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The FDA has temporarily suspended Rocket Pharmaceuticals’ pivotal gene therapy study after a patient’s death, sending the company’s stock down 63% in early trading. Despite the setback, company leaders remain optimistic about the treatment’s future.
What was happening in the clinical trial?
RP-A501, the experimental therapy developed by Rocket, goes against Dannon's disease, a condition affecting the heart muscle. A 12-patient trial conducted by the New Jersey biotechnology company engaged in the use of brand-new AAV9 gene therapy in restoring normal heart function through direct gene delivery onto cardiomyocytes.
The trouble began when one patient developed capillary leak syndrome about a week after receiving the treatment. CEO Gaurav Shah explained that while the patient was initially recovering, a severe infection over the weekend led to his death.
“There were other medical complications and procedural complications in the week or so afterwards, and actually the patient was at that time stable and doing potentially well enough that we were cautiously optimistic over recovery, and the capillary leak was improving. Unfortunately, over the weekend ... he developed an acute systemic infection that accelerated his demise.” Shah said.
C3 inhibitor connection
Investigators believe the culprit may be a newly added drug called a C3 inhibitor. Rocket introduced the drug to prevent blood vessel complications that they had previously seen in patients. While it successfully prevented those original problems, both patients who received the C3 inhibitor developed capillary leak syndrome.
“We learned from the first case and intervened, so we didn’t see the same events in the second patient,” Shah noted, highlighting the company’s commitment to patient safety.
Moving forward despite challenges
The FDA imposed a clinical hold to fully investigate the incident before the patient died. Rocket is actively working with regulators to address safety concerns and modify its treatment protocols.
Shah stressed that the company’s other heart programs are unaffected and on schedule. With $318 million in funding, Rocket has extended its financial runway to 2027, ensuring continued research and development.
Rocket, which ended March with $318.2 million, is “prioritizing investments into its AAV platform while conducting an internal strategic review to optimize value for the rest of the pipeline,” Shah said.
Market Response and Expert Opinions
Investors did not seem to take the news quite positively initially, but analysts of William Blair gave cautiously optimistic views related to the potential, as the way would still be viable once these safety changes were made, noting that Rocket's other cardiovascular program uses different technology and will not face the same risks.
Then again, it is readily admitted that the setback-even if it can be temporarily accepted, rather than terminal for the promising gene therapy program.
(Input from various sources)
(Rehash/Muhammad Faisal Arshad/MSM)
