Antisense Therapeutics' [ASX:ANP) has announced it has submitted to the US FDA its application for orphan drug designation of ATL1102 for the treatment of Duchenne muscular dystrophy (DMD).
The company recently announced positive results from a phase two study of ATL1102 in DMD.
In the study, ATL1102 met the primary endpoint of safety and tolerability It also met secondary endpoints that assessed its activity and efficacy.
The company says it now defining its clinical development and regulatory path for the US.
According to managing director Mark Diamond, “This is an important regulatory and commercial milestone in the further development of ATL1102 for DMD and represents another key step forward towards bringing this potentially transformative therapy to patients with a significant unmet need."
The FDA can grant orphan designation to a drug intended for the treatment of rare diseases that affect fewer than 200,000 people in the US. The regulator provides incentives to help accelerate the development of products for rare diseases. These can include tax credits towards the cost of clinical trials, the waiver of US prescription drug filing fees and orphan product exclusivity for seven years upon marketing authorisation.
The company said it is also in the process of applying for Orphan Drug designation for ATL1102 in DMD to the European Medicines Agency and expects to submit its application in the current quarter.