Bristol-Myers Squibb Company — Onureg EMA EPAR public assessment report (EMEA/H/C/004761, 2021)
Bristol-Myers Squibb Company · 2021 · EMA EPAR public assessment report · company profile →
Report summary
This EMA assessment report details the regulatory approval of Onureg (oral azacitidine) for maintenance therapy in adult acute myeloid leukemia (AML) patients in remission who are not candidates for hematopoietic stem cell transplantation. The report covers the drug's chemical, pharmaceutical, and biological characteristics, including its manufacturing, stability, and non-clinical studies. It outlines azacitidine's pharmacokinetics, noting rapid absorption and low oral bioavailability, and its mechanism of action involving DNA hypomethylation. Clinical trials, including a pivotal Phase 3 study, demonstrated significant improvements in overall survival (9.9 months) and relapse-free survival (5.4 months) compared to placebo. Despite gastrointestinal toxicities, the CHMP concluded a favorable benefit-risk balance, recommending marketing authorization.
Key findings — cited to the page
| NOEC (Daphnia sp. Reproduction Test) | 730 µg/L | · p.29 |
| Approved indication for Vidaza (azacitidine) | treatment of adult patients who are not eligible for hematopoietic stem cell transplant (HSCT) with: intermediate-2 and high-risk MDS according to the International Prognostic Scoring System (IPSS), chronic myelomonocytic leukaemia (CMML) with 10 -29% marrow blasts without myeloproliferative disorder, Acute myeloid leukemia (AML) with 20 -30% blasts and multi -lineage dysplasia, according to WHO classification, AML with >30% marrow blasts according to the World Health Organization (WHO) classification. | Vidaza · EU · p.11 |
| median relapse-free survival | 5.4 months | oral azacitidine · p.98 |
| approved indication | maintenance therapy in adult patients with acute myeloid leukaemia (AML) who achieved complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following induction therapy with or without consolidation treatment and who are not candidates for, including those who choose not to proceed to, hematopoietic stem cell transplantation (HSCT) | Onureg · p.99 |
| CHMP positive opinion date | 22 April 2021 | Onureg · p.7 |
| Proposed indication | maintenance therapy in adult patients with acute myeloid leukaemia (AML) who achieved complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following induction therapy with or without consolidation treatment and who are not candidates for, including those who choose not to proceed to, hematopoietic stem cell transplantation (HSCT) | Onureg · p.8 |
| Marketing Authorisation application submission date | 30 April 2020 | Onureg · p.6 |
| median OS improvement | 9.9 months | oral azacitidine · p.98 |
| gastrointestinal toxicities incidence | 92 % | oral azacitidine · p.98 |
| marketing authorisation recommendation | recommended | Onureg · p.99 |
| Proposed starting dose | 300 mg orally, once daily | Onureg · p.12 |
| shelf-life | 36 months | · p.17 |
Source: Bristol-Myers Squibb Company ↗ · License: EMA - reuse permitted with acknowledgement. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Oncology library →