Vertex Gears Up for exa-cel Launch, Acknowledges Vast Opportunity with Cautious Outlook

Vertex Readies for exa-cel Launch, Cautiously Eyes Enormous Opportunity

After a prolonged journey in development, the first CRISPR-based gene editing therapy has recently received approval from an FDA advisory committee and is edging closer to its launch in the United States. Vertex Pharmaceuticals, eagerly awaiting potential approval, is diligently preparing for the therapy’s introduction.

Exa-cel, scheduled for an FDA decision on its use in sickle cell disease by December 8, will also undergo a second indication evaluation in beta thalassemia by March 30, 2024. Vertex’s Chief Operating Officer, Stuart Arbuckle, emphasized the enormous potential of the drug, describing it as a “multibillion-dollar” opportunity during the company’s third-quarter earnings call.

The company is actively setting the stage for Exa-cel’s launch, which encompasses establishing authorized treatment centers and collaborating with payers. Arbuckle previously highlighted the plan to have approximately 50 treatment centers in the US and 25 in Europe to serve around 32,000 eligible patients suffering from severe forms of sickle cell disease and beta thalassemia. This patient population consists of 25,000 individuals with sickle cell disease and 7,000 with beta thalassemia.

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“Vertex has delivered another strong quarter across the business. We remain relentless in our commitment to reach more patients with our cystic fibrosis medicines, while preparing for the potential launch of exa-cel in multiple geographies. Our R&D pipeline continues to make remarkable progress and we have a milestone-rich period coming up, with multiple major, near-term milestones, including completion of the Phase 3 pivotal trials for the vanzacaftor triple in cystic fibrosis and VX-548 in acute pain, as well as the Phase 2 VX-548 study data read-out in diabetic peripheral neuropathy.”

– Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex

Vertex does not anticipate an immediate surge in Exa-cel sales following a potential FDA approval. Instead, the company envisions 2024 as a “foundational year” for the therapy, with the first patients embarking on their journey to receive this groundbreaking treatment.

Arbuckle underlined the extensive timeline of the “Exa-cel patient journey,” which unfolds in three phases, each spanning several months. The process commences with phase 1, during which doctors assess a patient’s suitability for the treatment. Afterward, the patient’s cells are collected and sent to Vertex’s manufacturing facilities for editing and quality control testing. Revenue is realized after patients receive the treatment, which necessitates a month-long hospital stay.

Also Read: Vertex And Lonza Partner For New Cell Therapy Manufacturing Facility, Creating Job Opportunities

Vertex’s imminent launch of Exa-cel is the result of its 2015 research collaboration with CRISPR Therapeutics. Under this partnership, Vertex leads global development, manufacturing, and commercialization of the therapy, sharing costs and profits in a 60/40 ratio with its partner.

In other aspects of its business, Vertex reported third-quarter revenues of $2.48 billion, marking a 6% increase from the same period in the previous year. The company has raised its full-year revenue guidance to $9.85 billion, surpassing its earlier projection of between $9.7 billion and $9.8 billion. This financial success underscores Vertex’s commitment to advancing innovative therapies and achieving significant growth.

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