FDA Approves Novartis’ Cushing’s Disease Medication 

By Danielle Bradshaw from In the Cloud Copy  

The Swiss-born pharmaceutical company, Novartis, has been given permission by the Food and Drug Administration (FDA) to begin distributing their oral tablet medication Isturisa, which can be used to treat Cushing’s disease in adults. Isturisa will be used by patients whose Cushing’s disease either can’t be treated by surgical procedures performed on their pituitary glands or who have had surgery, but their symptoms still haven’t been alleviated.  

What Exactly Is Cushing’s Disease? 

Cushing’s disease, a form of Cushing syndrome (or hypercortisolism), is a rare disease that happens when a person’s body produces larger than normal amounts or the cortisol hormone for long periods of time. Overproduction of the hormone is caused by pituitary tumors which stimulates the adrenal gland into making too much cortisol.  

The disease is typically found in adults around the ages of 30 to 50-years-old and it’s more likely – three times as much – to be found in women than men. There are two ways in which a person can become afflicted with Cushing’s disease: they either naturally produce too much cortisol within their bodies or they take an (oral) corticosteroid medication.  

When properly regulated by the body, cortisol can help to manage blood pressure, regulates metabolism, aids with memory formation, and supports the healthy development of fetuses in pregnant women. An overabundance of cortisol – Cushing syndrome – can result in a host of physical and health issues such as: 

  • A rounded face 
  • fatty lump between the shoulders 
  • Purple or pink-colored stretch marks 
  • Decreased bone density (osteoporosis) 
  • High blood pressure 
  • Type two diabetes  

Results and Side Effects Discovered bIsturisa Testing 

The way Isturisa treats Cushing’s disease is by targeting and inhibiting the 11-beta hydroxylase enzyme which then stops the production of cortisol. The oral drug’s ability to do so effectively and safely was tested in a trial with 137 adult patients – most of whom were women – that averaged around 41-years-old.  

Each participant was started off with a 2mg dose of the drug that they took twice a day. The dosages of Isturisa could then be gradually increased over a twoweek period to 30mg twice a day throughout the course of the 24-week study. At the end of the test period, it was found that about half of the participants’ cortisol levels were lowered to less hazardous levels and had normalized.  

An additional 12-weeks of testing was then done with the patients that took well to the Isturisa treatments where either placebo drugs or Isturisa were randomly distributed to these participants. At the end of this particular testing period, the FDA was able to report that 86% of the patients that took part and were receiving the Isturisa had maintained their normalized cortisol levels as compared to the 30% who were on the placebo drug.  

While the results of the Isturisa clinical trials are indeed good news for those suffering from Cushing’s disease, there have been some commonly observed side effects that come from taking the drug.  

There have been reports of: 

  • Headaches 
  • Vomiting 
  • Fatigue 
  • Nausea
  • Edema (swelling caused by the body retaining fluid) 
  • Hypocortisolism (cortisol levels that are too low) 
  • Adrenal insufficiency 
  • QTc prolongation (condition typified by problems with the heart’s rhythm) 

In the wake of the testing, the FDA granted Isturisa an orphan drug designation. Orphan drug designations are granted to any sort of biological agent – in this case, a medical drug – which is made to treat any kind of rare disease, illness, or condition.  

 

 

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