The US regulator has granted Antisense Therapeutics' (ASX:ANP) ATL1102 orphan designation for the treatment of Duchenne muscular dystrophy (DMD).
DMD is a rare and fatal muscle-wasting disease where inflammation in the muscle leads to fibrosis and death of muscle tissue.
ATL1102 is designed to reduce inflammation. It was recently assessed in a phase two study in adolescent non-ambulant patients with DMD, meeting the primary endpoint of the study with confirmation of the drug’s safety and tolerability.
The company said ATL1102 has also demonstrated strong effects on disease progression parameters with improvement or stabilization across multiple measures of muscle function and strength.
Orphan drug designation is granted to encourage and provide special assistance to companies that develop drugs for effective treatment of rare diseases that affect fewer than 200,000 people in the US.
Various incentives may include tax credits towards the cost of clinical trials, waiver of US prescription drug filing fees and orphan product exclusivity upon marketing authorisation for seven years.
It could also mean the company might receive a Rare Pediatric Disease Designation priority review voucher. It adds substantial commercial value and momentum with respect to the clinical development and commercialisation plans for ATL1102 in the US.
According to Antisense Therapeutics CEO, Mark Diamond, “The Orphan Drug Designation is a very significant commercial milestone for the Company. ODD provides a cost reduction and regulatory streamlining mechanism made available by the FDA for Companies developing therapies for rare diseases along with key additional intellectual property protection for ATL1102 in DMD with seven years of marketing exclusivity from generic competition. This FDA recognition spotlights the significant unmet need for patients living with DMD and ATL1102’s potential to markedly improve the quality of life of those boys so desperate for an effective treatment.”