Rocket Pharma Trial: Patient Death & Safety Changes
- Rocket Pharmaceuticals' gene therapy trial for Danon disease has been paused by the Food adn Drug Administration (FDA) after a patient's death.
- The patient, who had received the pre-treatment regimen in early May, developed capillary leak syndrome about a week after the gene therapy infusion.
- According to CEO Gaurav Shah, the patient initially appeared stable but later developed an acute systemic infection.
Rocket Pharmaceuticals’ Trial Halted After Patient Death; Gene Therapy role Probed
Rocket Pharmaceuticals’ gene therapy trial for Danon disease has been paused by the Food adn Drug Administration (FDA) after a patient’s death. The clinical trial was evaluating RP-A501, a therapy designed to treat the rare inherited metabolic disorder that weakens heart muscle. The company is investigating whether a complement inhibitor, administered to improve safety, played a role in the adverse event.
The patient, who had received the pre-treatment regimen in early May, developed capillary leak syndrome about a week after the gene therapy infusion. This condition, characterized by fluid leaking from capillaries, can cause a life-threatening drop in blood pressure. Rocket Pharmaceuticals voluntarily paused dosing and informed the FDA, which then imposed a clinical hold.
According to CEO Gaurav Shah, the patient initially appeared stable but later developed an acute systemic infection. A second patient who received the C3 inhibitor also showed signs of capillary leak syndrome but is now improving after a reduced course of immune-suppression.
Shah emphasized that the C3 inhibitor was specific to the Danon gene therapy program and does not effect other rocket programs. The company is conducting a extensive analysis to determine the root cause of the adverse events.
“We are considering that as one option, one thought, one idea for root cause,” Shah said. “We’re doing a comprehensive root cause analysis pretty neutrally objectively and this is one idea, so current focus, but just one idea.”
The trial aimed to enroll 12 patients, but the clinical hold has cast uncertainty on the timeline. As of the first quarter, rocket’s cash position was $318.2 million. The company is now reducing expenditures to extend its financial runway into 2027.
Following the announcement, Rocket’s stock price plummeted more than 60%. Leerink Partners analyst Mani Foroohar noted that the setback undermines the company’s credibility, especially after previous regulatory challenges with other gene therapies.
What’s next
Regulatory scrutiny could lead to changes in the Danon program’s study design, potentially requiring increased enrollment and longer follow-up periods to better assess the therapy’s risk/benefit profile.