Gene therapy for inherited retinal diseases receives orphan drug designation
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Key takeaways:
- HG004 received orphan drug designation for the treatment of inherited retinal diseases caused by RPE65 mutations.
- HuidaGene is planning a multinational clinical trial.
The FDA granted orphan drug designation for HG004, a gene replacement therapy for the treatment of inherited retinal diseases caused by RPE65 mutations, HuidaGene Therapeutics announced in a press release.
The designation offers the company “the potential for certain benefits, including tax credits for clinical development, exemptions for certain FDA application fees, 7 years of post-approval market exclusivity, and assistance in the drug development process,” the release said.
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In addition, drug development and registration action in the U.S. will be accelerated, with HG004 eligible for incentives such as FDA support for clinical studies.
The FDA cleared the investigational new drug application for the therapy in January, allowing HuidaGene to move forward with a planned multinational clinical trial.
“Receiving ODD is an important milestone as we are advancing our HG004 gene replacement therapy program to clinical trial designed to provide safe, durable and high-quality treatment to children and adults suffering from RPE65 mutation-associated inherited retinal diseases,” Xuan Yao, PhD, co-founder and CEO of HuidaGene, said in the release. “It also underscores the importance of bringing this novel therapy to patients with severe visual impairment or blindness, and strongly motivates us to expedite the clinical development of HG004.”
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