Experimental Treatment for NF-1 Gets Orphan Drug Designation

According to a story from BioPortfolio, the biopharmaceutical company SpringWorks Therapeutics recently announced that the US Food and Drug Administration (FDA) has given the company’s experimental product PD-0325901 Orphan Drug designation. This designation is for treating the rare disorder neurofibromastosis type I (NF-1). SpringWorks is dedicated to the development of therapies for severe cancer and rare diseases.

About Neurofibromatosis Type I (NF-1)

Neurofibromatosis Type I (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 typically reserved for therapies that are meant to treat diseases and conditions that are considered rare, which is defined as any illness affecting less than 200,000 patients in the US. The treatment must demonstrate potential advantages in safety or effectiveness compared to current treatments or must be for a disease that has no currently available therapies. The designation provides several benefits for the recipient company, such as tax breaks, the waiving of certain fees, and a seven year period of market exclusivity if the therapy gains approval for public use.

It is estimated that about 100,000 people are affected by NF-1 in the US, and there is an urgent need for the development of an effective treatment for the disorder, particularly for those patients whose severe disease prevents them enjoying an acceptable quality of life. A Phase 2B clinical trial testing PD-0325901 is expected to begin early in 2019. 


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