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Lucie Ellis-Taitt: Why Hemophilia Gene Therapies Are Failing?

Lucie Ellis-Taitt/X

Aug 19, 2026, 13:51

Lucie Ellis-Taitt: Why Hemophilia Gene Therapies Are Failing?

Lucie Ellis-Taitt, Executive Editor – In Vivo at Pharma Intelligence, now Citeline, Editor in Chief, Health and Medtech at Norstella, shared a post on LinkedIn:

”Two of the three hemophilia gene therapies approved between 2022 and 2024 have already left the market: Roctavian withdrawn, Beqvez discontinued. Only Hemgenix remains, at narrow scale.

These were products that worked clinically. One infusion cut annualized bleeding rates by 96 percent over seven years in one study.

So what happend?

The commercial model, not the science. Manufacturers priced one-time therapies at 2.9 million dollar-3.5 million dollar on the promise of lifetime savings that no long-term data could yet support.

Meanwhile the comparator they meant to displace-modern prophylaxis-kept getting better, giving payers acceptable control at a cost they already understand and can manage. Asked to trade a known quantity for an expensive, irreversible unknown, many patients and payers chose to wait.

The uncomfortable takeaway for every program that follows: the variables that sank hemophilia (a ‘good enough’ comparator, high eligibility attrition, unproven durability, payers unready for multimillion-dollar single payments) aren’t bugs specific to this indication. They’re structural features of the modality.

Until reimbursement frameworks can absorb the cost and uncertainty of single-administration therapies, regulatory approval will remain the easier half of the problem.”

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