UK osteopetrosis drug granted FDA Orphan Drug status

RNA delivery and therapeutics company SiSaf Ltd has announced that its siRNA therapeutic for patients with Autosomal Dominant Osteopetrosis Type 2 (ADO2) has been granted Orphan Drug Designation by the FDA.

In addition, due to the serious manifestations of this rare skeletal disorder in children, SIS-101-ADO has been granted Rare Pediatric Disease Designation for the treatment of Autosomal Dominant Osteopetrosis.

FDA Orphan Drug Designation provides incentives such as tax credits for clinical trials, exemption from user fees and expanded marketplace exclusivity.

The Rare Pediatric Disease Designation entitles SiSaf to apply for a priority review voucher that can be used to have the drug approval process expedited by the US drug regulator.

SiSaf’s Founder and CEO, Dr. Suzanne Saffie-Siebert, said:

“Being granted Orphan Drug Designation and Rare Pediatric Disease Designation is a major milestone in our drive to move our revolutionary siRNA treatment forward to alleviate the pain and suffering that Osteopetrosis ADO2 inflicts.

“SIS-101-ADO ushers in the potential for a new era of personalised care and treatment options for ADO2 and other rare bone and skeletal diseases.”

The FDA awarded SIS-101-ADO Rare Pediatric Disease Designation due to the serious or life-threatening manifestations of ADO that primarily affect children including blindness from optic nerve compression, anomalies in dental and craniofacial development and scoliosis.

There are currently no approved treatments for Osteopetrosis ADO2 and no other treatments currently in the clinical trials process.

If approved, SIS-101-ADO would be the first treatment for Osteopetrosis ADO2 and could provide life-altering benefits for those who suffer from the disease.

SiSaf is currently preparing for first-in-human clinical trials.

Saffie-Siebert added:

“SIS-101-ADO and other Bio-Courier formulated drugs will not only be able to treat rare skeletal disorders but can clear the way for therapeutics for other rare diseases once thought impossible to treat.”