{"id":61271,"date":"2026-03-25T15:34:12","date_gmt":"2026-03-25T07:34:12","guid":{"rendered":"https:\/\/flcube.com\/?p=61271"},"modified":"2026-03-25T15:34:13","modified_gmt":"2026-03-25T07:34:13","slug":"changchun-high-tech-wins-fda-orphan-drug-designation-for-gensci128-tp53-y220c-reactivator-targets-gastric-cancer","status":"publish","type":"post","link":"https:\/\/flcube.com\/?p=61271","title":{"rendered":"Changchun High-Tech Wins FDA Orphan Drug Designation for GenSci128 \u2013 TP53 Y220C Reactivator Targets Gastric Cancer"},"content":{"rendered":"\n

Changchun High & New Technology Industries (Group) Inc.<\/strong> (SHE: 000661<\/a>) announced that GenSci128<\/strong> received Orphan Drug Designation (ODD)<\/strong> from the U.S. Food and Drug Administration (FDA)<\/strong> for the treatment of gastric cancer<\/strong>. The selective small-molecule TP53 Y220C reactivator<\/strong>, already in clinical trials for pancreatic, ovarian, breast, and colorectal cancers<\/strong>, now holds dual ODD status<\/strong> (pancreatic + gastric), validating its pan-tumor potential<\/strong> for the TP53 Y220C mutation<\/strong> across multiple solid malignancies.<\/p>\n\n\n\n

Regulatory Milestone<\/h2>\n\n\n\n
Item<\/th>Detail<\/th><\/tr><\/thead>
Agency<\/strong><\/td>U.S. Food and Drug Administration (FDA)<\/td><\/tr>
Designation<\/strong><\/td>Orphan Drug Designation (ODD)<\/strong> \u2013 gastric cancer indication<\/td><\/tr>
Product<\/strong><\/td>GenSci128 (selective small-molecule TP53 Y220C reactivator)<\/td><\/tr>
Developer<\/strong><\/td>Changchun High & New Technology Industries (Group) Inc. (SHE: 000661)<\/td><\/tr>
Target Mutation<\/strong><\/td>TP53 Y220C<\/strong> \u2013 structural mutation in tumor suppressor p53<\/td><\/tr>
Previous ODD<\/strong><\/td>Pancreatic cancer<\/strong> (awarded earlier)<\/td><\/tr>
Clinical Status<\/strong><\/td>Approved for trials in China and U.S.<\/strong> for TP53 Y220C+ solid tumors<\/td><\/tr><\/tbody><\/table><\/figure>\n\n\n\n

Product Profile & Mechanism<\/h2>\n\n\n\n
Attribute<\/th>GenSci128 Specification<\/th><\/tr><\/thead>
Mechanism<\/strong><\/td>Selective small-molecule reactivator<\/strong> of mutant p53 protein<\/td><\/tr>
Target Mutation<\/strong><\/td>TP53 Y220C<\/strong> \u2013 conformational mutation that destabilizes p53 tumor suppressor function<\/td><\/tr>
Therapeutic Effect<\/strong><\/td>Restores wild-type p53 activity<\/strong> \u2192 cell cycle arrest, apoptosis induction in cancer cells<\/td><\/tr>
Tumor Spectrum<\/strong><\/td>Pan-solid tumor potential<\/strong> \u2013 gastric, pancreatic, ovarian, breast, colorectal cancers with Y220C mutation<\/td><\/tr>
Preclinical Data<\/strong><\/td>Favorable efficacy and safety<\/strong> profile demonstrated<\/td><\/tr><\/tbody><\/table><\/figure>\n\n\n\n

TP53 Y220C Mutation Context<\/h2>\n\n\n\n
Parameter<\/th>Clinical Significance<\/th><\/tr><\/thead>
Mutation Prevalence<\/strong><\/td>~1\u20132% of solid tumors<\/strong> (~10,000\u201315,000 annual cases globally across indications)<\/td><\/tr>
Current Standard of Care<\/strong><\/td>No approved targeted therapies; chemotherapy\/immunotherapy with limited efficacy in TP53-mutant tumors<\/td><\/tr>
GenSci128 Differentiation<\/strong><\/td>First-in-class<\/strong> selective Y220C reactivator; addresses “undruggable” p53<\/strong> with structure-based drug design<\/td><\/tr>
Orphan Drug Value<\/strong><\/td>7-year U.S. exclusivity<\/strong>, tax credits, waived FDA fees \u2013 critical for rare mutation subset<\/td><\/tr><\/tbody><\/table><\/figure>\n\n\n\n

Strategic Context & Development Pathway<\/h2>\n\n\n\n
Factor<\/th>Implication<\/th><\/tr><\/thead>
Dual ODD Strategy<\/strong><\/td>Gastric + pancreatic<\/strong> designations cover highest-unmet-need GI malignancies; supports basket trial design<\/strong> across Y220C+ solid tumors<\/td><\/tr>
China-U.S. Parallel Development<\/strong><\/td>NMPA and FDA IND approvals<\/strong> enable synchronized global trials; accelerates regulatory pathways<\/td><\/tr>
Companion Diagnostic<\/strong><\/td>TP53 Y220C testing required for patient selection; partnership with NGS providers critical for commercialization<\/td><\/tr>
Revenue Potential<\/strong><\/td>Niche but premium pricing<\/strong> (~$200,000\u2013300,000 annually) in orphan indications; peak sales $300\u2013500 million globally<\/strong> if approved in 2\u20133 tumor types<\/td><\/tr><\/tbody><\/table><\/figure>\n\n\n\n