Extra 6 Months Market Exclusivity for Drugs Re-purposed for Orphan Indications

In Clinical Development, Regulation & Government by Cameron

A bill submitted to the US House of Representatives would grant an extra six months of market exclusivity to products that are re-purposed to treat rare diseases under the OPEN ACT

Last week Representative Gus Bilirakis introduced H.R. 5750, the Orphan Product Extensions Now Accelerating Cures & Treatments (OPEN ACT). The purpose of the act is to incentivise developers to re-purpose already marketed drugs to treat new rare indications.

Should developers receive a written request from the FDA, they will be eligible to apply for an extra six months market exclusivity. If the developer can demonstrate safety and efficacy in a rare indication, entirely different to populations their product is currently approved for (i.e. no rare subsets of existing indications).

This new bill seems to be loosely based on a similar system currently operating whereby, when invited, developers can receive another six months market exclusivity if they can demonstrate the safety and effectiveness of their product in paediatric populations of the already approved indication.

It’s not entirely clear how many six month periods can be added onto the length of exclusivity for  a single drug, whether that’s a paediatric plus an OPEN ACT extension, or even multiple OPEN ACT extensions. Developers such as Vertex and Alexion who have received designations and approvals for a single products across multiple rare subsets could well use the OPEN ACT to extend their market exclusivity as they continue to add to the indications treatable by their products.

You can see the introduction of the bill in the video below.