Overcoming Orphan Drug Exclusivity


Orphan drug exclusivity doesn’t need to halt your therapy products from reaching potential patients. See how FDA regulations and helpful oversight from orphan drug consultants can allow second-to-market therapies to receive approval.  


Download our white paper to learn the specifics of this regulatory process and how second-to-market drugs stand a chance against exclusivity rights. 

Overcoming Orphan Drug Exclusivity White Paper

Work Within FDA Regulations to Receive Therapy Approval

The Orphan Drug Act (ODA) has incentivized the research and development of therapies for rare diseases. Since its passage in 1983, the ODA has opened new doors and provided alternative and even first-time treatments for individuals affected by rare diseases. The creation of orphan drugs is critical to improving the quality of life for those diagnosed with these conditions. 


However, FDA regulations give first-to-market orphan drugs 7 years of market exclusivity pending approval. This incentive leaves other manufacturers facing large roadblocks toward bringing their therapy to market prior to the expiration of the 7-year exclusivity window. Yet, FDA policy does leave workarounds within the legislation for other drug developers to receive second-to-market approval during this timeframe (specific language can be found in 21 CRF 316.31(a)). 


The leveraging of this policy stipulation has been used multiple times, including the recent example of the approval for Ruzurgi (amifampridine) by Jacobus Pharmaceuticals. The formal process and application requirements to receiving second-to-market orphan drug approval call for the aid of experienced orphan drug regulatory consultants. Through proper protocols and guidance, overcoming orphan drug exclusivity is possible. 

Who is BioPharma Global?


BioPharma Global is a full-service regulatory affairs consulting firm specializing in orphan products to treat rare diseases and non-orphan products to treat indications with unmet medical needs. We offer services such as orphan drug designations and other expedited review programs, provide support for meetings with the FDA and EMA, IND filings, and comprehensive consulting on projects for regulatory strategy.