FDA Approves Inqovi for MDS and CMML

 

In a recent press release, the U.S. Food and Drug Administration (FDA) announced their approval of Inqovi for patients with myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML). The drug therapy, which consists of decitabine and cedazuridine, offers a less invasive and more accessible form of treatment than prior options.

Inqovi Approval

Developed by Astex Pharmaceuticals, Inqovi (decitabine and cedazuridine) presents a unique option for patients with MDS and CMML. In the past, treatment required visiting a hospital or doctor’s office for intravenous injections. Now, patients can take Inqovi in the comfort of their own homes. For the treatment, patients take one tablet daily for 5 consecutive days within a 28-day cycle.

Inqovi was approved following positive clinical trial results in which patients no longer required transfusions over a 2-month period. Although the therapy is promising, there are some associated side effects, including:

  • Mouth sores
  • Joint and muscle pain
  • Constipation
  • Fatigue
  • Hemorrhage
  • Fever
  • Low white blood cell count
  • Nausea
  • Fetal harm

In addition to its treatment approval, Inqovi was also granted Priority Review and Orphan Drug designation.

MDS and CMML

MDS

Myelodysplastic syndromes (MDS) are progressive conditions which prevent healthy levels of platelets, white blood cells, and red blood cells from growing in bone marrow. Instead of maturing and leaving the bone marrow, the cells either die or fail to move. Life expectancy varies based on the severity of the condition. In half of all cases, MDS progresses to acute myeloid leukemia (AML). Generally, MDS affects males more than females and occurs in patients over the age of 60.

Altogether, there are five subsets of MDS: refractory anemia, refractory anemia with sideroblasts, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia (CMML). While many patients experience no symptoms, those who do may experience:

  • Anemia
  • Neutropenia (low white blood cell count)
  • Fatigue
  • Heart palpitations
  • Pale skin
  • Shortness of breath and/or chest pain
  • Frequent lung, sinus, skin, and urinary tract infections
  • Easy bruising and bleeding
  • Low platelet counts

Learn more about MDS here.

CMML

Chronic myelomonocytic leukemia (CMML) is a blood cancer which begins in bone marrow. However, it may then spread into the blood. CMML is characterized by too many monocytes (a type of white blood cell), spleen enlargement, and abnormal cell growth. Around 15-30% of patients with CMML will progress to AML.

Symptoms include:

  • Anemia
  • Fatigue
  • Excess monocytes (when blood tested)
  • Frequent infections
  • Severe nosebleeds
  • Fever
  • Unintended weight loss
  • Loss of appetite

Learn more about CMML here.

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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