(Reuters) -Rocket Pharmaceuticals said on Friday it has withdrawn its application for U.S. approval of its experimental gene therapy for a rare inherited blood disorder. The company said the move was driven by business strategy rather than concerns about the safety or efficacy of the therapy RP-L102. The gene therapy was being tested in patients […]
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Rocket Pharmaceuticals withdraws US application for blood disorder gene therapy
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(Reuters) -Rocket Pharmaceuticals said on Friday it has withdrawn its application for U.S. approval of its experimental gene therapy for a rare inherited blood disorder.
The company said the move was driven by business strategy rather than concerns about the safety or efficacy of the therapy RP-L102.
The gene therapy was being tested in patients with Fanconi anemia, a disorder in which impaired bone marrow reduces blood cell production.
The condition affects about 1 in 136,000 newborns worldwide, according to National Institutes of Health data.
The withdrawal preserves the company’s ability to engage with regulators at a later date if it is able to find an external partner to advance the program, Rocket said.
The company added that it had stopped new internal spending on the therapy and also withdrew its application for European approval in July.
The U.S. Food and Drug Administration had halted a mid-stage study testing Rocket’s gene therapy, RP-A501, for a genetic disorder called Danon disease after a patient died from a rare complication. The agency lifted the clinical hold in August.
(Reporting by Padmanabhan Ananthan in Bengaluru; Editing by Vijay Kishore)