FDA Approval could happen after Phase 1b11 Sep 2023 20:00
Very interesting paper
ORR of >15% for regulatory approval of an anticancer drug is 76% and rises to 89% if the ORR is >30%
No wonder AS stated 50% chance with confidence…
For comparison, the ORRs of approved oncology drugs as monotherapy are often >20%20. Of note, an analysis of drug approvals has revealed that an ORR of >30% with a monotherapy is likely to lead to accelerated approval by the FDA20. Indeed, the positive predictive power of an ORR of >15% for regulatory approval of an anticancer drug is 76% and rises to 89% if the ORR is >30%
Phase I trials as valid therapeutic options.
The design adaptations made in phase I trials in the past few years (in aspects including dosing63, biomarkers8,9,17,30,56,70, safety14, survival71 and responses18) have helped them to become valid therapeutic options. We propose that researchers can anticipate therapeutic responses in contemporary phase I trials and therefore these trials can be considered to have therapeutic intent.
RNS Number : 3595D
Avacta Group PLC
21 June 2023
Dr Alastair Smith, Chief Executive of Avacta Group plc commented:
"The continued positive safety profile of AVA6000 at these dose levels compared with standard doxorubicin is remarkable. We are seeing a significant reduction in the incidence and severity of all doxorubicin side effects. Analysis of the tumour biopsies to date also confirms that enough doxorubicin is being released in the tumour to have a THERAPEUTIC effect.
If even higher doses of AVA6000 are tolerated then this may make a significant difference to the outcomes for patients in the upcoming efficacy study.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6868302/
Accordingly, in the past few years, the FDA has approved investigational drugs on the basis of results from phase I studies. Examples include ceritinib, which was approved for patients with ALK-rearranged non-small-cell lung cancer (NSCLC) on the basis of an overall response rate (ORR) of 58% reported in a phase I study;7,8 and the immune-checkpoint inhibitor (ICI) pembrolizumab, which was approved for patients with melanoma on the basis of a 38% ORR observed in a dose-expansion cohort of a first-in-human phase I trial.
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