Gene therapy's slow rollout offers a reality check

Why it matters: The slow rollout illustrates the potential disconnect between cutting-edge science and patient benefit.

• There's plenty of optimism that gene therapies' launch kinks will get worked out.
• But it serves as a reality check as the U.S. faces a pipeline chock-full of novel drugs and devices that the health system isn't used to incorporating — and patients may not immediately be comfortable with using.

"They've just begun to enter the health care system, which was designed decades ago. So changing the status quo takes time, and this is just the first chapter in a long story," said Stephen Majors, head of communications at the Alliance for Regenerative Medicine.

Driving the news: Second-quarter earnings calls over the last few weeks have shed light on the commercial progress — or lack thereof — made by some gene therapies, including new treatments for sickle cell disease and hemophilia.

• Bluebird Bio, which has three gene therapies on the market, reported that 27 patients total have begun treatment with their products so far this calendar year, and it anticipates 85 starts by the end of the year.
• Of those 27, 19 initiated treatment with Zynteglo, a beta thalassemia treatment. Four patients each began treatment with Skysona, which treats cerebral adrenoleukodystrophy, and Lyfgenia, one of two sickle cell gene therapies the FDA approved late last year.

The other newly approved sickle cell treatment, Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy — which is also approved to treat beta thalassemia — has begun the treatment process with 20 patients.

• BioMarin Pharmaceutical announced it was narrowing its efforts to the U.S., Germany and Italy after only treating five patients — three in the U.S. and two in Italy — with Roctavian, its hemophilia A gene therapy, during Q2. That equated to $7 million in revenue.
• Roche reported revenues of only about $12 million during the first half of 2024 for Luxturna, which was approved in 2017 to treat an inherited form of vision loss.

By the numbers: CVS has previously estimated that 8,000 U.S. adults would be candidates for Roctavian and 42,500 patients with sickle cell would be eligible for Vertex's Casgevy and Bluebird's Lyfgenia.

• In other words, the drugs' approval didn't exactly open the floodgates for patients to immediately begin receiving them.

• "This ramp is in retrospect, I think, easy to understand. Of course, everyone was hoping there would be thousands and thousands of sickle cell patients being treated in the first six months," said Mark Trusheim, strategic director of Tufts' New Drug Development Paradigms initiative.
• "But I think if you looked at the facilities that were qualified to do this and how many doctors and nurses they have, I think that was probably an unreasonable expectation."

What's happening: Receiving many of the gene therapies is a very long and intensive process, which involves harvesting a patients' cells, modifying them in a lab and then reinfusing them back into the patient.

• Even before that can happen, pharma companies have to negotiate payment rates with insurers and establish relationships with hospitals capable of performing the collection and infusion processes.
• At launch, "you may only have a dozen hospitals across the country that are ready from both a skill standpoint and an infrastructure standpoint to deliver the therapies," Trusheim said.
• Some of the companies touted insurance coverage agreements or the expansion of treatment centers as markers of progress on their earnings announcements.

And, of course, patients have to be willing to go through all of this.

• "This is gene editing. This is gene therapy. This is something that patients are understandably going to have questions about," Majors said.
• A reluctance to shake up their existing treatment may be an especially big factor among hemophilia patients, Stat reported earlier this year.