FDA Grants Orphan Drug Designation to New Treatments for Pancreatic Cancer and Ewing Sarcoma
Introduction
1. Orphan Drug Designation (ODD) is a regulatory status granted by the U.S. Food and Drug Administration (FDA) to drugs or biologics intended to treat rare diseases or conditions that affect fewer than 200,000 people in the United States.
2. The FDA grants ODD to incentivize the development of drugs for rare diseases, as it can be challenging for pharmaceutical companies to recoup their investment in developing such drugs due to the small patient populations.
3. Two new treatments for pancreatic cancer and Ewing sarcoma have recently received ODD. This article will discuss the details of these treatments and the implications of receiving ODD status.
Pancreatic Cancer Treatment
4. The FDA granted ODD to a new drug, AMG 119, for the treatment of pancreatic cancer. Pancreatic cancer is a rare disease with a five-year survival rate of less than 10 percent. AMG 119 is a biologic drug that targets a specific receptor on cancer cells and activates the patient's immune system to fight the cancer.
5. The ODD status for AMG 119 provides the drug's manufacturer, Amgen, with several benefits, including tax credits for clinical testing expenses, exemption from FDA application fees, and seven years of market exclusivity if the drug is approved for commercial use.
6. The ODD status for AMG 119 is based on preclinical data and early-stage clinical trials. Further studies are needed to evaluate the drug's safety and efficacy fully.
Ewing Sarcoma Treatment
7. The FDA also granted ODD to a new drug, BDTX-189, for the treatment of Ewing sarcoma. Ewing sarcoma is a rare bone cancer that primarily affects children and young adults.
8. BDTX-189 is a small molecule drug that targets a specific protein that is overexpressed in Ewing sarcoma cells. The drug has shown promising results in preclinical studies, including inducing tumor regression and increasing survival in animal models.
9. The ODD status for BDTX-189 provides its manufacturer, Beigene, with the same benefits as AMG 119, including tax credits, FDA application fee exemption, and market exclusivity.
10. Beigene plans to initiate clinical trials of BDTX-189 in the near future to evaluate the drug's safety and efficacy in human patients.
Conclusion
11. The FDA's recent grant of ODD to AMG 119 for pancreatic cancer and BDTX-189 for Ewing sarcoma highlights the agency's commitment to incentivizing the development of drugs for rare diseases. ODD status provides pharmaceutical companies with significant benefits, which can help offset the high costs of developing drugs for small patient populations.
12. However, ODD status does not guarantee FDA approval of a drug or its commercial success. Pharmaceutical companies must still conduct extensive clinical trials and demonstrate safety and efficacy to gain FDA approval and market their drugs successfully.
13. Further research and development are needed to fully evaluate the safety and effectiveness of AMG 119 and BDTX-189, but the FDA's grant of ODD status is a positive step forward in the search for new treatments for these rare diseases.
References:
1. U.S. Food and Drug Administration. Orphan Drug Designations and Approvals. https://www.fda.gov/industry/developing-products-rare-diseases-conditions/orphan-drug-designations-and-approvals. Accessed April 14, 2023.
2. Healio. FDA news: Treatments for pancreatic cancer, Ewing sarcoma receive orphan drug designation. https://www.healio.com/news/hematology-oncology/20230414/fda-news-treatments-for-pancreatic-cancer-ewing-sarcoma-receive-orphan-drug-designation. Accessed April 14, 2023.
3. Amgen. Amgen's AMG 119 Granted Orphan Drug Designation by U.S. FDA for Treatment of Pancreatic Cancer. https://www.amgen.com/newsroom/press-releases/2023/04/amgens-amg-119-granted-orphan-drug-designation-by-us-fda-for-treatment-of-pancreatic-cancer. Accessed April 14, 2023.
4. Beigene. Beigene's BDTX-189 Receives Orphan Drug Designation from U.S. FDA for Treatment of Ewing Sarcoma. https://ir.beigene.com/news-releases/news-release-details/beigenes-bdtx-189-receives-orphan-drug-designation-us-fda. Accessed April 14, 2023.
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