4D pharma plc (AIM: DDDD), a pharmaceutical company focusing on significant new therapeutic areas, announced today that the U.S. Food and Drug Administration ("FDA") has granted orphan drug designation for Rosburix™, for the treatment of ulcerative colitis in paediatric patients ("PUC").
Rosburix™ is 4D's second live biotherapeutic to receive orphan designation, having received orphan designation from the FDA for the treatment of paediatric Crohns with Thetanix™ in September 2013.
PUC is an inflammation, or painful swelling of the lining of the large intestine or colon. The inflammation can lead to sores and ulcers which can bleed, produce pus and cause pain. Although some patients with PUC do show periods of remission, PUC is a long term condition, requiring management throughout their life. Despite a number of approved therapeutic options for treatment, surgical removal of the colon is still required in many cases and in the US it is estimated that 8% of paediatric patients undergo colectomy at 1 year, 15% at 3 years, and 20% at 5 years following diagnosis.
It is estimated that in the US, 28 of every 100,000 children are affected with PUC and it is one of the most common gastrointestinal conditions managed by gastroenterologists in the US. There is a clear, unmet medical need for better therapeutic treatments for children suffering from UC.
"4D is committed to developing the new class of live biotherapeutics and delivering novel therapies to patients that could make significant differences to their lives" stated Duncan Peyton 4D's Chief Executive Officer. "Having first been granted orphan designation by the FDA for Thetanix™, this orphan designation for Rosburix™ shows that 4D pharma continues to lead the development of this new therapeutic class."