Azacitidine is a Small Molecule owned by Bristol-Myers Squibb, and is involved in 138 clinical trials, of which 92 were completed, 41 are ongoing, and 5 are planned.

Azacitidine is DNA (cytosine-5-)-methyltransferase 1(DNMT1) inhibitor. Inhibition of DNMT1 leads to hypomethylation of DNA. The cytotoxic effects of azacitidine may result from multiple mechanisms, including inhibition of DNA, RNA and protein synthesis, incorporation into RNA and DNA, and activation of DNA damage pathways. Non-proliferating cells are relatively insensitive to azacitidine. Incorporation of azacitidine into DNA results in the inactivation of DNA methyltransferases, leading to hypomethylation of DNA. DNA hypomethylation of aberrantly methylated genes involved in normal cell cycle regulation, differentiation and death pathways may result in gene re-expression and restoration of cancer-suppressing functions to cancer cells.

The revenue for Azacitidine is expected to reach a total of $372m through 2033. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. View the complete picture with the Azacitidine NPV Report.

Azacitidine was originated by Pharmacia & Upjohn and is currently owned by Bristol-Myers Squibb. Nippon Shinyaku is the other company associated in development or marketing of Azacitidine.

Azacitidine Overview

Azacitidine (Vidaza, Azafuridine, Celazadine) is a pyrimidine analog acts as an anti-anemic, anti-neoplastic agent. It is formulated as lyophilized powder for suspension and solution for intravenous, and subcutaneous route of administration. It is indicated for the treatment of myelodysplastic syndromes (MDS). It is indicated used for all 5 FAB (French-American-British) subtypes of myelodysplastic syndromes (MDS) which include refractory anemia (RA), refractory anemia with ringed sideroblasts (RARS), refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEB-T) and chronic myelomonocytic leukemia (CMMoL). Vidaza is also indicated for the treatment of adult patients aged 65 years or older with acute myeloid leukemia (AML) who are not eligible for hematopoietic stem cell transplantation (HSCT) and  indicated for the treatment of adult patients who are not eligible for hematopoietic stem cell transplantation (HSCT) with intermediate-2 and high-risk myelodysplastic syndromes (MDS) according to the International Prognostic Scoring System (IPSS), chronic myelomonocytic leukemia (CMML) with 10-29% marrow blasts without myeloproliferative disorder and acute myeloid leukemia (AML) with 20-30% blasts and multi-lineage dysplasia. Vidaza is indicated for the treatment of pediatric patients aged one month and older with newly diagnosed JMML.

Injectable azacitidine is under development for newly diagnosed advanced myelodysplastic syndrome, myeloproliferative neoplasm, osteosarcoma, multiple myeloma and relapsed or refractory non-Hodgkin lymphoma, Hodgkin lymphoma, adult T-cell leukemia/lymphoma, extranodal NK-/T-cell lymphoma, nasal type, enteropathy-associated T-cell lymphoma, monomorphic epitheliotropic intestinal T-cell lymphoma, hepatosplenic T-cell lymphoma, subcutaneous panniculitis-like t-cell lymphoma, primary cutaneous anaplastic large cell lymphoma, primary cutaneous T-cell lymphoma, primary cutaneous CD8+ T-cell lymphoma, transformed mycosis fungoides, primary cutaneous CD4+ small/medium T-cell lymphoproliferative disorder, angioimmunoblastic T-cell lymphoma, follicular T-cell lymphoma, nodal peripheral T-cell lymphoma with TFH phenotype, ALK- and ALK+ anaplastic large-cell lymphoma.

It was also under development for treatment of as a second line therapy for transitional cell cancer (urothelial cell cancer), epithelial ovarian cancer, fallopian tube cancer, peritoneal cancer pancreatic cancer, nasopharyngeal carcinoma, cervical carcinoma, anal carcinoma and merkel cell carcinoma (MCC).

Nippon Shinyaku Overview

Nippon Shinyaku develops, manufactures, and sells ethical pharmaceuticals and functional foods. The company offers a wide range of products including drugs for pain, inflammation, and allergies; urological diseases; hematologic malignancies; gastrointestinal disorders; cardiovascular and metabolic diseases among others. Nippon Shinyaku also provides functional food ingredients including health food ingredients, preservatives, spices and condiments, and protein preparations. Its functional food products find application in meat processing, fish processing, dairy product, prepared food, confectionery and bakery, and beverage, among others. The company operates business through a network of offices and research laboratories located in Japan, China, the UK and the US. Nippon Shinyaku is headquartered in Minami-ku, Kyoto, Japan.

The company reported revenues of (Yen) JPY137,484 million for the fiscal year ended March 2022 (FY2022), an increase of 12.8% over FY2021. In FY2022, the company’s operating margin was 20.6%, compared to an operating margin of 21.4% in FY2021. In FY2022, the company recorded a net margin of 16.8%, compared to a net margin of 17% in FY2021. The company reported revenues of JPY35,517 million for the second quarter ended September 2022, a decrease of 0.3% over the previous quarter.

Quick View – Azacitidine

Report Segments
  • Innovator (NME)
Drug Name
  • Azacitidine
Administration Pathway
  • Intravenous
  • Subcutaneous
Therapeutic Areas
  • Infectious Disease
  • Oncology
Key Companies
Highest Development Stage
  • Marketed

GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

To create this model, GlobalData takes into account factors including patent law, known and projected regulatory approval processes, cash flows, potential applicable patients, drug margins, company expenses, and pricing estimates. Combining these data points with GlobalData’s world class analysis creates high value models that companies can use to help in evaluation processes for each drug or company.

The rNPV method integrates the probability of a drug reaching a clinical stage into the cash flow at that time, which provides a more accurate rNPV, as it considers the probability that the drug never makes it through the clinical pathway to commercialization. GlobalData’s rNPV model uses proprietary likelihood of approval (LoA)and phase transition success rate(PTSR) data for the indication in the highest development stage, which can be found on GlobalData’s Pharmaceutical Intelligence Center.