Medical company Paradigm Biopharmaceuticals (ASX: PAR) has received clearance from the US Food and Drug Administration for an investigational new drug (IND) application relating to pentosan polysulfate sodium (PPS) for the treatment of knee osteoarthritis.
The clearance was announced this morning within the 30-day review period and paves the way for the company to make an entrance into the US market.
In August, Paradigm submitted an expanded access application for the experimental PPS treatment of 10 patients, with knee osteoarthritic pain and concurrent bone marrow lesions, who have failed to respond to standard of care.
Also known as “compassionate use,” expanded access provides a pathway for access to investigational drugs, biologics and medical devices used to diagnose, monitor or treat patients with serious diseases for which there are no comparable or satisfactory therapy options available.
Clearance of the application provides validation of the safety data and product quality relating to PPS, and confirmation that it meets an unmet medical need.
Paradigm chief executive officer Paul Rennie said it paves the way for two new pre-IND applications – for osteoarthritis and the rare disease mucopolysaccharidosis – planned for submission to the USFDA by year end.
The company also plans to file a number of related submissions to global regulatory authorities.
Unmet medical need
The physician treating the patients under Paradigm’s approved IND is a former professional US footballer who works primarily with sportspeople including retired NFL players for whom knee injuries are common.
Osteoarthritis can be a major cause of disability for such patients and there are currently no registered treatments available to modify the course of the disease, representing a considerable unmet medical need.
Research shows osteoarthritis to be more than three times more prevalent in retired NFL players under the age of 60 than in the general US population.
It is estimated there are 33 million people in the US currently diagnosed with osteoarthritis.
Paradigm’s recent Phase 2b clinical trial of its injectable pentosan polysulfate sodium (iPPS) drug Zilosul was shown to reduce pain, improve joint function and slow the progression of osteoarthritis.
At mid-afternoon, shares in Paradigm Biopharmaceuticals were down 5% to $1.995.