Regeneron Pharmaceuticals, and Sanofi announced that the US Food and Drug Administration (US FDA) has granted Breakthrough Therapy designation to dupilumab for the treatment of adults with moderate-to-severe atopic dermatitis (AD) who are not adequately controlled with topical prescription therapy and/or for whom these treatments are not appropriate. Dupilumab is an investigational therapy blocking IL-4 and IL-13, two cytokines required for the Th2 immune response. The designation is based on positive results from phase 1 and 2 clinical trials.
"Moderate-to-severe atopic dermatitis is a debilitating, life-altering disease with very limited treatment options. Many patients suffer for years with widespread inflamed skin, intense itch, sleep disturbances and other challenges," said Julie Block, chief executive officer, National Eczema Association. "We are thrilled to see the FDA recognizing the need to expedite and prioritise potential new options for these patients."
Breakthrough Therapy designation was created by the FDA to expedite the development and review of drugs that target serious or life-threatening conditions. A Breakthrough Therapy drug must show preliminary clinical evidence of a substantial improvement on a clinically significant endpoint over available therapies, or over placebo if there is no available therapy. The designation includes all of the Fast Track program features, as well as more intensive FDA guidance and discussion.
The Breakthrough Therapy designation is distinct from both accelerated approval and priority review, which can also be granted to the same drug if relevant criteria are met. A phase 3 worldwide clinical program for dupilumab in adults with moderate-to-severe atopic dermatitis is ongoing Dupilumab, a fully-human monoclonal antibody, is directed against the shared IL-4 receptor alpha subunit, which blocks signalling from both IL-4 and IL-13. IL-4 and IL-13 are key cytokines that are required for the initiation and maintenance of the Th2 (Type 2 helper T-cell) immune response, which is believed to be a critical pathway in allergic inflammation. Dupilumab was created using Regeneron's pioneering VelocImmune technology and is being co-developed with Sanofi in atopic dermatitis, asthma and chronic sinusitis with nasal polyposis. Dupilumab is an investigational agent under clinical development and its safety and efficacy have not been fully evaluated by any regulatory authority.
Moderate-to-severe atopic dermatitis, a serious form of eczema, is a chronic inflammatory disease characterized by an allergic response driven by Type 2 helper T cells. Moderate-to-severe forms of atopic dermatitis can be characterized by pronounced pruritus (itch), cutaneous dryness, and skin lesions marked by redness, infiltration/papulation, crusting/oozing, and lichenification (skin thickening), with periods of lesion exacerbation. Intense itching, scratching, and skin damage can lead to secondary infections. Atopic dermatitis is often associated with other inflammatory disorders such as asthma. Moderate-to-severe atopic dermatitis can negatively impact patients' lives and is associated with a high burden to society in terms of direct costs of medical care and prescription drugs and loss of productivity.