FDA Grants Rare Pediatric Disease Designation to Omeros’ MASP-3 Inhibitor Zaltenibart for Treatment of C3 Glomerulopathy
October 24 2024 - 8:45AM
Business Wire
-- Phase 3 Studies on the Horizon in
both PNH and C3G --
Omeros Corporation today announced that
zaltenibart (OMS906) has received rare pediatric disease
designation from the U.S. Food and Drug Administration (FDA) for
the treatment of complement 3 glomerulopathy (C3G), an ultra-rare,
progressive renal disorder primarily afflicting children and young
adults. Caused by dysregulation of the alternative pathway of
complement, there is no approved treatment for C3G, which often
leads to end-stage renal disease within 10 years of diagnosis.
Zaltenibart is the most proximal inhibitor of the alternative
pathway. It blocks mannan-binding lectin-associated serine
protease-3 (MASP-3), the key activator of the alternative pathway,
stopping the conversion of pro-complement factor D (pro-CFD) to
mature CFD. Phase 3 clinical trials for zaltenibart in C3G are
slated to begin next year.
“C3G is devastating for children as well as for adults, and our
receipt of FDA’s rare pediatric disease designation is a welcome
acknowledgment of zaltenibart as a potential therapeutic for this
disease that has no approved treatment,” stated Gregory A
Demopulos, chairman and CEO of Omeros. “With zaltenibart clinical
studies ongoing in both PNH and C3G and preparations underway to
begin Phase 3 trials, we look forward to bringing zaltenibart to
market, expanding its list of targeted indications and
demonstrating its advantages over other alternative pathway
inhibitors.”
Companies awarded a rare pediatric disease designation receive a
rare pediatric disease priority review voucher from FDA when the
designated drug is approved for the associated indication in the
pediatric population. The voucher allows the recipient company to
obtain FDA priority review of either a New Drug Application (NDA)
or Biologics License Application (BLA) for a different product
and/or indication, reducing the review time and accelerating any
granted approval and subsequent market entry by at least four
months. The voucher may be used by the original recipient, or it
can be sold to another company for the purchaser’s use.
Omeros is also advancing zaltenibart for the treatment of
paroxysmal nocturnal hemoglobinuria (PNH), an ultra-rare and
life-threatening blood disease. Omeros has received orphan drug
designation from FDA for zaltenitbart in this indication. Having
recently held productive meetings with regulatory authorities – an
end-of-phase 2 meeting with FDA and a scientific advice meeting in
Europe – Omeros has a clear path to and is focused on initiating
phase 3 studies for zaltenibart in PNH later this quarter. In 2023,
the PNH treatment market size was $3.9 billion and is projected to
grow to $10.1 billion in 2032.
About Omeros Corporation
Omeros is an innovative biopharmaceutical company committed to
discovering, developing and commercializing small-molecule and
protein therapeutics for large-market and orphan indications
targeting immunologic disorders, including complement-mediated
diseases and cancers, as well as addictive and compulsive
disorders. Omeros’ lead MASP-2 inhibitor narsoplimab targets the
lectin pathway of complement and is the subject of a biologics
license application pending before FDA for the treatment of
hematopoietic stem cell transplant-associated thrombotic
microangiopathy. Omeros’ long-acting MASP-2 inhibitor OMS1029 has
successfully completed Phase 1 single- and multiple-ascending dose
clinical studies. OMS906, Omeros’ inhibitor of MASP-3, the key
activator of the alternative pathway of complement, is advancing
toward Phase 3 clinical trials for paroxysmal nocturnal
hemoglobinuria and complement 3 glomerulopathy. Funded by the
National Institute on Drug Abuse, Omeros’ lead phosphodiesterase 7
inhibitor OMS527 is in clinical development for the treatment of
cocaine use disorder. Omeros also is advancing a broad portfolio of
five novel cellular and molecular immuno-oncology programs. For
more information about Omeros and its programs, visit
www.omeros.com.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of Section 27A of the Securities Act of 1933 and
Section 21E of the Securities Exchange Act of 1934, which are
subject to the “safe harbor” created by those sections for such
statements. All statements other than statements of historical fact
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“plan,” “potential,” “predict,” “project,” “should,” “slate,”
“target,” “will,” “would” and similar expressions and variations
thereof. Forward-looking statements, including statements regarding
the timing and anticipated outcome of regulatory events or
processes, the availability of clinical trial data, the prospects
for obtaining FDA approval of any drug candidate or in any
indication, expectations regarding the initiation, continuation or
results of clinical trials evaluating Omeros’ drug candidates,
expectations regarding future cash expenditures, and expectations
regarding the sufficiency of our capital resources to fund
operations, are based on management’s beliefs and assumptions and
on information available to management only as of the date of this
press release. Omeros’ actual results could differ materially from
those anticipated in these forward-looking statements for many
reasons, including, without limitation, unanticipated or unexpected
outcomes of regulatory processes in relevant jurisdictions,
unproven preclinical and clinical development activities, failure
by Congress to reauthorize the priority review voucher program or
other legislative developments, our financial condition and results
of operations, any inability to obtain capital needed to fund
planned operations, regulatory processes and oversight, challenges
associated with manufacture or supply of our investigational or
clinical products, changes in reimbursement and payment policies by
government and commercial payers or the application of such
policies, intellectual property claims, competitive developments,
litigation, and the risks, uncertainties and other factors
described under the heading “Risk Factors” in our Annual Report on
Form 10-K filed with the Securities and Exchange Commission on
April 1, 2024 and in our subsequently filed Quarterly Reports on
Form 10-Q. Given these risks, uncertainties and other factors, you
should not place undue reliance on these forward-looking
statements, and we assume no obligation to update these
forward-looking statements, whether as a result of new information,
future events or otherwise, except as required by applicable
law.
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version on businesswire.com: https://www.businesswire.com/news/home/20241024637899/en/
Jennifer Cook Williams Cook Williams Communications, Inc.
Investor and Media Relations IR@omeros.com
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