Korea-US Aim for 2nd Consecutive US Approval... FDA Issues 'Red Light' Citing "Least Effective Treatment"

FDA Advisory Committee Documents Released
Serious Concerns Raised About Efficacy and Safety

"If Approved, It Would Be the Least Effective Treatment"
Safety Issues: 85% Side Effects, 57% Dose Reduction

Stock Prices Also Down: Spectrum -37%, Hanmi -15%

[Asia Economy Reporter Lee Chun-hee] Following the FDA approval of the new drug for neutropenia treatment, 'Rolontis,' Hanmi Pharmaceutical's research and development (R&D) strategy aiming for back-to-back FDA new drug approvals has hit a red light with the anticipated approval of the non-small cell lung cancer treatment 'Poziotinib' within the year. Concerns are growing that approval will be difficult as the FDA raised strong doubts about Poziotinib's efficacy and safety through its advisory committee.

According to the FDA on the 21st, the FDA released related meeting materials ahead of the Oncology Drug Advisory Committee (ODAC) meeting scheduled to be held online from 9 a.m. on the 22nd (local time, 10 p.m. on the 22nd Korean time).

According to these materials, the advisory committee expressed strong concerns about Poziotinib's efficacy, stating, "If granted accelerated approval, this would be the least effective targeted therapy for lung cancer approved to date."

Portion of the briefing document by the U.S. Food and Drug Administration (FDA) mentioning the efficacy of Hanmi Pharmaceutical's non-small cell lung cancer treatment 'Poziotinib'

The advisory committee expressed concern that the objective response rate (ORR), which refers to the proportion of patients who show a measurable reduction in tumor size among all patients, is low at around 28%. The committee directly mentioned AstraZeneca-Daiichi Sankyo's antibody-drug conjugate (ADC) anticancer new drug 'Enhertu' (generic name: trastuzumab deruxtecan), which was recently approved domestically, with an ORR of 58%. They also pointed out that the median duration of response (mDoR) for Poziotinib is 5.1 months, which is lower compared to Enhertu's 8.7 months.

The advisory committee also viewed the safety profile of Poziotinib as concerning due to side effects. Among 368 patients receiving a daily 16 mg dose of Poziotinib, 85% experienced grade 3-4 adverse events, and 57% required dose reductions, indicating low drug tolerance in terms of safety profile. While the committee believed that such side effects might be alleviated in alternative dosing groups, they stated that additional analysis is needed regarding the efficacy at the current dose.

With this news, there is a growing outlook that FDA approval of Poziotinib, with a decision deadline set for November 24, will be difficult. Following Rolontis clearing the FDA hurdle on the 10th, expectations had been high for Poziotinib as well, but the stock prices of Hanmi Pharmaceutical and its U.S. partner Spectrum Pharmaceuticals have turned red. Spectrum's stock closed at $0.66 on the 21st (local time), down 37.5% from the previous closing price. Hanmi Pharmaceutical also traded in the 240,000 KRW range, down nearly 15% from the previous closing price after this news became known.

This content was produced with the assistance of AI translation services.