FDA Grants Orphan Drug Designation to PTX-252 for Treatment of Acute Myeloid Leukemia


PTX-252 is a novel molecular drug designed to increase cancer cells' sensitivity to chemotherapy in the treatment of acute myeloid leukemia.

The FDA granted orphan drug designation to PTX-252 for the treatment of acute myeloid leukemia (AML), according to a press release from Pleco Therapeutics BV. PTX-252 is a novel molecular drug designed to increase cancer cells' sensitivity to chemotherapy, according to the company.1

White blood cells with nucleus and granule | Image Credit: stockdevil - stock.adobe.com

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"We are very excited to receive this designation for PTX-252 from the FDA," Ivo Timmermans, CEO of Pleco Therapeutics, said in the press release. "This milestone underscores our commitment to innovative therapies for rare diseases and it brings hope to AML patients who have limited treatment options. Our team is dedicated to advancing this therapy through clinical development as swiftly as possible."1

According to the American Cancer Society, AML starts in the bone marrow and can quickly move to the blood, spreading through the lymph nodes, liver, spleen, central nervous system, and testicles. Most often, cells that become cancerous would normally turn into white blood cells, according to the organizations. However, it is possible that it develops in other blood cells.2

Pleco therapeutics collaborated with Hyloris Pharmaceuticals SA for the development of PTX-252. Orphan drug designation is granted to drugs that treat or prevent life-threatening rare diseases. This can include drugs with either no currently approved treatments or drugs that provide a significant clinical benefit when compared with existing treatments, according to the press release.1

"Securing orphan drug designation for PTX-252 stands as a testament to our unwavering commitment to advancing the frontiers of scientific discovery within the repurposing space,” Stijn Van Rompay CEO of Hyloris, said in the press release.1

In 2023, the estimated number of cases for AML was 20,380, which accounted for approximately 1% of all new cancer cases. From 2013 to 2019, the 5-year relative survival rate was 31.7%. The estimated number of deaths in 2023 was 11,310, which accounted for approximately 1.9% of all cancer deaths, according to the National Institutes of Health. The median age of diagnosis was 69 years but ranged from 65 to 74 years old. The median age of death was 73 years with an age range of 75 to 84 years old.The organization also said that the responses to treatment can vary by patient.3

Currently, there are different treatment options for those with AML, with 2 phases: a remission induction therapy and post-remission therapy. Additionally, patients with AML will also receive care for adverse events of treatment, which could include treating infections to treating myelosuppression, according to the National Cancer Institute.4

Therapy options currently include chemotherapy, radiation therapy, chemotherapy with stem cell transplants, and targeted therapy. For untreated AML, the standard of care depends on the subtype of the disease and can include combination chemotherapy, combination chemotherapy with midostaurin for those with an AML mutation in the FLT3 gene, combination chemotherapy with gemtuzumab ozogamicin, an antibody drug conjugate, intrathecal chemotherapy for CNS treatment, and supportive care, according to the National Cancer Institute.4


  1. Pleco Therapeutics receives FDA Orphan Drug Designation. News release. Pleco Therapeutics. January 16, 2024. Accessed January 16, 2024. https://www.plecotherapeutics.com/news/39/86/Pleco-Therapeutics-receives-FDA-Orphan-Drug-Designation.html
  2. American Cancer Society. What is Acute Myeloid Leukemia (AML)?. August 21, 2018. Accessed January 16, 2024. https://www.cancer.org/cancer/types/acute-myeloid-leukemia/about/what-is-aml.html
  3. SEER Cancer Stat Facts: Acute Myeloid Leukemia. National Cancer Institute. Bethesda, MD. Accessed January 16, 2024. https://seer.cancer.gov/statfacts/html/amyl.html
  4. PDQ Adult Treatment Editorial Board. PDQ Acute Myeloid Leukemia Treatment. Bethesda, MD: National Cancer Institute. Updated September 8, 2023. Accessed January 16, 2024. https://www.cancer.gov/types/leukemia/patient/adult-aml-treatment-pdq
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