Antisense Therapeutics (ASX:ANP) has announced that the US FDA has granted Orphan Drug designation to the Company’s drug ATL1103 for treatment of Acromegaly.
ATL1103 is an antisense drug designed to block growth hormone receptor expression in advanced clinical development and is a potential treatment for diseases associated with excessive growth hormone action including acromegaly.
Orphan drug designation is granted by the FDA to drugs intended for the safe and effective treatment of rare diseases that affect fewer than 200,000 people in the US. The FDA provides incentives for companies to develop products for rare diseases which may include tax credits towards the cost of clinical trials, waiver of US prescription drug filing fees and orphan product exclusivity upon marketing authorisation, which means that the FDA may not approve any other applications to market the same drug for the same indication for seven years.
The process of applying for Orphan Drug designation for ATL1103 in acromegaly to the European Medicines Agency has commenced. Orphan Drug designation in the European Union qualifies the sponsor of the drug for 10 years of marketing exclusivity following marketing authorisation. Other benefits relate to assistance in developing clinical protocols, reduced fees, and access to EU-funded research grants.
According to Antisense managing director, Mark Diamond, “This is an important regulatory and commercial milestone in the further development of ATL1103 and represents another key step forward towards bringing this potentially transformative therapy to patients with a significant unmet need.”