SynaptixBio, the only company licensed to commercialise a treatment for the rare, deadly disease H-ABC, cites structural factors and lack of incentives for the UK trailing other countries.
Dan Williams PhD, CEO at leading rare disease biotech SynaptixBio, said; “While the UK is known for high-quality science and innovation, it has seen a sharp contraction in biotech fundraising. Without an equivalent to the FDA PRV program, UK rare disease biotechs rely heavily on public markets, private investment, or acquisition by larger global pharma to secure capital. “
Market analysis by Schroder’s, from April this year, stated: “With public markets grappling with valuation volatility, the UK’s “golden triangle” of innovation – spanning London, Oxford, and Cambridge – continues to produce the next generation of biotech champions.
By comparison, investment in rare disease biotechs has increased in the US following renewal of the FDA’s Priority Review Voucher (PRV) program, according to SynaptixBio.
In February this year the PRV (the Rare Pediatric Disease Priority Review Voucher) program was signed into law and will remain in place until reviewed again in September 2029.
According to VC firm V-Bio, “Reauthorisation of the FDA’s Rare Pediatric Disease Priority Review Voucher (PRV) scheme has restored financial certainty and sparked intense interest from large pharma in the region.”
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.
Williams continued: “The US dominates because the PRV program creates a highly valuable and, more importantly, tradable asset. VCs and private equity firms are far more willing to invest in rare disease biotechs simply because they provide a financial return on investment.”
PRVs have been sold recently for between $150M to $200M.
“Historically, the UK’s Achilles’ heel has been the “Valley of Death”- the gap between brilliant seed-stage science and the massive capital required for clinical trials. Too often, UK companies were forced to list in New York just to access the depth of capital needed to scale.”
Rare disease statistics
Around 1 in 17 people will be affected by a rare disease during their lifetime. In the UK, this amounts to over 3.5 million people, but only around 5% of rare diseases have an approved treatment. The vast majority of the c10,000 known rare diseases have no effective therapy.
Around 80% of rare diseases are monogenic, caused by a mutation in a single gene, which makes them more suitable for highly targeted treatments such as gene silencing.
To find out more about SynaptixBio please visit https://www.synaptixbio.com/.
