Back to Newsroom
Back to Newsroom

Deverra Therapeutics Granted FDA Regenerative Medicine Advanced Therapy and Orphan Drug Designations for DVX101 (Dilanubicel) for the Treatment of Acute Myeloid Leukemia

Tuesday, 19 March 2024 06:00 AM

Designations Validate Preliminary Data and Enhances Ability to Offer Dilanubicel to Cancer Patients

SEATTLE, WA / ACCESSWIRE / March 19, 2024 / Deverra Therapeutics Inc., a clinical-stage biotechnology company developing allogeneic universal donor cell therapy treatments to fight cancer and other life-threatening diseases, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT)designation to its lead candidate (dilanubicel) for use in first-line treatment for acute myeloid leukemia (AML).

The FDA Center for Biologics Evaluation and Research (CBER) granted RMAT designation based on the potential of DVX101 (dilanubicel) to address the significant unmet medical need in treatment of patients with newly diagnosed AML, where the 5-year overall survival rate remains less than 30%.

Dilanubicel was recently granted orphan drug designation (ODD) for the treatment of AML. The FDA, through its Office of Orphan Products Development (OOPD), grants orphan drug designation to drugs and biologics intended for treatment, prevention or diagnosis of a rare disease or condition that affects fewer than 200,000 people in the U.S. under the Orphan Drug Act. Orphan drug status provides incentives, including tax credits, user fee exemptions and waiver of certain administrative fees for clinical trials, and seven years of market exclusivity after approval.

"We are thrilled to receive both RMAT and ODD for our lead candidate, dilanubicel, for the treatment of AML," said Dr. Colleen Delaney, MD, MSc, Chief Science Officer, EVP of Research and Development at Deverra Therapeutics. "Achieving these statuses for dilanubicel provides an added level of collaboration and commitment from the FDA, enhancing our efforts to offer this allogeneic off-the-shelf cell therapy to cancer patients by enabling real-time discussions on patient populations, trial design, product quality, and manufacturing. These designations validate the preliminary data and our belief in the potential of dilanubicel as a first-in-class, immediately available allogeneic cell therapy to address an urgent unmet need in the treatment of AML. Our goal is to improve early remission rates in patients with newly diagnosed AML undergoing standard of care chemotherapy which is critical to survival and quality of life. We look forward to working closely with the FDA under the RMAT and ODD designation programs on the continued development of dilanubicel for AML and to one day provide accessible, affordable, and potentially life-saving allogeneic cell therapies for all AML patients in need."

About Regenerative Medicine Advanced Therapy (RMAT)
Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the development and review processes for promising regenerative medicine/cell and gene therapies. An investigational cell therapy is eligible for RMAT designation if it is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the therapy has the potential to address unmet medical needs for the disease or condition. Advantages of the RMAT designation include the benefits of the fast track and breakthrough therapy designation programs, including but not limited to early sponsor interactions with the FDA and intensive guidance from the agency on efficient and expedited drug development. Importantly, this includes the ability to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, and priority review of the biologics license application (BLA). Per FDA's website, since the inception of the RMAT program in December 2016, the total number of RMAT requests received by the FDA is 248, with 101 of those requests (41%) being granted RMAT designation to date.

About DVX101 (Dilanubicel) for AML
DVX101 (dilanubicel), a universal donor, ex vivo expanded hematopoietic stem and progenitor cell product, is being developed as an off-the-shelf, universal, cryopreserved product for the treatment of AML. Preliminary clinical efficacy studies demonstrate statistical significance in complete remission (CR) rates (defined as CR and CR with incomplete count recovery, CRi) in subjects receiving dilanubicel with standard of care chemotherapy compared to the control arm (chemotherapy alone). It also shows that infusion of dilanubicel was well tolerated and did not increase toxicity compared to the control arm.

For patients with newly diagnosed AML, achieving CR after initial standard of care chemotherapy is an unmet medical need, especially for those with intermediate and high risk disease. Furthermore, achieving CR is critical to survival, has been correlated with improved event-free and overall survival, and is necessary to gain access to definitive therapies such as allogeneic stem cell transplant.

The severity and life-threatening nature of AML underscores the importance of early detection and prompt, aggressive treatment. Despite the significant advances in medical treatments, AML remains a serious health threat that requires more effective therapies and improved survival rates. Preliminary clinical evidence demonstrates that dilanubicel has the potential to address this unmet medical need and results in improved efficacy over the current upfront standard of care while maintaining an acceptable safety profile.

About Acute Myeloid Leukemia (AML)
AML is an extremely aggressive hematological cancer that, if left untreated, progresses rapidly and is life-threatening, with fatal consequences generally within a few months from diagnosis. Even with appropriate treatment, AML is associated with a high mortality rate secondary to high morbidity and high rate of post-remission disease recurrence with standard-of-care treatment, making it a serious health concern. The incidence of AML increases with age, typically affecting people 60 years old and older, but it can affect people of all ages. With current therapies, the 5-year relative survival rate for people 20 and older with AML is less than 30 percent.

About Deverra Therapeutics, Inc.
Deverra Therapeutics, Inc. is a clinical stage biotechnology company developing allogeneic, off-the-shelf universal donor cellular immunotherapies for on-demand treatment of patients with cancer and infectious diseases. Deverra Therapeutics has exclusive license to the proprietary Notch-mediated stem cell expansion and directed differentiation platform from the Fred Hutchinson Cancer Center based on more than 20 years of federally funded research and multiple clinical trials. Deverra Therapeutics' innovative and well-established platform can be used to generate a range of unmodified and modified (engineered) progenitor and immune cells to boost the patient's blood and immune system to eliminate cancer and virally infected cells. Deverra Therapeutics currently has three clinical trials, with one pending (dilanubicel) for upfront therapy for newly diagnosed Acute Myeloid Leukemia (AML) patients, and two out-licensed (DVX201-COV-01 & DVX201-AML-01) to Coeptis Therapeutics that are utilizing an unmodified NK cell therapy in COVID-19 and in AML and Myelodysplastic Syndrome (MDS). For more information on the Company, please visit

Colleen Delaney, MD, MSc
Scientific Founder, CSO and EVP of R&D
Deverra Therapeutics, Inc.
[email protected]

Media and Investor Contact:
Alpha IR Group
Jackie Marcus or Josh Carroll
[email protected]

SOURCE: Deverra Therapeutics Inc.

Company Update
Back to newsroom
Back to Newsroom
Share by: