launch preparations, which include setting up authorized treatment centers and working with payers. Arbuckle previously noted that about 50 treatment centers in the U.S. and 25 in Europe should suffice to reach approximately 32,000 eligible patients with severe forms of the diseases. That number includes 25,000 patients with sickle cell disease and 7,000 with beta thalassemia.

Vertex doesn't expect that exa-cel sales will begin pouring in right away upon a potential FDA approval. Instead, the company anticipates 2024 being a “foundational year" for the therapy as the first patients embark on their road to receiving the groundbreaking treatment. 

Arbuckle emphasized the lengthy timeline of the “exa-cel patient journey," which consists of three phases that each span several months. After phase 1, when doctors determine whether a patient is a fit for the treatment, the process of cell collection can begin. 

Next, the patient’s cells are sent to Vertex’s manufacturing facilities to undergo editing and quality control testing. The company finally captures revenue after a patients' receipt of the treatment, which requires a monthlong hospital stay.

“We’ve been asked a lot of questions about what the launch dynamics will look like for exa-cel, so we thought it was important to remind people of what the patient journey is,” Arbuckle said, adding that “whatever the journey to get there, we see this as being a very large commercial opportunity.”

Vertex is nearing this launch thanks to its 2015 research collaboration with CRISPR Therapeutics. Under that deal, Vertex is leading global development, manufacturing and commercialization of the therapy, splitting costs and profits 60/40 with its partner.

“We feel very optimistic about the exa-cel opportunity, and we’re looking forward to launching,” Arbuckle added.

Elsewhere in its business, Vertex generated third-quarter revenues of $2.48 billion, a 6% increase from last year's third quarter. The company hiked up its full-year revenue guidance to $9.85 billion, up from its previous projection of between $9.7 billion and $9.8 billion.