The MFDS designated Mobocertinib as an orphan drug

Ipilimumab, added for use in metastatic direct colorectal cancer

The MFDS (Minister Eui-kyung Lee) announced that Mobocertinib was newly designated as an orphan drug, and it announced on June 1 that target diseases were added for three types, such as Ipilimumab, designated as an orphan drug.

 

An orphan drug is a drug that is used for the purpose of diagnosing or treating a rare disease, a drug that has no or no substitute drug, or is significantly improved in safety or effectiveness than a substitute drug, and has been designated by the MFDS.

 

In order to support the development of treatments for rare and incurable diseases, the MFDS firstly authorizes orphan drugs and operates 'Orphan drug designation system' so that it can separately set data, standards, and conditions for approval according to the nature of the disease.

 

The main announcement of this orphan drug designation was that Mobocertinib was designated for the treatment of patients with non-small cell lung cancer with epidermal growth factor receptor mutation, and Ipilimumab has been further designated for use in recurrent metastatic colorectal cancer in adult patients with DNA mismatch repair defects, etc.

 

Also, Ixazomib was added to maintenance therapy of patients with multiple myeloma who received autologous hematopoietic stem cell transplantation, and Ravulizumab was added as a treatment for patients with atypical hemolytic uremic syndrome.

 

Atypical Hemolytic Uremic Syndrome (aHUS) is a disease that occurs in blood clots in blood vessels, interferes with blood flow, destroys red blood cells, and causes hemolytic anemia.

 

An official from the MFDS said, "The orphan drug designation is expected to help in the development of treatments for people with rare and intractable diseases., and will continue to develop policies to ensure patient care opportunities in the future."