Kiadis Pharma Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for ATIR101™
September 20 2017 - 1:00AM
Business Wire
Regulatory News:
Kiadis Pharma N.V. (“Kiadis Pharma” or the “Company”)
(Euronext Amsterdam and Brussels: KDS), a clinical stage
biopharmaceutical company developing innovative products to make
bone marrow transplantations safer and more effective for patients
suffering from blood cancers and inherited blood disorders, today
announces that the US Food and Drug Administration (FDA) has
granted ATIR101™, Kiadis Pharma’s lead investigational product for
blood cancers, the Regenerative Medicine Advanced Therapy (RMAT)
designation.
Arthur Lahr, CEO of Kiadis Pharma, commented: “To receive
the RMAT designation from the FDA is an important milestone for
Kiadis Pharma and a recognition by the FDA of the significant
potential for ATIR101™ to help patients receive safer and more
effective bone marrow transplantations. RMAT is analogous to the
Breakthrough Therapy designation, and a clear validation of
ATIR101™ towards doctors and investors. We are now going to work
even closer with the FDA to agree a path to make this cell therapy
treatment available for patients in the US as soon as possible. In
Europe ATIR101™ was filed for registration in April 2017 and we
continue to prepare the Company for the potential European launch
in 2019.”
The RMAT pathway is analogous to the Breakthrough Therapy
designation designed for traditional drug candidates and medical
devices, and was specifically created by the US Congress in 2016 to
get important new cell therapy and advanced medicinal products to
the patient earlier. Just like the Breakthrough designation, it
allows companies developing regenerative medicine therapies to
interact with the FDA more frequently in the clinical testing
process, and RMAT-designated products may be eligible for priority
review and accelerated approval.
A regenerative medicine is eligible for the 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 drug has the potential to address unmet
medical needs for such a disease or condition.
For more information on RMAT designation, visit the FDA
website:https://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/ucm537670.htm).
FDA has already requested an additional meeting with Kiadis
Pharma and will now work closely with the Company to provide
guidance on the subsequent development of ATIR101™ for improved
overall survival and reduced transplant related mortality for
patients receiving a haploidentical hematopoietic stem cell
transplantation (HSCT).
About Kiadis Pharma
Kiadis Pharma is focused on cell-based immunotherapy products,
as an adjunctive to a haploidentical hematopoietic stem cell
transplantation (HSCT), for the treatment of blood cancers and
inherited blood disorders. The Company’s product candidates have
the potential to make allogeneic HSCT safer and more effective for
patients.
Based on the positive results from the single dose Phase II
trial with lead product ATIR101™ in patients with blood cancer, the
Company submitted a Marketing Authorization Application (MAA) to
the European Medicines Agency (EMA) in April 2017, for approval of
ATIR101™ across the European Union as an adjunctive treatment in
HSCT for malignant disease. In addition, Kiadis Pharma has received
regulatory approval in various countries to start dosing patients
in a Phase III trial with ATIR101™ that will be performed across
Europe and North America. ATIR101™ has been granted Orphan Drug
Designations both in the US and Europe.
The Company’s second product candidate, ATIR201™, will address
beta thalassemia, an inherited blood disorder.
Kiadis Pharma was granted an Advanced Therapy Medicinal Product
(ATMP) certificate for manufacturing quality and non-clinical data
by the EMA.
The Company’s shares are listed on Euronext Amsterdam and
Euronext Brussels.
Company website: www.kiadis.com
Forward Looking Statements
Certain statements, beliefs and opinions in this press release
are forward-looking, which reflect Kiadis Pharma’s or, as
appropriate, Kiadis Pharma’s directors’ current expectations and
projections about future events. By their nature, forward-looking
statements involve a number of risks, uncertainties and assumptions
that could cause actual results or events to differ materially from
those expressed or implied by the forward-looking statements. These
risks, uncertainties and assumptions could adversely affect the
outcome and financial effects of the plans and events described
herein. A multitude of factors including, but not limited to,
changes in demand, competition and technology, can cause actual
events, performance or results to differ significantly from any
anticipated development. Forward looking statements contained in
this press release regarding past trends or activities should not
be taken as a representation that such trends or activities will
continue in the future. As a result, Kiadis Pharma expressly
disclaims any obligation or undertaking to release any update or
revisions to any forward-looking statements in this press release
as a result of any change in expectations or any change in events,
conditions, assumptions or circumstances on which these
forward-looking statements are based. Neither Kiadis Pharma nor its
advisers or representatives nor any of its subsidiary undertakings
or any such person's officers or employees guarantees that the
assumptions underlying such forward-looking statements are free
from errors nor does either accept any responsibility for the
future accuracy of the forward-looking statements contained in this
press release or the actual occurrence of the forecasted
developments. You should not place undue reliance on
forward-looking statements, which speak only as of the date of this
press release.
View source
version on businesswire.com: http://www.businesswire.com/news/home/20170919006698/en/
Kiadis Pharma:Karl Hård, +31 611 096 298Head of IR &
Communicationsk.hard@kiadis.comorConsilium Strategic
Communications:Mary-Jane Elliott, Philippa Gardner, Lindsey
Neville, Hendrik Thys, +44 203 709
5708kiadis@consilium-comms.com