The UK’s Medicines & Healthcare Products Regulatory Agency Grants CEL-SCI a Pediatric Waiver, Bringing Multikine® One Step Closer to Future Marketing Clearance

Rhea-AI Summary

CEL-SCI (NYSE American: CVM) has received a pediatric waiver from the UK's Medicines & Healthcare Products Regulatory Agency (MHRA) for Multikine®, its treatment for head and neck cancer. This waiver exempts CEL-SCI from evaluating Multikine in patients under 18 years old as part of the license and marketing clearance review in the UK. CEO Geert Kersten stated that this decision accelerates the path to marketing approval in the UK, while the company prepares to start its confirmatory FDA Registration Study in the U.S. and other locations. The waiver is seen as a positive step towards future marketing clearance for Multikine.

Positive

• Granted pediatric waiver by UK's MHRA for Multikine
• Accelerates path to marketing approval in the UK
• Preparing to commence FDA Registration Study in the U.S. and elsewhere

Negative

• None.

Insights

Medical Research Analyst

The MHRA's pediatric waiver for Multikine® is a significant regulatory milestone for CEL-SCI. This decision streamlines the approval process by eliminating the need for pediatric studies, which are often complex and time-consuming. The waiver reflects the rarity of head and neck cancer in children, acknowledging that adult data may suffice for this population.

However, investors should note that this waiver is specific to the UK market and doesn't guarantee similar decisions in other jurisdictions. The impact on overall development timelines and costs may be , as the company still needs to complete its FDA Registration Study. While positive, this news doesn't directly address the efficacy or safety profile of Multikine, which remain the key determinants for ultimate approval and commercial success.

Regulatory Affairs Specialist

The MHRA's decision to grant a pediatric waiver for Multikine® is a strategic regulatory win for CEL-SCI. This waiver effectively reduces the regulatory burden and potentially accelerates the path to market in the UK. It's particularly noteworthy as it demonstrates the MHRA's recognition of the unique challenges in developing treatments for rare pediatric cancers.

However, it's important to understand that this waiver is market-specific. CEL-SCI will likely need to navigate similar processes with other regulatory bodies, including the FDA and EMA. While this decision is favorable, it doesn't guarantee regulatory success elsewhere. Investors should focus on the upcoming FDA Registration Study, as its results will be pivotal for Multikine's global regulatory prospects and commercial potential.

VIENNA, Va.--(BUSINESS WIRE)-- CEL-SCI Corporation (NYSE American: CVM) today reported it has received a decision letter from the United Kingdom’s Healthcare Products Regulatory Agency (MHRA) granting Multikine (Leukocyte Interleukin, Injection)* a product specific waiver for the treatment of head and neck cancer in a pediatric population of people up to 18 years of age. As a result, CEL-SCI will not be required to evaluate Multikine in a pediatric population as part of license and marketing clearance review in the UK.

“While we prepare to commence our confirmatory FDA Registration Study of Multikine here in the U.S., and elsewhere, we are very pleased to receive continued positive responses from the UK’s healthcare regulatory agency. This pediatric waiver accelerates our path to marketing approval in the UK,” stated CEL-SCI CEO Geert Kersten.

About CEL-SCI Corporation

CEL-SCI believes that boosting a patient’s immune system while it is still intact should provide the greatest possible impact on survival. Multikine is designed to help the immune system "target" the tumor at a time when the immune system is still relatively intact and thereby thought to be better able to mount an attack on the tumor.

Multikine (Leukocyte Interleukin, Injection), a true first-line cancer therapy, has been dosed in over 740 patients and received Orphan Drug designation from the FDA for neoadjuvant therapy in patients with squamous cell carcinoma (cancer) of the head and neck. Multikine significantly extended life in its target patient population demonstrating a 73% survival rate with Multikine vs. only 45% without Multikine at 5 years after treatment. Based on this very strong data, the FDA agreed to CEL-SCI’s target patient selection criteria and gave the go-ahead to conduct a small, focused, confirmatory Registration Study which will enroll 212 patients. CEL-SCI will enroll newly diagnosed locally advanced primary head and neck cancer patients who present with no lymph node involvement (determined via PET scan) and with low PD-L1 tumor expression (determined via biopsy), representing over 100,000 patients (globally) annually.

The Company has operations in Vienna, Virginia, and near/in Baltimore, Maryland.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. When used in this press release, the words "intends," "believes," "anticipated," "plans" and "expects," and similar expressions, are intended to identify forward-looking statements. Such statements are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Factors that could cause or contribute to such differences include an inability to duplicate the clinical results demonstrated in clinical studies, timely development of any potential products that can be shown to be safe and effective, receiving necessary regulatory approvals, difficulties in manufacturing any of the Company's potential products, inability to raise the necessary capital and the risk factors set forth from time to time in CEL-SCI's filings with the Securities and Exchange Commission, including but not limited to its report on Form 10-K for the year ended September 30, 2023. The Company undertakes no obligation to publicly release the result of any revision to these forward-looking statements which may be made to reflect the events or circumstances after the date hereof or to reflect the occurrence of unanticipated events.

* Multikine (Leukocyte Interleukin, Injection) is the trademark that CEL-SCI has registered for this investigational therapy. This proprietary name is subject to FDA review in connection with the Company's future anticipated regulatory submission for approval. Multikine has not been licensed or approved for sale, barter or exchange by the FDA or any other regulatory agency. Similarly, its safety or efficacy has not been established for any use.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240904760257/en/

Gavin de Windt

CEL-SCI Corporation

(703) 506-9460

Source: CEL-SCI Corporation

FAQ

What regulatory decision did CEL-SCI (CVM) receive for Multikine from the UK's MHRA?

CEL-SCI received a pediatric waiver from the UK's Medicines & Healthcare Products Regulatory Agency (MHRA) for Multikine, exempting the company from evaluating the treatment in patients under 18 years old for head and neck cancer.

How does the MHRA's decision impact CEL-SCI's (CVM) path to marketing approval for Multikine in the UK?

The pediatric waiver accelerates CEL-SCI's path to marketing approval for Multikine in the UK by eliminating the requirement to evaluate the treatment in a pediatric population as part of the license and marketing clearance review process.

What is CEL-SCI's (CVM) next step for Multikine's development in the United States?

CEL-SCI is preparing to commence a confirmatory FDA Registration Study for Multikine in the United States and other locations, as stated by CEO Geert Kersten.