In this episode, we discuss Ultragenyx’s Priority Review Voucher that sold for $210 million, the efficacy of newborn screening on MLD, and ethical considerations needed to design and conduct clinical trials.
Ultragenyx sells PRV for $210M
This past week, Ultragenyx sold its Priority Review Voucher for $210 million.
The company received a PRV for the approval of their glycogen storage disease type Ia treatment, which was approved earlier this year.
As we have discussed on this podcast, the priority review voucher, or PRV, is an initiative the FDA introduced over a decade ago to encourage companies to develop therapies for pediatric conditions. And that program seems to be effective.
We have followed several PRV transactions for years, and $210 million is toward the high end of the range, which has often been around $100 million to $150 million range.
What’s interesting is why these vouchers are so valuable. They allow a company to shorten the FDA review period from 10 months to 6 months. In a competitive market, getting a product approved four months sooner can be worth a tremendous amount of money.
For smaller biotech companies, selling a voucher can also provide meaningful capital to support broader development programs. So this is more than a regulatory story—it’s also a story about how the economics of rare disease drug development work.
MLD and Newborn Screening
A recent publication looked at the efficacy of newborn screening to detect and treat metachromatic leukodystrophy, or MLD, in Europe.
MLD is a rare neurodegenerative disorder. Children with the condition are born looking healthy but as toxins buildup and reach a threshold, cognitive, motor, and behaviour abilities quickly decline. MLD does have a treatment available, but it is only indicated for patients who are diagnosed before that decline occurs. Which creates a bit of a catch-22; the baby looks healthy, so there is no reason to test them for this very rare condition. And once symptoms appear, it is too late to start treatment. Hence the need for newborn screening.
The study from Europe, shows that newborn screening dramatically changes the outcomes in these children. They found 7 babies with early onset MLD. All seven were treated and so far, their neurological functions are normal. That only happens if they get treated before symptoms appear. The treatment, developed by Orchard Therapeutics is an ex vivo gene therapy. And Orchard Therapeutics is working, along with several advocacy groups, to get MLD on NBS panels.
Identify patients that can benefit from treatment is a major challenge in rare disease.
That’s especially true for conditions like MLD, where treatment may be most effective before symptoms appear and irreversible neurological damage occurs.
Newborn screening can help close that gap by identifying affected babies before they become visibly ill. But getting a disease added to screening programs requires evidence, coordination, and time. The bigger lesson is that developing a treatment is only one part of the equation. We also need systems that identify patients early enough for that treatment to make a difference.
Ethical Considerations for Clinical Trials
Another publication by the International Society of CNS Clinical Trials and Methodology (ISCTM) Orphan Diseases Working Group looked at ethical considerations needed to design and conduct clinical trials.
This working group has its work cut out for it, but it’s necessary. There are many ethical dilemmas for rare neurological diseases, especially those involving children.
For example, we all want these trials to be placebo-controlled studies, but that can be difficult, or perceived to be unethical, if there is no standard treatment to act as the control group. Other considerations, such as age and cognitive abilities, can also create ethical concerns that need to be addressed.
Developing a therapy for an ultra-rare disease is already difficult because patient populations are small, recruitment is challenging, and the evidence base may be limited.
Companies also need a clear understanding of what constitutes an acceptable and scientifically meaningful clinical trial. Without that guidance, they face greater uncertainty about whether a study will generate evidence regulators can use to evaluate a treatment.
The goal isn’t to lower the standards for evidence. It’s to develop approaches that are both ethically sound and practical for these very small patient populations. That can help researchers generate meaningful results and give companies greater confidence to invest in diseases that might otherwise be overlooked.
References
Ultragenyx sells PRV for $210M
CheckRare’s Priority Review Voucher
https://checkrare.com/priority-review-voucher
Metachromatic leukodystrophy and newborn screening
https://link.springer.com/article/10.1186/s13023-026-04620-y
Ethical considerations for clinical trials
https://link.springer.com/article/10.1186/s13023-026-04635-5