FDA Orphan Drug Designation awarded to rare disease therapies

Demyelination of neuron

UK-based biotech SynaptixBio has secured a second Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA).

The company 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 devastating, life-limiting rare diseases. Our whole strategy is centred around achieving these prestigious 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 awarded a £490,000 BioMedical Catalyst grant from Innovate UK to tackle less common variants of the disease late last year.

Earlier last year the biotech successfully led a second round of investment, taking the total up to £13.2m ($16.7m), which will take it up to the start of in-human clinical trials later this year.

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.

Antisense oligonucleotide technology

SynaptixBio is using antisense oligonucleotide (ASO) technology to tackle TUBB4A-related leukodystrophies; ASOs can alter the expression of genes, in this case a specific ASO molecule targets the mutated TUBB4A gene to stop it forming toxic proteins, which in turn help build the cells that form myelin sheaths surrounding nerve fibres in the brain. With the toxic protein suppressed, other proteins step in to help form normal myelin.

The technology has been proven in the treatment of other dystrophies, including Duchenne muscular dystrophy, and is quick and cost-effective to develop.

Disease research is carried out by the world’s leading centre for leukodystrophy studies, the Children’s Hospital of Philadelphia (CHOP), under a sponsored research agreement.

SynaptixBio has signed a worldwide exclusive license to intellectual property from CHOP, enabling commercialisation of a treatment​.

Diana Spencer, Senior Digital Content Editor, DDW

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