Pharmaceutical company Sarepta Therapeutics just received approval for its new $300,000 muscular dystrophy drug on September 19, even though some questions remain about its efficacy. However, the company has even more to look forward to as they have also been awarded an extremely rare fast track voucher that boost approval.
This fast tracking is known as the Rare Pediatric Disease Program Voucher Program. It was established by the US Food and Drug Administration—and approved by Congress—in 2007 as an effort to encourage drug development for less common tropical diseases, later to be expanded to rare pediatric disorders, as is the case, here.
“Rare Pediatric Disease designation builds upon the Fast Track and Orphan Drug designations which the FDA has already awarded to ezutromid, recognizing a significant unmet medical need in the treatment of [Duchenne muscular dystrophy (DMD)],” explains Summit CEO Glyn Edwards in a recent press release. “We plan to leverage these regulatory advantages in the continued clinical development of ezutromid, which is currently in a Phase 2 clinical trial called PhaseOut DMD, to bring ezutromid to patients in need as quickly as possible.”
A company awarded this voucher has the option to use it to get a quicker government review of one of its future drugs. It can also sell the voucher to another company.
However, former FDA official Dr. Tim Coté, who now runs a consulting firm for rare disease drugs, explains “The only people who would buy a priority review voucher would be someone who had something that wouldn’t merit its own priority review but they want the priority review.”
In other words, companies that are willing to pay for these vouchers are also the most likely trying to get access to a particular medicine for treating a more common disease on the market before another—competing–drug hits the market.
He goes on to say, “It might be a blockbuster — say a new statin. A priority review would make all the difference in the world.”
What Sarepta plans to do with this voucher, though, might be unclear. Nancy Goodman, the executive director of Kids v Cancer and champion of the bill supports the program. In fact, she created her foundation soon after her son died of medullolastoma seven years ago. Medulloblastoma, of course, is a rare pediatric brain cancer.
At the time, she said, there were no drugs to treat her 10-year-old son. Now, she comments, “I believe and hope we will see a whole crop of pediatric disease drugs because of the program.”