Full Press Release Details
Akari Therapeutics Granted FDA Rare Pediatric
Disease Designation of Nomacopan for the Treatment of Pediatric HSCT-TMA
NEW YORK and LONDON, November 10, 2022 (GLOBE
NEWSWIRE) - Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage biotechnology company developing advanced therapies for autoimmune and
inflammatory diseases, today announced the U.S. Food and Drug Administration (FDA) has granted the Rare Pediatric Disease Designation
to nomacopan for the treatment of pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA). The Rare
Pediatric Disease Designation is an important addition to the Orphan Drug and Fast Track designations previously granted by the FDA for
nomacopan in pediatric HSCT-TMA.
"Families with children who have a hematopoietic
stem cell transplant-related TMA face grim prospects with high mortality rates and no approved treatment options," said Rachelle
Jacques, President and CEO of Akari. "The Rare Pediatric Disease Designation was created by the FDA because families need so much
more than hope for their children, they need meaningful treatment options. We are pleased the FDA has recognized the significant needs
that exist for children with HSCT-TMA, and we will continue to urgently pursue our mission to advance nomacopan to approval on their behalf."
The FDA Rare Pediatric Disease Designation and
Voucher Program is a recognition of the significant need that exists for approved treatments in rare pediatric diseases and is intended
to encourage development of these treatments. Under this program, a sponsor who receives an approval of a new drug application (NDA) or
biologics license application (BLA) for a rare pediatric disease may be eligible for a Priority Review Voucher (PRV). A PRV is valuable
because it can be redeemed to obtain priority review for a subsequent marketing application for a different product or may be sold to
About Akari Therapeutics
Akari Therapeutics, plc (Nasdaq: AKTX) is a biotechnology
company developing advanced therapies for autoimmune and inflammatory diseases. Akari's lead asset, investigational nomacopan, is a bispecific
recombinant inhibitor of complement C5 activation and leukotriene B4 (LTB4) activity. Akari's pipeline includes a Phase 3 clinical trial
program investigating nomacopan for severe pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA),
as well as pre-clinical research of long-acting PAS-nomacopan in geographic atrophy (GA). For more information about Akari, please visit
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 there; 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 FDA and EMA and any other similar foreign regulatory authorities of other
competing or superior products brought to market; risks resulting from unforeseen side effects; risk that the market for nomacopan may
not be as large as expected risks associated with the impact of the COVID-19 pandemic; inability to obtain, maintain and enforce patents
and other intellectual property rights or the unexpected costs associated with such enforcement or litigation; inability to obtain and
maintain commercial manufacturing arrangements with third party manufacturers or establish commercial scale manufacturing capabilities;
the inability to timely source adequate supply of our active pharmaceutical ingredients from third party manufacturers on whom the company
depends; unexpected cost increases and pricing pressures and risks and other risk factors detailed in our public filings with the U.S.
Securities and Exchange Commission, including our most recently filed Annual Report on Form 20-F filed with the SEC. Except as otherwise
noted, these forward-looking statements speak only as of the date of this press release and we undertake no obligation to update or revise
any of these statements to reflect events or circumstances occurring after this press release. We caution investors not to place considerable
reliance on the forward-looking statements contained in this press release.
For more information
Schleifstein PR (917) 763-8106