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FDA suspends early trials of rare heart disease gene therapy RP-A501

 Last Update: 2021-05-21
 Source: Internet
 Author: User

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In the past year, the market value of Rocket Pharmaceuticals has surpassed established companies such as Bluebird Bio and UniQure.

But recently, Rocket Company disclosed that the US FDA has temporarily suspended an early trial of RP-A501.

It is worth mentioning that, out of safety or production concerns, gene therapy has encountered a series of setbacks in the US regulation.

Danon disease is a rare neuromuscular and cardiovascular disease.

Rocket's gene therapy RP-A501 is designed to restore heart function in patients.

As of now, no gene therapy has been proven to successfully treat cardiovascular disease.

Last year, Rocket did disclose that a patient receiving high-dose drugs had an immune response and must be treated with Alexion Pharmaceuticals’ C5 inhibitor Soliris.

Rocket CEO Gaurav Shah said the FDA's requirements are divided into "two parts.

Rocket believes that the company can solve this problem quickly.

However, investment bank Stifel analyst Dae Gon Ha pointed out, “Usually, the unpredictable and conservative FDA may cause longer than expected delays.

But Dae Gon Ha and investment bank SVB Leerink analyst Mani Foroohar agree that Rocket's future will not be affected.

Reference source: FDA unexpectedly grounds a gene therapy for a rare heart disease

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