PTC Therapeutics’ PTC518 Huntington's Disease Programme Receives FTA From US FDA

PTC Therapeutics’ PTC518 Huntington's Disease Programme Receives FTA From US FDA

By, Admin 30 September 2024

Overview

PTC Therapeutics announced that the FDA has granted Fast Track designation to the PTC518 programme for the treatment of Huntington's disease. Fast Track designation is awarded to promising therapies for diseases of high unmet need.

Words from the CEO: PTC Therapeutics

  • The granting of Fast Track designation to the PTC518 programme further supports the potential of PTC518 to provide a disease modifying therapy to Huntington's disease patients,"" said Matthew B. Klein, M.D., chief executive officer of PTC Therapeutics. 
  • We look forward to working collaboratively with FDA to continue to advance the program as efficiently as possible.

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.

Outcomes from PIVOT-HD Study

  • In June 2024, PTC announced positive results from the 12-month interim data readout of the PIVOT-HD study of PTC518 Huntington's disease patients. 
  • The 12-month data demonstrated durable dose-dependent lowering of mutant Huntingtin protein in blood cells, reaching 43% at the 10-milligram dose level along with dose-dependent lowering of mutant Huntingtin protein in the CSF at levels similar to the blood in Phase 2 patients. 
  • In addition, at 12 months, there was dose-dependent favourable clinical effect on key disease measurements, including the total motor score and the cUHDRS scale. 
  • Importantly, the interim results also demonstrated PTC518 to be safe and well tolerated with no treatment-related NfL spikes.

About Fast Track Approvals

  • 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. 
  • Programmes 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 (HD)

  • Huntington's disease (HD) is a fatal, hereditary, genetic disorder of the central nervous system. 
  • 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. 
  • HD usually presents in people who are in their 30s or 40s. Symptoms can present earlier in life, and this is called the Juvenile HD. 
  • There are also cases of infantile HD, when symptoms develop in children who are younger than 10 years old. 
  • While symptoms vary from person to person, the disease primarily affects the brain and results in abnormal movements, difficulties with speech, swallowing and walking, as well as a number of other symptoms including behavioural, cognitive and motor symptoms. 
  • While there are therapies approved for specific disease symptoms, currently, there is no cure for HD and there are no approved drugs that delay the onset or slow disease progression.

About The Company: PTC

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.