Under the FDA's Rare Paediatric Disease Priority Review Voucher program, which came into operation last year, a drug sponsor may request a priority review voucher from the FDA when submitting an NDA for a rare paediatric disease indication. If the FDA agrees to grant the request, the drug sponsor receives a priority review voucher if its drug receives marketing approval.
The priority review voucher can be redeemed by the drug sponsor to obtain priority review for a subsequent marketing application for a different drug. “Priority review” means that the FDA will review the drug within 6 months instead of the standard 10 months. Alternatively, the voucher can be sold to the highest bidder and the prices are rising steeply. Last month, Retrophin sold a priority review voucher it had received to Sanofi for $245 million. Last week, United Therapeutics agreed to sell its priority review voucher to AbbVie for $350 million. (1)
Two companies which separately received rare paediatric disease designation from the FDA last week are Sarepta and BioMarin. (2, 3) Both companies have recently submitted NDAs for their treatments for Duchenne muscular dystrophy (the background to this was provided in a post last November titled Moms, Regulators, Biotech Startups and the Battle). If either Sarepta and/or BioMarin are granted marketing approval, the win will come with the added bonus of a sellable asset potentially worth $350 million. (4)
It begs the question - what qualifies as a “rare pediatric disease” in the eyes of the FDA? In draft guidance provided by the FDA late last year it is stated
Under our interpretation of section 529(a), a drug would qualify as a drug for a “rare pediatric disease” if the entire prevalence of the disease or condition in the U.S. is below 200,000 and if more than 50% of patients with the disease are 0 through 18 years of age. Another way a drug may qualify as a drug for a “rare pediatric disease” is if it is for an “orphan subset” of a disease or condition that otherwise affects 200,000 or more persons in the U.S., and if this subset is primarily (i.e., more than 50%) comprised of individuals aged 0 through 18 years. …
An applicant cannot receive a rare pediatric disease priority review voucher if the application seeks approval for an adult indication in the original rare pediatric disease product application. We interpret this criterion to mean that, to preserve voucher eligibility, the applicant cannot seek approval for a different adult indication (i.e., for a different disease/condition) in the original rare pediatric disease application. If the applicant seeks approval for use by pediatric and adult populations with the rare pediatric disease, the applicant will still be eligible for a voucher if the approved use includes pediatric use, as described in Question 3. If the applicant obtains approval for use only in an adult population with the rare pediatric disease, the applicant is ineligible for a voucher. Thus, under this interpretation, an applicant can preserve voucher eligibility even if the applicant seeks approval for use by adults in addition to pediatric patients with the rare pediatric disease. One reason we are interpreting the statute in this way is to avoid incentivizing sponsors toexclude adults affected by the rare pediatric disease from clinical trials or to exclude adult data from the subsequent marketing application solely for the sake of voucher eligibility, when such exclusions may not be scientifically or ethically acceptable. (5)
Last month Neuren announced that it had received encouragement from the FDA to conduct a brief paediatric tolerability clinical trial in which higher doses of trofinetide will be tested in Rett syndrome patients below the age of 16. Neuren said that the trial will enable it to confirm the optimum dose levels for its Phase 3 trial, as well as generating useful information on the treatment of children and younger adolescents.
I have no idea whether Neuren is either planning to or could be eligible to request rare paediatric disease designation for Rett syndrome but it is emerging as an intriguing possibility.
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