ExCellThera Announces EMA’s Acceptance under Accelerated Assessment of Market Authorisation Application (MAA) for UM171 Cell Therapy for Patients with Hematological Malignancies who Lack a Readily Available Suitable Donor
25 June 2024 - 12:00PM
ExCellThera Inc. (ExCellThera), a world leader in enhanced blood
stem cell expansion and therapies, announced today that the Market
Authorisation Application (MAA) for UM171 Cell Therapy
(INN-dorocubicel) has been accepted under an accelerated assessment
procedure by the European Medicines Agency (EMA), for adult
patients with hematological malignancies requiring a stem cell
transplant who lack a readily available suitable donor.
The EMA’s Committee for Medicinal Products for
Human Use (CHMP) granted UM171 Cell Therapy accelerated assessment
as it was deemed of major interest from a public health point of
view. Accelerated assessment aims to reduce the timeframe for the
CHMP to review a MAA compared to the standard procedure and follows
the EMA granting Orphan Medicinal Product designation and access to
the Priority Medicines scheme (PRIME) in 2020.
“We are excited that the EMA has accepted this
regulatory submission under an accelerated assessment procedure, as
UM171 Cell Therapy has immense potential to help address a high
unmet medical need for patients with hematological malignancies who
require a stem cell transplant but lack a readily available
suitable donor,” said David Millette, CEO of ExCellThera. “We now
look forward to working closely with the EMA in the coming months
to bring this therapy to the patients who need it.”
About UM171 Cell Therapy
UM171 Cell Therapy (INN-dorocubicel), developed
by Cordex Biologics, a wholly owned subsidiary of ExCellThera, has
been evaluated in 120 patients to treat hematologic malignancies in
clinical trials in the United States, Europe and Canada. UM171 Cell
Therapy has received orphan drug designation and regenerative
medicine advanced therapy (RMAT) designations from the FDA as well
as orphan medicinal product designation, advanced therapy medicinal
product (ATMP) classification and priority medicines (PRIME)
designation from the EMA.
UM171 Cell Therapy has successfully completed
Phase 2 trials in patients with high and very high-risk acute
leukemias and myelodysplasias who have limited treatment options
with low survival outcomes and high incidence of relapse under the
current standard of care, including patients with refractory or
active disease, patients requiring a second transplant, and
patients with TP53 mutations or other genetic abnormalities. The
initiation of a Phase 3 trial in this patient population is planned
for the second half of 2024.
The use of UM171 Cell Therapy in other patient
populations, including patients with multiple myeloma, pediatric
patients, and patients with non-malignant hematological diseases,
is also being explored.
UM171 Cell Therapy is an investigational cell
therapy, and its safety and efficacy have not been established by
the FDA, EMA, Health Canada or any other health authority.
About ExCellThera and UM171
Technology
ExCellThera is a world leader in enhanced blood
stem cell expansion. ExCellThera’s proprietary EnhanceTM platform
for cell expansion and metabolic fitness is designed to deliver a
greater dose of functional therapeutic stem cells by expanding HSCs
from any source and counteracting the effects of culture or gene
editing induced stress. ExCellThera partners with biopharmas to
help them develop best-in-class cell and gene therapies by
leveraging the technologies that form the EnhanceTM platform,
including the proprietary molecule UM171 which has a first- and
best-in-class mechanism of action for ex vivo expansion and
metabolic fitness of HSCs. For additional information, visit
excellthera.com, and follow us on LinkedIn.
Investor and Media Contact
Jean Proulx
jean.proulx@excellthera.com