Rocket Pharmaceuticals’ clinical setback, sees analyst slash forecasts

Rocket Pharmaceuticals (NASDAQ: RCKT) has come under regulatory scrutiny after a patient death prompted the US FDA in May to halt a key clinical trial of its flagship gene therapy. The pause cast doubts over the future of RP-A501, an experimental treatment for Danon disease — a rare and often fatal genetic condition that damages heart and skeletal muscle.

Prior to the clinical setback, analysts had projected a strong commercial runway for RP-A501. Risk-adjusted revenue linked to Danon disease was estimated at $54 million in 2026, rising to $150 million in 2027, and grow steadily to $1.2 billion by 2035. The therapy was anticipated to anchor Rocket’s expansion, with total company revenues forecast to climb from $104 million in 2026 to $2 billion by the end of the decade.

But those estimates have been sharply revised downward. Visible Alpha consensus shows analysts now projects RP-A501 sales at $31 million in 2026 — 43% below earlier forecasts — and $112 million in 2027, down 26%. Long-term projections for 2035 have been cut by a third, to $822 million. Total company revenue forecasts have also been reduced in tandem, reflecting more conservative expectations for both regulatory approval and market adoption.

The mid-stage trial suspension has left Rocket at a pivotal juncture, as it faces mounting operational uncertainty, regulatory investigations, and potential litigation over allegations it failed to disclose known risks.