FDA Grants Fast Track Designation to PTC518 Huntington's Disease Program
"The granting of Fast Track designation to the PTC518 program further supports the potential of PTC518 to provide a disease modifying therapy to Huntington's disease patients," said
PTC518 was discovered using PTC's innovative splicing platform, following the successful discovery and development of Evrysdi® (risdiplam) for the treatment of spinal muscular atrophy.
In
About Fast Track Designation:
Fast Track is a process designed by the FDA to facilitate the development and expedite the review of investigational treatments that demonstrate a potential to address unmet medical needs in serious or life-threatening conditions. Programs with Fast Track designation can benefit from early and more frequent interactions with the FDA to discuss the product candidate's development plan in addition to a rolling submission of the marketing application. Product candidates with Fast Track designation may also be eligible for priority review and accelerated approval.
About PTC518
PTC518, a small molecule that can be taken orally, reduces the production of the mutated Huntingtin protein that leads to injury and death of the neuron, which results in disease progression. The orally bioavailable small molecule penetrates the blood brain barrier, is selective, titratable, and not effluxed.
About Huntington's Disease
Huntington's disease (HD) is a fatal, hereditary, genetic disorder of the central nervous system.1 It is caused by a defective gene. This gene produces a protein, called Huntingtin, which is involved in the functioning of the nerve cells in the brain (neurons). When the gene is defective, it produces an abnormal (or mutated) Huntingtin protein that is toxic and causes neuron damage and neuron death.2 HD usually presents in people
About
PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to children and adults living with rare disorders. PTC's ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC's mission is to provide access to best-in-class treatments for patients
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References:
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World Health Organization , 2020. 8A01.10 Huntington disease. Available at: https://icd.who.int/browse11/l-m/en#/http://id.who.int/icd/entity/2132180242 Accessed October 2021. - Gatto EM, González Rojas N, Persi G, et al. Clin Parkinsonism Rel Disord 2020;3:100056.
- Tabrizi SJ, Flower MD, Ross CA, et al. Nat Rev Neurol 2020;16(10):529–546.
- Roos RAC. Orphanet J Rare Dis 2010;5:40.
- Kirkwood SC, Su JL, Conneally P, et al. Arch Neurol 2001;58(2):273–278.
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