Chinese Academy of

Cytarabine is a small-molecule antineoplastic agent approved globally for acute myeloid leukemia (AML) and other hematologic malignancies. The drug is administered via injection and belongs to the therapeutic class of antineoplastic and immunomodulating agents (L01). Cytarabine has an established regulatory footprint: approved in the United States under multiple generic applications (NDA016793, NDA021041, NDA209401 and numerous ANDAs), approved in Australia (PBS codes 4357H, 7227J, first listed December 2011), and withdrawn from the European market (DepoCyte, EMEA/H/C/000317, authorized June 2017, now withdrawn).

The program profiled here—IIT2016007 (Chidamide)—is sponsored by Xiyuan Hospital of China Academy of Chinese Medical Sciences and is currently in Phase 3 development for AML, with an active status and latest milestone dated 27 March 2026. The internal code designation suggests a potential combination or investigator-initiated trial (IIT) design. Three clinical trial identifiers are associated: NCT03031249, NCT03031262, and NCT06744504. The mechanism of action and specific target remain undisclosed. Peak sales projections, consensus positioning, and expected next milestones have not been disclosed. This profile reflects cytarabine's role as a foundational AML therapy with multiple approved formulations globally and ongoing clinical investigation in China.

Acute myeloid leukemia remains a serious hematologic malignancy with significant unmet medical need, particularly in elderly and relapsed/refractory populations. Cytarabine is a cornerstone therapy in AML treatment regimens, often combined with anthracyclines or used in intensive chemotherapy protocols. The drug's long clinical history and multiple approved formulations underscore its established role in standard-of-care AML management.

The Phase 3 trial activity in China (NCT03031262, NCT06744504) suggests investigation of cytarabine in combination regimens or novel formulations tailored to Chinese patient populations. This development pathway is commercially significant given the large AML patient population in China and the region's growing emphasis on oncology drug development and approval pathways.

Competitive positioning: Cytarabine competes with approved therapies including Vyxeos Liposomal (Jazz Pharmaceuticals, a liposomal formulation of cytarabine and daunorubicin), as well as targeted agents such as FLT3 inhibitors, IDH inhibitors, and venetoclax-based combinations that have expanded AML treatment options in recent years. The established generic landscape in the US (13+ approved applications) indicates mature market competition and price pressure, while ongoing clinical trials in China may support market expansion or regulatory approval of novel formulations or combinations.

Drug Class: Antineoplastic and immunomodulating agent (ATC L01).

Modality: Small molecule.

Route of Administration: Injection (intravenous or intrathecal).

Mechanism of Action: Not yet disclosed in this profile.

Target: Not yet disclosed in this profile.

Related Therapies: Vyxeos Liposomal (liposomal formulation of cytarabine + daunorubicin, approved); conventional cytarabine formulations (multiple generic suppliers); other AML therapies including venetoclax, FLT3 inhibitors, and IDH inhibitors.

First Approval: Cytarabine has been approved in the United States since at least the 1980s–1990s (NDA016793, NDA021041 predate modern ANDA submissions). Australian approval first listed December 2011. European approval of DepoCyte (liposomal cytarabine) authorized 26 June 2017, subsequently withdrawn.

Patent Status: Not yet disclosed; cytarabine is a well-established molecule with expired composition-of-matter patents; generic formulations dominate the US market.

United States (FDA): Cytarabine is approved via NDA016793 (original), NDA021041, and NDA209401 (liposomal formulation, Vyxeos). Thirteen ANDA approvals for generic cytarabine are documented (ANDA071248, ANDA071249, ANDA071471, ANDA071472, ANDA071868, ANDA072168, ANDA072945, ANDA074245, ANDA075206, ANDA075383, ANDA076512, ANDA200914, ANDA200915, ANDA200916, ANDA201784, ANDA205696, ANDA206189, ANDA206190, ANDA208485, ANDA211937, ANDA211938), indicating a mature generic market.

European Union (EMA): DepoCyte (liposomal cytarabine, EMEA/H/C/000317) was authorized on 26 June 2017 under Marketing Authorization Holder Pacira Limited; this product has since been withdrawn from the EU market.

Australia (TGA): Cytarabine is approved and listed on the Pharmaceutical Benefits Scheme (PBS) with codes 4357H and 7227J; first listed 1 December 2011; sponsor Pfizer Australia Pty Ltd.

China (NMPA): Cytarabine is in clinical trials in China. Associated NCT IDs include NCT03031262, NCT03356080, NCT04121819, and NCT07301138, indicating ongoing investigator-initiated or sponsor-led development activity. Regulatory approval status in China not yet disclosed.

Strategic Implications: The Phase 3 program (IIT2016007) sponsored by Xiyuan Hospital of China Academy of Chinese Medical Sciences suggests a China-focused development strategy for cytarabine, potentially investigating novel formulations, dosing schedules, or combination regimens tailored to Chinese AML populations. The latest milestone date of 27 March 2026 indicates active trial conduct; however, the absence of disclosed milestone details, expected next milestones, and peak sales projections limits visibility into program trajectory and commercial expectations.

Competitive Implications: Cytarabine's established generic market in the US (13+ approved applications) and approved status in Australia and EU (withdrawn) reflect a mature, commoditized therapeutic. The Phase 3 activity in China may support market expansion, regulatory approval of novel formulations (e.g., liposomal or sustained-release variants), or combination strategies that differentiate from generic cytarabine. Vyxeos Liposomal (Jazz Pharmaceuticals) remains the primary value-added competitor in the liposomal cytarabine space.

Future Catalysts: Completion and publication of Phase 3 trial results (NCT03031262, NCT06744504) in AML; potential NMPA approval or label expansion in China; regulatory decisions on novel formulations or combinations; competitive responses from other AML therapy sponsors.

Expected Milestones: Phase 3 completion and data readout (expected timing not yet disclosed); regulatory submission to NMPA (if applicable); approval decision (timing not yet disclosed).