FDA Orphan Drug Designation, August 2019

FDA Orphan Drug Designation, August 2019

The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the treatment, prevention or diagnosis of a rare disease or condition, which is one that affects less than 200,000 persons in the US or meets cost recovery provisions of the act. This orphan drug designation provides a guarantee of seven-year exclusivity deal to market the drug and protects it from generic competition.

In 2017, the FDA set out to modernize its orphan drug designation process. FDA aimed to address the backlog of orphan drug designation requests and streamline the process for future requests, making it easier for drug manufacturers to address these rare diseases.

Cancer is now being viewed more and more frequently as a matrix of rare diseases, which are often highly differentiated, based on the presence or absence of specific genetic biomarkers, and these smaller populations of cancer types with shared characteristics lend themselves to orphan drug development. Today, more than 45% of all approved orphan drugs are intended to treat narrowly defined patient subgroups within oncology. Blockbuster oncology drugs such as Merck’s Keytruda and Bristol-Myers Squibb’s Opdivo will also sit at the top of the list in 2022.  

To read out the complete blog with companies list, please click on the link: https://fda-orphan-drug-designation-august19.blogspot.com/2019/09/the-orphan-drug-designation-program.html

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