FDA Halts Rocket Pharmaceuticals’ Danon Disease Trial

Rocket Pharmaceuticals ( (RCKT) ) has issued an update.

On May 27, 2025, Rocket Pharmaceuticals announced that the FDA placed a clinical hold on its Phase 2 trial of RP-A501 for Danon disease following a serious adverse event that led to a patient’s death. The company is conducting a root cause analysis and remains in dialogue with the FDA, prioritizing patient safety and aiming to resume the trial as soon as possible. Rocket has sufficient funds to continue operations into 2027 and is focusing on optimizing its AAV platform.

The most recent analyst rating on (RCKT) stock is a Buy with a $29.00 price target. To see the full list of analyst forecasts on Rocket Pharmaceuticals stock, see the RCKT Stock Forecast page.

Spark’s Take on RCKT Stock

According to Spark, TipRanks’ AI Analyst, RCKT is a Underperform.

Rocket Pharmaceuticals’ overall score reflects its position as a biotechnology firm in the development phase, with significant financial risks due to its lack of revenue and reliance on external financing. Technical indicators suggest potential volatility, while the negative P/E ratio highlights current valuation challenges. The company’s strong equity position is a positive factor, but ongoing cash burn remains a critical concern.

To see Spark’s full report on RCKT stock, click here.

More about Rocket Pharmaceuticals

Rocket Pharmaceuticals, Inc. is a late-stage biotechnology company that develops genetic therapies for rare diseases. The company focuses on creating transformative options for complex disorders using a multi-platform approach, including adeno-associated viral vector-based therapies for cardiovascular conditions and lentiviral vector-based therapies for hematological disorders.

Average Trading Volume: 2,929,750

Technical Sentiment Signal: Sell

Current Market Cap: $675.5M

Learn more about RCKT stock on TipRanks’ Stock Analysis page.