DSP-5336 Granted Orphan Drug Designation for AML

Currently, clinical-stage oncology company Sumitomo Pharma Oncology, Inc. is working to develop DSP-5336 for patients with acute myeloid leukemia (AML). The company even began recruiting for a Phase 1/2 study which will explore DSP-5336 for those with AML or acute lymphocytic leukemia (ALL). Patients may enroll whether they do or do not have MLL gene arrangements or NPM1 genetic mutations. 

Even more excitingly, shares the company in a recent news release, DSP-5336 has earned Orphan Drug designation from the FDA. This status is given to drugs or biologics intended to treat, diagnose, or prevent rare conditions. In the U.S., a rare condition is one which affects fewer than 200,000 individuals. Drug developers – such as Sumitomo Pharma Oncology – also receive benefits with this status. These include fee waivers, tax credits, and seven years of market exclusivity upon approval. 

So what exactly is DSP-5336? This investigational small molecule treatment inhibits the ability for menin and mixed-lineage leukemia (MLL) protein to bind together. NPM1 and MLL mutations or rearrangements help drive cancer progression. Menin, on the other hand, plays a role in cell growth, strength, and stability, as well as some other bodily processes. Because of this, when menin binds with MLL, it can cause cancer growth. By stopping this process, DSP-5336 aims to prevent the cancer from spreading, and address and treat it at its source. 

What is Acute Myeloid Leukemia (AML)? 

Also known as acute myelogenous leukemia, AML is a blood and bone marrow cancer. This cancer often progresses rapidly, making early diagnosis and treatment crucial. In patients with AML, the bone marrow creates abnormal myeloblasts, platelets, and/or red blood cells. These abnormal cells then crowd out healthy cells, causing a host of problems. Risk factors include smoking cigarettes, being male, being older in age, chemical or radiation exposure, previous cancer treatment, or genetic disorders. Symptoms can (but do not always) include:

  • Fever
  • Easy bruising and bleeding
  • Lethargy and fatigue
  • Appetite loss
  • Unintended weight loss
  • Enlarged spleen and/or liver
  • Bone pain
  • Pallor (extremely pale skin)
  • Frequent or recurrent infections
  • Unusual bleeding (ex: frequent nosebleeds; bleeding from the gums)
  • Shortness of breath
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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