Okay, I’m going to be perfectly honest: I don’t quite know what to make of this. On the one hand, you have some excellent news for people living with mucopolysaccharidosis II (MPS II)—AKA Hunter syndrome—in the form of a possible new gene therapy approved for pediatric use.
Regenxbio, a biotech company developing gene therapies using their own specialized delivery platform, received a pediatric disease designation for its gene therapy targeted at Hunter syndrome. Which means that if the therapy successfully receives approval for a pediatric indication, it will a) become only the second approved therapy for patients with Hunter syndrome, and b) earn Regenxbio a coveted “pediatric priority review voucher.”
Which is… what exactly?
Basically, it’s a “hurry up and review my drug faster” coupon the company can use to nearly halve FDA review time on another drug from the usual 10 months to six months. And it doesn’t have to be another Hunter syndrome drug or even a pediatric drug.
Regenxbio could use it to give priority review to any of their other gene therapies in the pipeline, from wet age-related macular degeneration (wet AMD) to homozygous familial hypercholesterolemia (HoFH).
Or… get this… they can sell it.
And not for chump change, either. Some companies have sold their vouchers for as much as $350 million—that kind of money can go a long way towards funding smaller, scrappier biotechs, and right now Regenxbio has two designations: their newest for Hunter syndrome and another one they got in 2016 for mucopolysaccharidosis I (Hurler syndrome). If they can turn both of those into pediatric indications, they stand to save either a combined eight months of review time or make a big pile of cash they can plow into further research and development.
Will that ultimately result in cost savings down the line for Hunters syndrome patients?
The Magic 8-ball’s a little unclear on that one. But at least one company is pursuing some innovative therapies for a difficult disease, and at least the FDA is trying to reward innovation instead of impeding it.