Rocket’s Gene Therapy Wins FDA Greenlight, Clearing Way for Future Products

 

While the accelerated approval unlocks only a small market opportunity for Rocket Pharmaceuticals, it will give the biotech a chance to prepare for future product launches, according to analysts at Jefferies.

Bouncing back from a 2024 rejection tied to manufacturing concerns, Rocket Pharmaceuticals’ gene therapy has now secured accelerated approval for the rare immune disease leukocyte adhesion deficiency-I—a decision that analysts say could help prime the company for future launches.

Leukocyte adhesion deficiency-I (LAD-I) is a relatively small market opportunity, analysts at Jefferies told investors in a note Thursday evening, forecasting below $50 million in sales. The disease, which compromises the immune system resulting in more frequent and severe infections, affects around 1 per 1 million people worldwide.

Rocket’s newly approved gene therapy, to be marketed as Kresladi, is likely to enter a sparse market, “perhaps treating <10 patients per year due to the rarity of the disease,” Jefferies added. The true value of Thursday’s approval, the analysts continued, is in how it will prepare the biotech for its future products.

“Kresladi’s US launch provides RCKT with experience in payor negotiations and commercial scaling, which can be leveraged for future launches,” they said. The approval also derisks Rocket’s gene therapy platform and manufacturing network, potentially making subsequent regulatory reviews run smoother.

Rocket is trading at $5.11 apiece in premarket hours on Friday, a 9% increase from Thursday’s closing price.

The FDA’s approval on Thursday was supported by pivotal Phase 1/2 data that in October 2023 returned a 100% overall response rate at 12 months. The most common side effects included anemia, low platelet counts, upper respiratory infections and liver enzyme elevations, the agency said in its announcement of the approval.

Kresladi was cleared under the FDA’s accelerated pathway, with full approval hinging on the validation of its clinical benefit in a confirmatory trial.

With Kresladi’s approval, investor attention now shifts to Rocket’s lead asset RP-A501, an investigational gene therapy for Danon disease, which Jefferies on Thursday said could unlock a $500 million opportunity for the biotech.

Last May, the FDA placed RP-A501 under clinical hold after a patient died of severe complications following infusion with the therapy. The patient in question developed capillary leak syndrome—fluids leaking out of blood vessels and into the surrounding tissues—which resulted in a drop in blood pressure.

The regulator lifted the hold in August, allowing Rocket to resume dosing in a mid-stage study with a recalibrated dose of RP-A501. Jefferies expects the biotech to restart the Phase 2 Danon study in the first half of 2026, according to its Thursday note.

https://www.biospace.com/fda/rockets-gene-therapy-wins-fda-greenlight-clearing-way-for-future-products