RARE Daily

FDA Grants Fast Track to Akari Bullous Pemphigoid Therapy

April 29, 2021

Rare Daily Staff

The U.S. Food and Drug Administration granted Fast Track designation to Akari Therapeutics for nomacopan, its experimental therapy for the treatment of patients with the rare autoimmune skin condition bullous pemphigoid.

Bullous pemphigoid (BP) is a severe orphan autoimmune inflammatory blistering skin disease with no approved treatments in the United States and Europe. It is a disease of the elderly that is primarily treated with steroids and immunosuppressants for six months or more, and can cause side effects and an approximately three-fold increase in mortality in the BP treated population. In BP patients there is evidence that both terminal complement activation (via complement component C5) and the lipid mediator leukotriene B4 (LTB4) have a central role in driving the disease.

Nomacopan (formerly known as Coversin), is a C5 complement inhibitor that also independently and specifically binds to and inhibits leukotriene B4 (LTB4) activity. Nomacopan has also been granted Orphan Drug designation for the treatment of BP by the FDA and the European Medicines Agency.

Fast track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. A drug that receives Fast Track designation benefits from more frequent communications and meetings with the FDA to review the drug’s development plan, including the design of the proposed clinical trials, use of biomarkers and the extent of data needed for approval.

The company said success in BP could potentially open up a range of other severe dermatological conditions for treatment with nomacopan where C5 and LTB4 are implicated, including hidradenitis suppurativa, epidermolysis bullosa acquisita, and mucous membrane pemphigoid.

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