Amryt, a biopharmaceutical company focused on rare and orphan diseases, announces that the US Food and Drug Administration (“FDA”) has granted Investigational New Drug (“IND”) clearance for AP101. This enables the Company to begin opening US clinical trial sites as part of its ongoing global EASE Phase III clinical trial in Epidermolysis Bullosa (“EB”).
EB is a rare genetic skin disorder that can cause skin to blister and tear from the slightest friction or trauma and can, in some cases, cause blistering and erosion of the epithelial lining of internal organs. Epidermolysis Bullosa is chronic, potentially disfiguring and life limiting. There are approximately 500,000 people living with EB worldwide and there are currently no approved treatments.
There are three main types of EB:
SIMPLEX (EBS): Although often life-altering, this is the most common subtype of EB, causing less troublesome disease compared to other types. With the exception of its most severe types, EB simplex only affects the skin; blistering is limited, sometimes just restricted to the hands and feet and where clothing causes friction.
DYSTROPHIC (DEB): DEB is one of the major forms of epidermolysis bullosa. In mild cases, blistering may primarily affect the hands, feet, knees, and elbows. Severe cases involve widespread blistering and wounding that can lead to blindness, scarring and the deformity and rigidity of joints. In such cases, there is also a high chance of developing squamous cell carcinoma of the skin before the age of 35.
JUNCTIONAL (JEB): A rare form of EB. JEB has a broad spectrum of severity from milder forms to the lethal form of generalised severe JEB with most (87%) not surviving beyond the first year of life. In all forms of JEB, the most problematic wounds
occur on the scalp and lower legs.
AP101 is currently in a Phase III clinical trial (EASE), the largest ever global Phase III study conducted in patients with EB. Trial sites are already open across Europe, Australasia, Latin America and the Middle East and an interim efficacy data readout is due later this year with the top-line data readout expected in Q2 2019.
Joe Wiley, CEO of Amryt Pharma, commented: “As part of the study design, it was always planned that US trial sites would become part of our EASE Phase III clinical trial. We are pleased with today’s IND clearance from the FDA as it will accelerate patient enrolment into the largest ever global EB trial undertaken. Moreover, the FDA’s decision is positive news for eligible American sufferers of this rare condition who can now participate in the study.”