Adaptimmune Announces U.S. FDA Acceptance of Biologics License Application for Afami-cel for the Treatment of Advanced Synovial Sarcoma with Priority Review
- The FDA has accepted Adaptimmune Therapeutics' BLA for afami-cel for priority review, indicating potential for approval.
- Afami-cel could be the first effective treatment option for synovial sarcoma in over a decade, addressing an unmet medical need.
- Clinical trials have demonstrated an impressive response rate of ~39% among heavily pre-treated patients with advanced synovial sarcoma.
- None.
Insights
The potential approval of afami-cel represents a significant advancement in oncological treatments, particularly for synovial sarcoma, a rare and aggressive form of cancer. Currently, treatment options are limited and the prognosis for advanced stages is typically poor. The introduction of an engineered T-cell therapy could offer a novel therapeutic approach, leveraging the body's own immune system to target and destroy cancer cells.
Engineered T-cell therapies, like afami-cel, involve modifying a patient's T-cells to recognize and attack cancer cells. This form of immunotherapy has been successful in treating certain blood cancers, but solid tumors present additional challenges due to their complex tumor microenvironments that can suppress immune responses. Afami-cel's progress into priority review suggests promising efficacy and safety data, which could lead to improved outcomes for patients with limited treatment options.
From a research perspective, the ~39% response rate reported in clinical trials for afami-cel is noteworthy, especially considering these are heavily pre-treated patients with advanced synovial sarcoma. Traditional second-line therapies for this cancer type have low objective response rates, which underscores the potential impact afami-cel could have on patient survival and quality of life. However, it is crucial to scrutinize the duration of response and any long-term survival benefits, as these are key indicators of the therapy's value.
Moreover, the economic implications of introducing a new therapy are substantial. The cost-effectiveness of afami-cel, in comparison to existing treatments, will be a critical factor for healthcare providers and payers. It is also important to consider the manufacturing complexity and scalability of T-cell therapies, which can influence the accessibility and price of the treatment for patients.
The acceptance of the Biologics License Application for afami-cel by the FDA is a pivotal event for Adaptimmune Therapeutics. Investors will watch closely as the PDUFA date approaches, as approval could lead to significant revenue generation for the company. If afami-cel becomes the first engineered T-cell therapy for solid tumors, it could capture a substantial market share due to the current unmet medical need.
Adaptimmune's statement about having the capabilities and capital to launch afami-cel post-approval is reassuring for stakeholders, indicating preparedness for commercialization. However, investors should consider the costs associated with bringing a new drug to market, potential competition and the broader implications of market adoption rates. It is also essential to analyze how the company plans to navigate the complex reimbursement landscape that often accompanies innovative but expensive therapies.
- If approved, afami-cel will be the first engineered T-cell therapy for solid tumors and the first effective treatment option for synovial sarcoma in more than a decade
Philadelphia, Pennsylvania and Oxford, United Kingdom--(Newsfile Corp. - January 31, 2024) - Adaptimmune Therapeutics plc (NASDAQ: ADAP), a company redefining the treatment of solid tumor cancers with cell therapy, today announced that the U.S. Food and Drug Administration (FDA) has accepted for priority review its Biologics License Application (BLA) for afami-cel, an investigational engineered T-cell therapy for advanced synovial sarcoma. The application has a Prescription Drug User Fee Act (PDUFA) target action date of August 4, 2024.
Adrian Rawcliffe, Adaptimmune's Chief Executive Officer: “The FDA’s acceptance of the BLA submission brings us one step closer to redefining treatment for people with synovial sarcoma. Our franchise has great potential and, if approved, we have the capabilities and the capital to launch afami-cel - the first engineered T-cell therapy on the market for a solid tumor cancer.”
Dennis Williams, PharmD, Senior VP of Late-Stage Development: "Historic outcomes are poor for advanced synovial sarcoma, with low objective response rates for second-line therapies and overall survival of less than 12 months for people who have received two or more prior lines of therapy. In clinical trials, afami-cel has demonstrated an impressive response rate of ~
This acceptance is supported by positive data from Cohort 1 of the pivotal trial SPEARHEAD-1, which met its primary endpoint for efficacy. Data from the trial were presented at the Connective Tissue Oncology Society (CTOS) 2023 Annual Meeting.
About Afami-cel
Afami-cel is an engineered T-cell receptor (TCR) T-cell therapy, targeted to the MAGE A4 cancer target, and designed as a single-dose treatment for advanced synovial sarcoma. The last FDA approved therapy for treatment in this setting was for Votrient in 2012. The BLA submission for afami-cel was supported by clinical data from the SPEARHEAD-1 pivotal trial, which has met its primary endpoint for efficacy. ~
About synovial sarcoma
There are more than 50 different types of soft tissue sarcomas which are categorised by tumors that appear in fat, muscle, nerves, fibrous tissues, blood vessels, or deep skin tissues.1 Synovial sarcoma accounts for approximately
1. https://www.cancer.org/cancer/types/soft-tissue-sarcoma/about/soft-tissue-sarcoma.html. 2. Synovial Sarcoma - NCI (cancer.gov). 3. Aytekin MN, et al. J Orthop Surg (Hong Kong). 2020;28(2).
About Adaptimmune
Adaptimmune is a cell therapy company out to redefine cancer treatment. With personalized medicines that radically improve the patient's experience with the therapy as much as the therapy itself, Adaptimmune is tackling difficult-to-treat solid tumor cancers so that patients and families may experience more unforgettable and important personal moments. The Company's unique engineered T-cell receptor (TCR) platform enables the engineering of T-cells to target and destroy cancers across multiple solid tumor types.
Forward-Looking Statements
This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 (PSLRA). These forward-looking statements involve certain risks and uncertainties. Such risks and uncertainties could cause our actual results to differ materially from those indicated by such forward-looking statements, and include, without limitation: the success, cost and timing of our product development activities and clinical trials and our ability to successfully advance our TCR therapeutic candidates through the regulatory and commercialization processes. For a further description of the risks and uncertainties that could cause our actual results to differ materially from those expressed in these forward-looking statements, as well as risks relating to our business in general, we refer you to our Annual Report on Form 10-K filed with the Securities and Exchange Commission for the year ended December 31, 2022, our Quarterly Reports on Form 10-Q, Current Reports on Form 8-K, and other filings with the Securities and Exchange Commission. The forward-looking statements contained in this press release speak only as of the date the statements were made and we do not undertake any obligation to update such forward-looking statements to reflect subsequent events or circumstances.
Adaptimmune Contacts
Investor Relations
Juli P. Miller, Ph.D., VP, Corporate Affairs and Investor Relations
T: +1 215 825 9310
M: +1 215 460 8920
Juli.Miller@adaptimmune.com
Media Relations
Dana Lynch, Senior Director of Corporate Communications
M: +1 267 990 1217
Dana.Lynch@adaptimmune.com
To view the source version of this press release, please visit https://www.newsfilecorp.com/release/196074
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