FDA Grants Rare Pediatric Disease Designation to Omeros’ MASP-3 Inhibitor Zaltenibart for Treatment of C3 Glomerulopathy

Rhea-AI Summary

Omeros announced that its MASP-3 inhibitor zaltenibart (OMS906) received rare pediatric disease designation from the FDA for treating C3 glomerulopathy (C3G), an ultra-rare renal disorder affecting children and young adults. The company plans to initiate Phase 3 trials for C3G in 2024. Zaltenibart is also being developed for paroxysmal nocturnal hemoglobinuria (PNH), with Phase 3 studies starting this quarter. The PNH market was valued at $3.9 billion in 2023 and is projected to reach $10.1 billion by 2032. If approved, Omeros will receive a priority review voucher that can accelerate FDA review of future applications.

Positive

• Received FDA rare pediatric disease designation for zaltenibart in C3G treatment
• Planned Phase 3 trials for both C3G and PNH indications
• Potential access to valuable priority review voucher upon approval
• Targeting PNH market projected to reach $10.1 billion by 2032
• Clear regulatory pathway established with FDA and European authorities

Negative

• No current FDA-approved treatments for C3G, indicating potential regulatory challenges
• Product still in development phase with no guaranteed approval
• Competing in established market with existing alternative pathway inhibitors

Insights

Medical Research Analyst

The FDA's rare pediatric disease designation for zaltenibart represents a significant milestone for Omeros. The designation not only validates the drug's potential in treating C3G but also comes with a valuable priority review voucher upon approval. These vouchers have historically sold for $100-200 million, providing substantial potential value.

The dual-indication strategy targeting both C3G and PNH positions zaltenibart strongly in the complement inhibitor market. With PNH market projections reaching $10.1 billion by 2032 and no approved treatments for C3G, the commercial opportunity is substantial. The MASP-3 inhibition mechanism offers a unique advantage by targeting the alternative pathway more proximally than existing treatments.

The planned Phase 3 trials in both indications mark a critical transition from development to potential commercialization. The regulatory clarity from FDA and European authorities particularly strengthens the PNH program's advancement timeline.

Market Research Analyst

This development significantly enhances Omeros' market position in rare diseases. The potential priority review voucher represents a valuable asset, either for accelerating future drug approvals or generating immediate capital through sale. The C3G market opportunity, while smaller due to its ultra-rare status, offers pricing power due to the lack of approved treatments.

The PNH program's progression to Phase 3 this quarter positions Omeros to compete in a rapidly growing market dominated by established players. The projected market growth from $3.9 billion to $10.1 billion suggests strong pricing power and expanding patient populations. The dual-indication strategy could create synergies in commercialization and market penetration efforts.

-- Phase 3 Studies on the Horizon in both PNH and C3G --

SEATTLE--(BUSINESS WIRE)--

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 are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “likely,” “look forward to,” “may,” “objective,” “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.

View source 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

Source: Omeros Corporation

FAQ

What did the FDA grant Omeros (OMER) for zaltenibart in 2023?

The FDA granted rare pediatric disease designation to Omeros' zaltenibart for the treatment of C3 glomerulopathy (C3G).

When will Omeros (OMER) begin Phase 3 trials for zaltenibart in PNH?

Omeros plans to begin Phase 3 trials for zaltenibart in PNH later this quarter.

What is the projected market size for PNH treatment by 2032 for Omeros (OMER)?

The PNH treatment market is projected to grow from $3.9 billion in 2023 to $10.1 billion by 2032.

What benefit does the rare pediatric disease designation provide to Omeros (OMER)?

The designation provides Omeros with a priority review voucher upon drug approval, which can accelerate FDA review of future applications by at least four months.