Gavreto (pralsetinib) Now Indicated for RET-Mutant Thyroid Cancer

Recently, the FDA expanded treatment indications for Gavreto (pralsetinib). According to MedPage Today, the drug was previously indicated for the treatment of patients with lung cancers with RET gene fusions. Now, this therapy is also approved in patients with RET-mutant medullary thyroid cancer and radioactive iodine (RI)-refractory RET fusion-positive thyroid cancer.


The decision to expand this indication stemmed from data from the Phase 1/2 ARROW clinical trial. Within this open-label trial, researchers evaluated Gavreto for patients aged 12+ with RET-altered tumors.

Gavreto is an orally-administered RET inhibitor. Patients should take 400mg 1x daily, and should not eat for 2 hours prior to and 1 hour following administration.

Within the ARROW study, 8 out of 9 patients with RI-refractory thyroid cancer responded to treatment. Additionally, the treatment response was sustained for 6 months or more. Alternately, 33 of 55 previously-treated patients with RET-mutant medullary thyroid cancer responded to treatment, with 79% having sustained responses; 19 of 29 previously untreated patients responded, with 84% having sustained responses.

Overall, Gavreto was safe and relatively well-tolerated. Some side effects did occur, which included:

  • Diarrhea or constipation
  • Fatigue
  • Muscle and joint pain
  • High blood pressure
  • Lowered calcium and hemoglobin levels

Thyroid Cancer

At the base of your throat sits your thyroid, a gland that creates hormones designed to regulate body temperature, weight, heart rate, and blood pressure. Thyroid cancer occurs when cancerous cells accumulate within the thyroid. While some cases of thyroid cancer are linked to genetics, other risk factors include being female and radiation exposure. Each year, an estimated 14 in every 100,000 people develop thyroid cancer. There are multiple types of thyroid cancer as well, such as papillary. Rare forms include follicular thyroid cancer, which starts in follicular cells and impacts those over 50; medullary thyroid cancer, which begins in the C cells and can metastasize throughout the body; and anapestic thyroid cancer, a fast-spreading form thought to develop from other forms of thyroid cancer. Symptoms of thyroid cancer include:

  • Neck and throat pain
  • Difficulty breathing and swallowing
  • Neck lumps
  • A chronic cough
  • Swollen lymph nodes
  • Hoarseness

Learn more about thyroid cancer.

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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