FDA grants orphan drug designation to OTX-2002 for hepatocellular carcinoma
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The FDA granted orphan drug designation to OTX-2002 for the treatment of hepatocellular carcinoma, according to the agent’s manufacturer.
OTX-2002 (Omega Therapeutics) is a messenger RNA therapeutic delivered through lipid nanoparticles. It is designed to downregulate c-MYC pre-transcriptionally via epigenetic modulation and potentially overcome MYC autoregulation, according to a Omega Therapeutics-issued press release. The MYC oncogene has been linked to aggressive disease in as many as 70% of patients with HCC, according to the release.
"HCC is a devastating illness that often develops resistance to current standard-of-care therapeutics,” Mahesh Karande, president and CEO of Omega Therapeutics, said in the release. “The FDA's decision to grant orphan drug designation for OTX-2002 underscores the need for novel therapies to address HCC and the potential of epigenomic programming to transform the treatment landscape.”
The phase 1/phase 2 MYCHELANGELO I trial is designed to assess the safety, tolerability and preliminary antitumor activity of OTX-2022 as single-agent therapy and combined with standard therapies for patients with relapsed or refractory HCC and other solid tumors associated with the MYC oncogene.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.
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