Benitec Biopharma (ASX: BLT) has received orphan drug designation from the FDA for its BB-301 which has been created to treat oculopharyngeal muscular dystrophy.
The US FDA granted the orphan drug designation which paves the way for Benitec to fast track BB-301 through development including clinical regulatory approvals, potential extension of patent duration, tax credits for qualified trials and an exemption from FDA application fees.
This latest grant follows orphan drug designation status from the European Medicines Agency early last year.
“We believe BB-301 represents a promising new approach for the treatment of oculopharyngeal muscular dystrophy and has potential to make a meaningful impact for patients who have this debilitating disease,” Benitec chief executive officer Greg West said.
He added the company was aiming to get the drug into human clinical trials by the end of the year.
“We also believe this program, if successful, can act as a proof of concept for our ground breaking ‘silence and replace’ technology for other therapeutic targets potentially expanding market opportunities for Benitec and paving the way for the development of other monogenic orphan disease programs in the future,” Mr West said.
Using DNA directed RNA interference, BB-301 aims to silence the mutant gene associated with oculopharyngeal muscular dystrophy, while reinstating a copy of the normal gene to restore function.
BB-301 is undergoing nonclinical safety studies and Benitec plans to lodge an investigational New Drug Application by the end of the year.
Benitec has also discussed regulatory development pathways for BB-301 with FDA, Health Canada and European bodies, as well as optimisation of production processes and protocols.
Oculopharyngeal muscular dystrophy results in the degeneration of muscle strength in multiple parts of the body.
Sufferers usually have severe difficulties with swallowing, eye lid drooping, tongue atrophy, weak voice and proximal lower and upper limb weakness.
The disease isn’t usually obvious until sufferers reach their 50s and 60s and, as it progresses, patients become malnourished, lose weight and become dehydrated. Sufferers also end up with regular bouts of aspiration pneumonia which often leads to death.
There is currently no cure for the disease.
By mid afternoon trade, shares in Benitec had risen more than 14% to A$0.27.