Experimental Treatment for Neurofibromatosis Type 1 Earns Orphan Drug Designation in the EU

According to a story from BioPortfolio, the biopharmaceutical company SpringWorks Therapeutics, Inc., has recently announced that the European Commission has given the company’s investigational drug candidate mirdametinib Orphan Drug designation. This drug, which is an MEK1 and MEK2 inhibitor, is in development to treat the rare genetic disorder neurofibromatosis type 1 (NF-1). SpringWorks, focuses on the development of breakthrough treatments for cancer and life-threatening rare diseases.

About Neurofibromatosis Type 1

Neurofibromatosis Type 1 (NF-1) is a genetic disorder that has impact on a number of the human body’s functional systems. NF-1 is caused by a mutation of a gene located on chromosome 17 which is responsible for the production of the protein neurofibromin. This mutation can be heritable, but about half of cases are the result of spontaneous mutation. Symptoms of NF-1 include epilepsy, tumors affecting the nervous system and skin, spots on the skin, scoliosis and other skeletal deformations, learning and mental impairment, and vision disorders. People with the disorder also have greater risk of cardiovascular disease and cancer compared to unaffected people. The severity of symptoms can vary greatly; some people live fairly typical lives, whereas others are faced with serious quality of life challenges. There is no cure, and treatment generally involves managing serious symptoms and complications as they appear. To learn more about NF-1, click here.

About Orphan Drug Designation

Orphan Drug designation is an incentive program intended to encourage the development of drugs to treat rare diseases. This is defined as a disease that affects no greater than 5 of every 10,000 people in the EU. To qualify for this designation, the drug candidate must display potential advantages in efficacy or safety over current treatments or must satisfy a medical need that is not being met. This designation confers several advantages to the recipient company, such as protocol assistance, the reduction of certain fees, and a ten year period of market exclusivity if the drug is approved by the European Medicines Agency (EMA).

Mirdametinib in the US

Mirdametinib has also earned Orphan Drug designation and Fast Track designation from the US Food and Drug Administration (FDA) for patients two years or older in age with plexiform neurofibromas (a type of tumor) that are inoperable, are progressing, or are otherwise causing substantial morbidity.


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