Vertex said it’s making strong progress bringing its CRISPR Therapeutics-partnered gene therapy to patients after a landmark approval for sickle cell disease in December.

Chief operating officer Stuart Arbuckle said Monday that more than 25 treatment sites for the therapy, called Casgevy, have been activated worldwide. As of mid-April, “five patients already had cells collected” — a first major step in the treatment process. The company declined to say how far along the patients are in the process or whether more have started since then.

“This is excellent progress given the short timeframe since approval, and the complexity and length of the patient journey,” Arbuckle told investors.

The news comes as bluebird bio announced it had completed the first cell collection for its rival sickle cell product, Lyfgenia, which was also approved in December. Casgevy won a label expansion the following month to treat people with transfusion-dependent beta thalassemia.

Launching the high-cost, high-complexity therapies is a challenge with few parallels in the drug industry. Both companies have worked to prepare treatment sites around the US that can take patients through the complex process of collecting cells, genetically altering them and then infusing them back into the body.

Vertex set Casgevy’s US list price at $2.2 million, while bluebird launched Lyfgenia at $3.1 million. Both companies have touted their success establishing coverage with insurers, and Vertex said Monday it has commercial contracts or policies to cover 200 million lives in the US. The company also has policies or contracts under negotiation with 18 states, and every state in the US has confirmed its intent to cover Casgevy on a case-by-case basis, according to Arbuckle.

Outside the US, Arbuckle said he is “particularly pleased” with progress in the Middle East, where the opportunity could outweigh the US given a high prevalence of sickle cell disease. Vertex is now working with local health professionals to increase its number of treatment sites in the region.

“Our work indicates that the eligible 12-plus sickle cell disease and beta thalassemia population in [Saudi Arabia] and Bahrain that we could serve is in excess of 23,000 patients — a potentially larger opportunity than even the US,” Arbuckle said.

Vertex cited Casgevy as one of the key reasons it’s confident in its 2024 revenue guidance of $10.55 billion to $10.75 billion, up from $9.87 billion in 2023.


Non-opioid pain submission

Also on Monday, Vertex reiterated that it’s on track to finish a rolling FDA submission this quarter for its non-opioid pain candidate, called suzetrigine, in moderate-to-severe acute pain. While Phase 2 results earlier this year suggest the drug can treat pain in certain settings without the risk of addiction, it will be up against cheap, widely available opioids.

Arbuckle said Monday that Vertex is “already engaging with key decision-makers across the formulary and access landscape,” and expects coverage decisions to come throughout the first year of suzetrigine’s potential launch.

If approved, Vertex plans to focus initially on a specific set of acute pain conditions and procedures, targeting orthopedic professionals, general and plastic surgeons, emergency department physicians, anesthesiologists and pain medicine specialists.

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