Introduction
Synribo, also known as omacetaxine mepesuccinate, is a chemotherapy agent approved for the treatment of adult patients with chronic or accelerated phase chronic myeloid leukemia (CML) who are resistant or intolerant to two or more tyrosine kinase inhibitors (TKIs). Here, we will delve into the clinical trials, market analysis, and projections for this drug.
Clinical Trials Overview
Efficacy and Safety
Clinical trials have demonstrated the efficacy of Synribo in patients with CML who have failed TKI therapy. Phase II clinical trials showed that omacetaxine mepesuccinate was active in patients with CML, including those with the BCR-ABL1T315I mutation, which is highly resistant to TKIs like imatinib, nilotinib, and dasatinib[3][5].
In these trials, Synribo was generally well-tolerated, although it was associated with significant adverse reactions. Myelosuppression, bleeding, and hyperglycemia were among the most common adverse effects. Myelosuppression occurred in all patients treated with Synribo, and it was often managed by delaying the next treatment cycle or reducing the days of treatment[1].
Ongoing and Future Trials
Besides its approved use, omacetaxine mepesuccinate is being studied in various other clinical trials. These include phase II and III trials assessing its activity alone or in combination with TKIs or other cytotoxic drugs in patients with CML resistant to TKI therapy. Additionally, phase I and II trials are ongoing for its use in acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS)[3].
Market Analysis
Market Landscape
Chronic myeloid leukemia (CML) is a rare myeloproliferative blood cancer characterized by the presence of the BCR-ABL fusion protein. The standard of care for CML involves TKIs, which have transformed most cases into a manageable, chronic condition. However, the emergence of resistance and intolerance to TKIs has created a need for alternative treatments like Synribo[2].
Sales Forecast
GlobalData's PharmaPoint report provides a detailed market analysis and sales forecast for Synribo. The report forecasts sales for Synribo in the top seven countries (the US, France, Germany, Italy, Spain, the UK, and Japan) from 2012 to 2022. The increasing prevalence of CML and the growing burden on healthcare systems due to the rising survival rates of CML patients are expected to drive the demand for Synribo[2].
Competitive Landscape
Synribo competes in a market dominated by TKIs, but its unique mechanism of action, which involves inhibiting protein synthesis without directly targeting BCR-ABL, sets it apart. This makes it a valuable option for patients who have failed TKI therapy. The competitive landscape includes other chemotherapy agents and emerging therapies, but Synribo's niche in treating TKI-resistant CML positions it as a significant player in this segment[2][3].
Projections and Future Outlook
Market Growth
The market for Synribo is expected to grow as more patients with CML develop resistance or intolerance to TKIs. The increasing awareness and diagnosis of CML, coupled with the expanding healthcare infrastructure, are likely to contribute to the growth of Synribo's market share. However, the market is also subject to the development of new therapies and the evolution of treatment guidelines, which could impact its long-term projections[2].
Production and Supply Chain
Historically, omacetaxine mepesuccinate has been derived from cephalotaxine, an alkaloid from the leaves of the Asian plum yew tree. However, recent research has paved the way for a new, chemically synthesized source of cephalotaxine, which could stabilize and potentially increase the supply of Synribo. This development is crucial for ensuring a consistent and reliable supply chain, which is essential for meeting the growing demand for the drug[4].
Key Takeaways
- Clinical Efficacy: Synribo has shown efficacy in treating CML patients resistant or intolerant to TKIs, particularly those with the BCR-ABL1T315I mutation.
- Adverse Reactions: Common adverse effects include myelosuppression, bleeding, and hyperglycemia, which are often managed by adjusting the treatment schedule.
- Market Forecast: Sales of Synribo are projected to grow driven by the increasing prevalence of CML and the need for alternative treatments to TKIs.
- Competitive Position: Synribo's unique mechanism of action positions it as a valuable treatment option in a market dominated by TKIs.
- Production Advancements: New chemical synthesis methods for cephalotaxine could improve the supply chain and ensure a consistent supply of Synribo.
FAQs
What is Synribo used for?
Synribo (omacetaxine mepesuccinate) is used for the treatment of adult patients with chronic or accelerated phase chronic myeloid leukemia (CML) who are resistant or intolerant to two or more tyrosine kinase inhibitors (TKIs)[3][5].
What are the common adverse reactions associated with Synribo?
Common adverse reactions include myelosuppression, bleeding, and hyperglycemia. Myelosuppression is particularly prevalent and often requires adjustments in the treatment schedule[1].
How does Synribo work?
Synribo inhibits protein synthesis by binding to the A-site cleft in the peptidyl-transferase center of the large ribosomal subunit. Unlike TKIs, it does not directly target the BCR-ABL protein[3][5].
What is the current market forecast for Synribo?
The market for Synribo is expected to grow from 2012 to 2022, driven by the increasing prevalence of CML and the need for alternative treatments to TKIs. Sales forecasts are available for the top seven countries, including the US, France, Germany, Italy, Spain, the UK, and Japan[2].
Is Synribo being studied for other indications?
Yes, omacetaxine mepesuccinate is being studied in clinical trials for other types of cancer, including acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS)[3].
How is Synribo produced?
Historically, Synribo has been derived from cephalotaxine, an alkaloid from the Asian plum yew tree. However, new research has enabled the chemical synthesis of cephalotaxine, which could improve the supply chain and ensure a consistent supply of the drug[4].
Sources
- SYN-40585 SYNRIBO Digital PI, PIl and IFU 5/2021 v3.indd
- Research and Markets: Synribo (Chronic Myeloid Leukemia) - Forecast and Market Analysis to 2022
- Aetna: Omacetaxine Mepesuccinate (Synribo) - Medical Clinical Policy
- Oregon State University Newsroom: Research paves way for new source for leukemia drug
- National Cancer Institute: Omacetaxine Mepesuccinate
