When does the FDA decline to award a Priority Review Voucher?

A sponsor being awarded a Priority Review Voucher (PRV) makes headlines, moves share prices and is a major milestone for any company. However, being awarded a voucher is not a given, and I review the reasons sponsors may be denied PRVs below.

Trawling through the FDA's archives, I've compiled 18 applications where, although the application was approved, the sponsor's request for a priority review voucher was denied.

• Active drug/moiety previously approved
  The application must contain no active ingredient/moiety that has been previously approved in any other application.

• Application was not eligible for priority review
  The application must be deemed eligible for priority review to be eligible to receive a PRV upon approval.
  

• Indication not eligible for a PRV
  To receive a rare pediatric disease PRV the application must be for a rare disease or condition that is a serious or life-threatening disease that primarily affect individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents.

• Clinical studies were not conducted by the sponsor
  To be eligible for a tropical disease PRV, the application must contain reports of one or more new clinical investigations (other than bioavailability studies) that are essential to the approval of the application and conducted or sponsored by the sponsor of the application.
  

• Sponsor did not notify FDA of intent to request a PRV at application submission
  The sponsor of a rare pediatric disease product application that intends to request a PRV must notify the FDA of such intent on submission of the  application.

• Application did not rely on clinical data from pediatric studies
  The application must rely on clinical data derived from studies examining a pediatric population and dosages of the drug intended for that population.

The table above details all 18 applications from my review. In many of these instances, such as where an application was not granted priority review, the sponsor would have known prior to approval that it would not receive a PRV.

However, where a sponsor is expecting a PRV but is ultimately denied one, the impact can be very significant. The most notable example is the PRV denial for Lyfgenia (bluebird bio), which ultimately led to the company's downfall and eventual sale to private equity.

Another notable example, for different reasons, was the PRV denial for Sezaby (Sun Pharma). Sun Pharma took the FDA to court, successfully appealed the denial decision, and was subsequently awarded a PRV at the beginning of this year, which it later sold for $195 million.

To get our detailed insights reports early, please sign up for updates (link at top of this page)