According to a story from Medscape, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recently issued a positive recommendation for the drug osilodrostat. This is an experimental treatment that is being developed as a treatment for Cushing’s syndrome. If the drug is cleared for use in the EU, it will mark the first ever approval for the medication.
About Cushing’s Syndrome
Cushing’s syndrome is a medical condition that appears as the result of prolonged cortisol exposure. Cushing’s syndrome is most commonly caused by the use of medications such as corticosteroids, but it can also be caused by abnormalities that cause the body to release more cortisol than usual, such as a tumor affecting the pituitary or adrenal glands. Cushing’s syndrome is also associated with diabetes, which is a common comorbidity. The syndrome can cause a variety of symptoms that can become more serious over time including cerebral atrophy, hypercholesterolemia, rapid weight gain, baldness, mood instability, depression, hirsutism, sexual dysfunction, muscle and bone weakness, menstrual abnormalities, osteoporosis, diabetes, hypertension, sleep problems, immune system suppression, and memory problems. Treatment may include the cessation of drug use when they are the cause, surgery to remove tumors or affected glands, or certain drugs that inhibit cortisol synthesis. To learn more about Cushing’s syndrome, click here.
New Therapies Needed
There aren’t many treatments available for this disease. Surgical removal of the pituitary gland is the most common treatment and the drug pasireotide can be useful for some patients, but at the end of the day there is a significant portion of patients that fail to see remission with these approaches. Therefore, the approval of a new treatment option would be welcome news for Cushing’s syndrome patients.
Typically, following a positive opinion from CHMP, the treatment in question will see an approval decision within the next sixty days. The recommendation follows positive results of osilodrostat in a phase 3 clinical trial, in which the experimental treatment was able to cause improvements in over 80 percent of patients. There is also another ongoing study of the drug that is expected to conclude next month.