Akari Therapeutics Receives FDA Fast Track Designation for Nomacopan for Pediatric Hematopoietic Stem Cell Transplant-Related Thrombotic Microangiopathy (HSCT-TMA)
- Fast Track designation by the
U.S. Food and Drug Administration( FDA) facilitates the development and expedites the review of new medicines like nomacopan that treat serious unmet medical needs and in Akari’s case, pediatric patients with HSCT-TMA.
- The Fast Track designation follows a successful pre-investigational new drug (IND)
FDAmeeting earlier this year regarding Akari’s proposed pivotal clinical trial program for HSCT-TMA.
FDAis working with Akari in its optimization of pediatric dosing for HSCT-TMA by Akari’s participation in the FDA Model-Informed Drug Development (MIDD) Program with supportive PK data provided by pediatric patients already treated with nomacopan as part of a named patient program.
- Proposed pivotal trial of nomacopan in pediatric patients with HSCT-TMA expected to start Q4 2019 in
North Americaand Europe.
Fast Track designation is intended to facilitate the development and expedite the review of new drugs which show promise in treating serious or life-threatening conditions and address unmet medical needs. Drugs that receive this designation benefit from more frequent communications and meetings with
HSCT-TMA is an orphan condition with an estimated fatality rate of more than 80% in children with the severe disease. There are currently no approved treatments for HSCT-TMA but there is strong evidence for the role of complement and potentially LTB4 in the etiology of the disease.
Akari is a biopharmaceutical company focused on developing inhibitors of acute and chronic inflammation, specifically for the treatment of rare and orphan diseases, in particular those where the complement (C5) or leukotriene (LTB4) systems, or both complement and leukotrienes together, play a primary role in disease progression. Akari's lead drug candidate, nomacopan (formerly known as Coversin), is a C5 complement inhibitor that also independently and specifically inhibits leukotriene B4 (LTB4) activity. Nomacopan is currently being clinically evaluated in four indications: bullous pemphigoid (BP), atopic keratoconjunctivitis (AKC), thrombotic microangiopathy (TMA), and paroxysmal nocturnal hemoglobinuria (PNH). Akari believes that the dual action of nomacopan on both C5 and LTB4 may be beneficial in AKC, BP and other orphan inflammatory conditions. Akari is also developing other tick derived proteins, including longer acting versions.
Cautionary Note Regarding Forward-Looking Statements
Certain statements in this press release constitute “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control. Such risks and uncertainties for our company include, but are not limited to: needs for additional capital to fund our operations, our ability to continue as a going concern; uncertainties of cash flows and inability to meet working capital needs; an inability or delay in obtaining required regulatory approvals for nomacopan and any other product candidates, which may result in unexpected cost expenditures; our ability to obtain orphan drug designation in additional indications; risks inherent in drug development in general; uncertainties in obtaining successful clinical results for nomacopan and any other product candidates and unexpected costs that may result therefrom; difficulties enrolling patients in our clinical trials; failure to realize any value of nomacopan and any other product candidates developed and being developed in light of inherent risks and difficulties involved in successfully bringing product candidates to market; inability to develop new product candidates and support existing product candidates; the approval by the
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Source: Akari Therapeutics Plc