Antisense Therapeutics announced today that the US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to the ATL1102 for treatment of Duchenne muscular dystrophy. ATL1102 is an antisense drug aimed at reducing muscle inflammation. PPMD is excited for this news and we are happy to see progress.
Read the Announcement from Antisense
ANP receives US FDA Orphan Drug Designation for ATL1102 for the treatment of DMD
-Antisense Therapeutics Limited [ASX:ANP | US OTC:ATHJY], (the Company) is pleased to announce that the US Food and Drug Administration (FDA) Office of Orphan Products Development has granted an Orphan Drug Designation (ODD) to the Company’s drug ATL1102 for treatment of Duchenne muscular dystrophy (DMD), a rare and fatal muscle wasting disease where inflammation in the muscle leads to fibrosis and death of muscle tissue.
ATL1102 is an antisense drug designed to reduce inflammation in human disease. It was recently assessed in a Phase II study in adolescent non-ambulant patients with DMD, meeting the primary endpoint of the study with confirmation of the drug’s safety and tolerability. ATL1102 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 by the FDA 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 for drugs with ODD 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.
Orphan Drug Designation together with receipt of a Rare Pediatric Disease (RPD) Designation from the FDA last month, which, at the time of marketing approval, may also entitle the Company to receive a RPD priority review voucher (which can be traded, with recent sales of over US$100m), adds substantial commercial value and momentum in respect of the clinical development and commercialisation plans for ATL1102 in the US.
Mark Diamond, Chief Executive Officer of Antisense Therapeutics said: “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.”