Magic drug approved for treatment of Neurofibromatosis type 1

US FDA has finally approved a magic drug for treating children with neurofibromatosis type 1. This magic medicine is the first drug approved for treatment of neurofibromatosis 1 treatment and is named as Koselugo (selumetinib). Currently FDA has given permission to treat children older than 2 years. The drug has shown significant effect in controlling the tumour growth in neurofibromatosis type 1.
Neurofibromatosis is a group of genetic disorders. The most common types are NF 1 and NF2. NF 1 occurs in 1 out of 3000 infants that are born in the United States. It is also called as von Recklinghausen’s disease. This is a progressive disfiguring disease which starts in the early childhood. Neurofibromatosis type 1 has multiple tumours that originates from the nerves. They include plexiform neurofibromas which are large tumours looks like a bunch of grapes. They occur anywhere in the body, including the face, limbs, areas around the spine and inside the abdomen that may affect other organs. In addition there are other symptoms like changes in skin pigmentation, skeletal abnormalities and various neurological symptoms. About 30-50% with NF1 develop plexiform neurofibromas.
Till now there was only surgical treatment available for neurofibromatosis type 1 and 2. Neurosurgeons used to remove the tumours which are symptomatic. Removal of all tumours are practically not possible as there may be thousands of small tumours in the body. These tumours often grow in the face. As the child grows tumour covers whole of the face and patients often go into depressive state due to the disfigurement that they suffer.

First Ever successful Medicine For treatment of Neurofibromatosis type 1

Koselugo or Selumetinib is the first drug approved in the treatment of neurofibromatosis 1 disorder. This is a boon to the children suffering from NF1. Selumetinib is a kinase inhibitor. In other words it blocks a particular enzyme which results in arresting the tumour growth. Koselugo is an oral tablet and it has to be taken twice a day.

What are the side effects of Koselugo ( Selumetinib) ?

There is no difference in side effect risk in young or older children. Also there is no sex difference. The most common side effects are the following

• vomiting
• rash
• abdominal pain
• diarrhea
• nausea
• fever
• mouth sores
• headache
• nail infection
• dry skin
• fatigue
• body ache
• itching

Serious side effects are often rare. They include

• severe skin rashes
• increased risk of bleeding
• visual problems including loss of eye sight
• heart muscle weakness