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Verastem Oncology Gains FDA Orphan Drug Status for Avutometinib and Defactinib in Pancreatic Cancer Treatment

NextEdge Admin

30 Jul 2024

2 min 21 sec

Verastem Oncology , a biopharmaceutical company focused on developing new medicines for patients, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to avutometinib, a RAF/MEK clamp, in combination with defactinib, a selective FAK inhibitor, for treating pancreatic cancer.

"At the ASCO 2024 Annual Meeting, we shared positive initial interim results from the ongoing RAMP 205 trial evaluating avutometinib and defactinib with standard chemotherapy for first-line metastatic pancreatic cancer," said Dan Paterson, president and CEO of Verastem Oncology. "The FDA's Orphan Drug Designation highlights the significant unmet need for pancreatic cancer treatments. We believe this combination could offer a new treatment approach for this challenging cancer. We look forward to presenting updated data from the RAMP 205 trial in early 2025."

During the ASCO meeting in June 2024, Verastem presented initial safety and efficacy results from the RAMP 205 trial. As of May 14, 2024, 41 patients were treated in four dose cohorts, with dose cohort 1 showing an 83% partial response rate. One dose-limiting toxicity was observed, and the dose level was cleared after additional enrollment. Of 26 patients with initial scans, 21 showed a reduction in target lesion diameters.

The FDA's Orphan Drug Designation supports treatments for diseases affecting fewer than 200,000 people in the U.S., offering benefits like tax credits, user fee exemptions, and seven years of market exclusivity upon approval. Read the press release here.