NEW FDA VIDEO ROUNDUP: In the latest edition of CMO Confidential, Harpreet Singh, MD, discusses the notable regulatory milestones in oncology and rare disease treatments from October.
The FDA's approval of Zolbetuximab (Vyloy) for gastric and gastroesophageal junction adenocarcinoma marked a step forward in targeted therapies, particularly for patients with CLDN18.2 expression. This approval highlights the importance of biomarker-driven treatments in oncology and has implications for the future.
The FDA released an update on the Rare Disease Innovation Hub aimed at streamlining the development of treatments for rare diseases. See why this is good news for innovators in the rare disease space.
In hematology, Asciminib (Scemblix) received accelerated approval for newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia. The substantial improvements in response rates compared to the investigator-selected tyrosine kinase inhibitors (IS-TKIs) is very noteworthy.
Watch now and follow Harpreet Singh, MD for future insights.
What does the latest news from FDA mean? I'm Harpreet Singh, Chief Medical Officer, former FDA Oncology Director, here to help innovators bring breakthrough therapies to patients. Let's dive into a few notable approvals for the month of October 1st UP. We have a very exciting breakthrough. On October 18th, FDA's Division of Oncology 3 approved Zoba Tucson AMAB with chemotherapy for first line gastric and GE junction adenocarcinoma for patients. Expressing claudin 18 this monoclonal antibody binds to clot in 18 two and is the first FDA approved therapy for this particular biomarker. FDA's decision was supported by results from 2 trials, GLOW and Spotlight. Collectively, these trials enrolled over 1000 patients who were randomized to the monoclonal antibodies Albertus amab plus chemotherapy versus chemotherapy alone. Both trials showed improvement in both progression free survival and overall survival for patients and frontline gastric and GE junction tumor when Zoltek Samad was added to chemotherapy. The approval of Zoladex Sabab for patients with frontline gastric or GE junction adenocarcinoma does represent an improvement over standard of care available therapy. Both the Glow and Spotlight trials did demonstrate a roughly 2 to three month improvement and both progression free survival and overall survival. Moving on to the hematologic space, on October 29th the FDA granted accelerated approval to a Cinema B which is marketed as Symbolix and developed by Novartis. The efficacy of Acidemia B was evaluated in the ASC for first trial, which randomized over 400 patients to either the novel agent or investigator's choice of oral tyrosine kinase inhibitor therapy. This is interesting because FDA Oncology has been recommending investigators choice as a control arm where appropriate. The primary endpoint is also of interest as major molecular response rate at 48 weeks. Which demonstrated a substantial difference between arms of 68% in the academic arm versus 49% and the investigators choice TK IR. What's interesting about this approval is that it was an accelerated approval but on a randomized trial. Most accelerated approvals in oncology are based on single arm trials and on overall response rate and duration of response. However, the use of MMR or major molecular. Response rate formed the basis for this early or accelerated approval. Novartis will need to follow this randomized trial out for time to event end points such as progression free survival and overall survival to submit to FDA to further confirm benefit and convert this accelerated approval in frontline patients to a regular or traditional approval. Of note, this agent was granted breakthrough therapy designation was granted priority review. And this review was conducted under FDA's project Orbis in collaboration with Health Canada and Switzerland Swissmedic. I will also note that Novartis was issued a post marketing commitment to gather more data on this agent and underrepresented populations. And this can include claims data and or real world data to fulfill this commitment. There are some great news coming out of FDA for biotech looking to develop innovative therapies in the rare disease space, the FDA. Has recently launched a Rare Disease Innovation Hub in an effort to advance treatment options for patients with rare diseases. This initiative is a growing recognition of the challenges which are unique to research and development in rare populations. By creating a centralized resource, the Hub will facilitate communication between drug developers and FDA, creating a more collaborative and expedited review process. The Rare Disease Innovation Hub will be Co led by leaders from both Seizure and Sieber, the Center for Biologic and Drug Evaluation and Research is respectively, as well as strategic leaders from each of the centers rare disease innovation programs given the success of the Oncology Center of Excellence. I'm optimistic that creating a single point of contact in this rare disease innovation hub will allow for new avenues for expedited drug development and enhanced collaboration between FDA and biotech looking to develop their assets in rare indications. Stay tuned for more insights from CMO Confidential.
