Characteristics of Drugs from Non-Global Companies for Hematologic Malignancies and Impact on Global Regulatory Approval
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View abstract on PubMed
Summary
This summary is machine-generated.Biotech start-ups are increasingly developing drugs for blood cancers. However, drugs from these non-global companies face higher rejection rates in the EU and Japan after US approval.
Area Of Science
- Hematology
- Oncology
- Drug Development
- Regulatory Affairs
Background
- The rise of non-global companies, including biotech start-ups, in drug development is notable.
- Understanding the characteristics and global regulatory impact of drugs from these companies is crucial.
- Focus on new molecular entities (NMEs) for hematologic malignancies approved in the US.
Purpose Of The Study
- To analyze NMEs for hematologic malignancies approved in the US between 2011 and 2022.
- To compare NMEs developed by non-global versus global companies.
- To assess the impact of NME origin on global regulatory approval, specifically in the EU and Japan.
Main Methods
- Utilized a public database to identify NMEs approved for hematologic malignancies in the US (January 2011 - December 2022).
- Categorized NMEs into 'non-global' and 'global' company submission groups.
- Analyzed US accelerated approval rates, post-approval trial status, and EU/Japan approval rates within two years of US approval.
Main Results
- Out of 48 NMEs, 19 (40%) were from non-global companies, predominantly US-based (68%).
- Non-global NMEs showed higher US accelerated approval rates (63%), with only 50% having confirmatory trials.
- Non-global NMEs had significantly higher 2-year unapproval rates in the EU (56% vs. 21%) and Japan (94% vs. 64%) compared to global NMEs.
Conclusions
- Many NMEs from non-global companies receive US accelerated approval based on early-phase trials (Phase I/II).
- NMEs developed by non-global companies demonstrate a higher likelihood of regulatory non-approval in the EU and Japan post-US approval.
- Further investigation into the long-term efficacy and regulatory pathways for NMEs from non-global entities is warranted.
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