Examples of treatments used off-label


Examples of treatments used off-label

Thalidomide is used off-label in dermatology to treat rare diseases where there are no alternative treatments available, such as Prurigo Nodularis, Osler-Weber-Rendu Disease or Jessner- Kanof Disease.

Thalidomide had been developed as a sedative agent and was taken off the market in 1961 after it was linked to thousands of birth defects. Despite its teratogenicity, it was reintroduced in 2008 as an authorized treatment for multiple myeloma following extensive research and development.

Later, it was approved for the treatment of Erythema nodosum leprosum, a skin complication in Lepra patients.

Pulmonary Arterial Hypertension is a life threatening condition, Sildenafil (Viagra) has been used off-label to treat the disease with good results. Now it has been recently authorised as an orphan drug under the name of Revatio with a new label that states Pulmonary Arterial Hypertension as an indication.

The antibiotic Fucidin used to treat bacterial infections caused by a specific germ is considered to be standard care for the treatment of specific infections in cystic fibrosis patients. However, the treatment is not available in every EU Member State.



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