FDA grants Orphan Drug Designation for Avutometinib and Defactinib for the Treatment of Pancreatic Cancer

“At the ASCO 2024 Annual Meeting, we reported positive initial interim results from the ongoing RAMP 205 trial evaluating avutometinib and defactinib in combination with standard of care chemotherapy in first-line metastatic pancreatic cancer,” said Dan Paterson, president and chief executive officer of Verastem Oncology. “The FDA Orphan Drug Designation for the combination of avutometinib and defactinib for the treatment of pancreatic cancer recognizes the substantial unmet treatment need for patients with pancreatic cancer. We believe avutometinib and defactinib in combination with standard of care has an opportunity to provide a different approach in treating this challenging cancer. We look forward to reporting updated data from across dose cohorts in the ongoing RAMP 205 trial in the first quarter of 2025.”

Share:

More News

“These data underscore gotistobart’s potential to redefine treatment for patients with hard-to-treat squamous NSCLC whose disease has progressed after initial therapy and who face limited options,” said Prof. Özlem Türeci, M.D., Co-Founder and Chief Medical Officer at BioNTech. “In second- and later lines of treatment, the clinical relevance of novel

“The collaboration with Ratio represents an exciting opportunity to explore the potential of novel radiopharmaceutical approaches designed to address the complexity and heterogeneity of cancer,” said Ben Hickey, President of RayzeBio. “The combination of our respective technologies and complementary development expertise creates a strong foundation for generating a novel differentiated

“We believe these results mark a potentially important step forward in the treatment of EGFR exon 20 insertion mutation-positive non-small cell lung cancer,” said Fabio Benedetti, MD, Global Chief Medical Officer, Taiho Pharmaceutical. “To address the unmet medical needs of patients and their families, we will continue working closely with

“Recent clinical data validated KAT6 as a targetable mechanism in the treatment of HR+/HER2- breast cancer, including those with actionable mutations,” stated Charles Morris, MBChB, MRCP, Chief Medical Officer of Prelude. “Dual KAT6A/B inhibitors, however, demonstrated overlapping toxicities with current backbone therapies that may limit the utility in earlier lines