A biotech firm working to develop the world’s first treatment for a rare and incurable disease has secured a second Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA).
SynaptixBio received its first ODD in early 2023 for a therapeutic that targets hypomyelination with atrophy of the basal ganglia and cerebellum (H-ABC), the most severe form of TUBB4A leukodystrophy.
This second ODD allows research and development of a therapy for another form of the disease, isolated hypomyelination.
Dan Williams, CEO at SynaptixBio, said: “This ODD is a huge boost to our efforts in tackling these life-limiting rare diseases. Our whole strategy is centred around achieving these designations, which make development of therapeutics easier and more cost-effective.”
Isolated Hypomyelination was identified relatively recently and little is known about it generally.
It appears to be like H-ABC but without atrophy of the basal ganglia and cerebellum, so effectively the H without the ABC. The symptoms are reported to be milder than H-ABC.
SynaptixBio was late last year awarded a £490,000 BioMedical Catalyst grant from Innovate UK specifically to tackle less common variants of the disease, so this ODD award represents the next step in their search for therapies.
Earlier last year SynaptixBio successfully led a second round of investment, taking the total up to £13.2m, which will take it up to the start of in-human clinical trials later this year.
An ODD enables firms to cut research costs through tax credits, secure grants to offset development expenses, whilst gaining exemption from some pre-marketing authorisation requirements and regulatory fees.
In addition, it grants after approval a potential seven years of market and data exclusivity.
This second ODD designation follows the award late last year of a Rare Paediatric Disease Designation (RPDD), which can lead to the award of a Priority Review Voucher (PRV) once a product is approved.
A PRV can expedite the FDA’s product review time, and it can be sold or transferred, for example to one of the big pharmaceutical companies (Big Pharma), which can potentially offset the high costs associated with the development of rare disease therapies.