In May 2025, the World Health Assembly (the supreme decision-making body of the World Health Organization) adopted a landmark resolution declaring rare diseases a global health priority.
And in February this year the FDA’s Priority Review Voucher (PRV) programme was re-authorised and signed into law; it will remain in place until reviewed again in September 2029.
The PRV scheme incentivises development of new treatments for rare paediatric conditions, among other things. When a company is awarded a PRV, it is entitled to a cut in drug review times from ten months to six months; alternatively, it can sell the voucher. Recent sales have been worth between $150m to $200m; in March this year, Cyprium Therapeutics sold its PRV to Fortress Biotech for $205m.
PRVs are considered to be a prime source of non-dilutive capital. When a PRV is sold, the cash generated does not require the seller to issue new equity.
So, perhaps unsurprisingly, investment in rare disease biotechs has increased following renewal of the programme. Big Pharma, venture capital firms and others are actively investing in smaller rare disease biotechs through early-stage funding rounds, asset acquisitions and partnerships.
Read the article in full here.