The legislation states that the clinical dossier of an
orphan drug or vaccine should be built on a realistic assessment of the qualitative and quantitative nature of the studies that can be performed.
However, consider a different view: Bristol-Myers Squibb was shielded from the possibility that other companies would have obtained Taxol or used the Government's own clinical data on Taxol trials to file an earlier FDA application - a particularly important issue when it was thought Taxol would be protected under the
Orphan Drug Act.
The United States Food and Drug Administration (FDA) has granted
orphan drug designation to United States-based Stoke Therapeutics for its lead product candidate, STK-001, an investigational new treatment for Dravet syndrome, a severe and progressive genetic epilepsy, it was reported yesterday.
Taiwan biopharma company OBI Pharma Inc (TPEx:4174) reported on Wednesday the receipt of the US Food and Drug Administration's (FDA)
orphan drug designation (ODD) for OBI-3424 for the treatment of Acute Lymphoblastic Leukemia (ALL),also known as Acute Lymphocytic Leukemia.
"We see the FDA's granting of
orphan drug designation for PCM-075 as underscoring the medical need for new therapies for patients with AML and an important step forward in our clinical development program."
The
Orphan Drug Act also provides a seven-year exclusivity period that blocks competition and gives other financial incentives to companies that develop drugs for diseases that affect fewer than 200,000 people.
Orphan drug designation qualifies a company for several benefits under the
Orphan Drug Act of 1983 (ODA), as amended.
Clavis Pharma has also applied for
orphan drug status for the product in the USA as a potential new treatment for pancreatic cancer.
Once orphan designation is granted, the TGA waives evaluation fees and sets up a priority evaluation pathway for processing
orphan drugs.
"Receiving EMEA
orphan drug designation for decitabine may accelerate the approval process in Europe by allowing us to combine the results of our Phase III program in the United States with previous European studies that have produced positive results," said Dr.
Nord is a federation of voluntary health agencies and with rare disorders dedicated to the identification, treatment, and cure of "orphan diseases." We worked for passage of the
Orphan Drug Act in 1983, and we continue to monitor the law to ensure that it lives up to its promise of promoting new treatments and cures for rare disorders.
These two biologicals/drugs have been designated as
orphan drugs pursuant to the
Orphan Drug Act of 1983.