XORTX Submits Orphan Drug Designation Application to the European Medicines Agency (EMA) to Treat Progressive Kidney Disease
29 Agosto 2023 - 8:00AM
XORTX Therapeutics Inc. (“
XORTX” or the
“
Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt:
ANU), a late-stage clinical pharmaceutical company focused on
developing innovative therapies to treat progressive kidney
disease, announces that it has submitted an Orphan Drug Designation
application for XORLO™ to the European Medicines Agency (the
“EMA”). The “orphan-drug designation request is for the use of
XORTX’s patented unique proprietary formulation of oxypurinol –
XORLO™ – for the treatment of autosomal dominant polycystic
kidney disease (ADPKD)”.
The orphan drug designation process (“ODD”)
initiated with the submission of this application and is made to
the EMA’s COMP (Committee for Orphan Medicinal Products) office. To
support this application, a focused data package was provided that
included: a review of the basic science related to the mechanism of
injury associated with aberrant purine metabolism and hyperuricemia
as well as the evidence that XORLO™ attenuates the
accelerating effect of kidney injury, analysis of the number of
European patients with ADPKD and arguments to support the
likelihood that the new therapy, XORLO™, will provide significant,
clinically meaningful benefit compared with existing treatment. The
EMA’s COMP office, will review this initial application package and
provide feedback and a decision, which is expected in December of
this year.
Dr. Allen Davidoff, CEO of XORTX, stated, “This
EMA ODD submission represents a key milestone for the Company
regarding new and existing discoveries made by XORTX and its novel
approach to slowing progression of kidney disease in ADPKD. It also
follows on receipt of ODD status granted by the U.S. Food and Drug
Administration in April 2023. Further updates will be provided once
the EMA COMP renders its decision.”
Benefits of EMA Orphan Designation
include: Reduced fees for protocol assistance, market
authorization applications and annual fees for authorized
medicines; Automatic access to centralized procedure for EMA
marketing authorization, access to research grants, a simplified
approval process and 10 years of market exclusivity. Further
information regarding the incentives for orphan designation are
available at:
https://www.ema.europa.eu/en/human-regulatory/research-development/orphan-designation/orphan-incentives
About Orphan Drug Designation in Europe
The EMA is responsible for reviewing
applications from sponsors for orphan designation. To qualify
for orphan designation, a medicine must meet a number
of criteria:
- it must be intended for the
treatment, prevention or diagnosis of a disease that
is life-threatening or chronically
debilitating;
- the prevalence of the
condition in the EU must not be more than five in 10,000 or it must
be unlikely that marketing of the medicine would generate
sufficient returns to justify the investment needed for its
development;
- no satisfactory method of
diagnosis, prevention or treatment of the condition concerned can
be authorized, or, if such a method exists, the medicine must be
of significant benefit to those affected by the
condition.
Applications for orphan
designation are examined by the EMA’s COMP office using the
network of experts that the EMA’s COMP office has built up. The
evaluation process takes a maximum of 90 days from validation.
https://www.ema.europa.eu/en/human-regulatory/overview/orphan-designation-overview
About the XRx-008 Program
Oxypurinol is a xanthine oxidase inhibitor
(“XOI”) with important pharmacologic
characteristics ideal for administration to individuals with ADPKD.
Key pharmacologic attributes include:
1/ The ability to act in the circulation, kidney
and cardiovascular tissue and inhibit the production of uric acid
thereby attenuate the related mechanism of injury whereby xanthine
oxidase accelerates the progression of renal and cardiac
diseases.
2/ XORLO™ provides substantially increased
absorption of oxypurinol. This approach provides an effective, well
tolerated drug with an extensive clinical safety experience
suggesting the Company’s XRx-008 program has the capacity to
provide an XOI with a superior product profile indicated to slow
the accelerating decline in kidney function in ADPKD patients.
About ADPKD
ADPKD is a rare disease that affects more that
10 million individuals worldwide.1,2 ADPKD is typically diagnosed
based upon expansion of fluid-filled cysts in the kidneys. Over
time, the increasing number and size of cysts can contribute to
structural and functional changes to kidneys and is frequently
accompanied by chronic pain which is a common problem for patients
with ADPKD.3 Expansion of cysts is thought to compress healthy
functioning tissue surrounding the cysts and contribute to further
loss of kidney function, fibrosis, impaired nutrient exchange and
impaired kidney function, leading ultimately to end-stage renal
disease.1 For individuals with progressing ADPKD, treatment
recommendations include anti-hypertensive treatment, dietary
restrictions, and, for a limited percentage of suitable patients
who can tolerated ADPKD specific pharmacotherapy.4 New, more
broadly applicable therapies to effectively slow decline of kidney
function in ADPKD are needed.
About XORTX Therapeutics Inc.
XORTX is a pharmaceutical company with two
clinically advanced products in development: 1) our lead, XRx-008
program for ADPKD; and 2) our secondary program in XRx-101 for
acute kidney and other acute organ injury associated with
Respiratory Viral infection. In addition, XRx-225 is a pre-clinical
stage program for Type 2 Diabetic Nephropathy. XORTX is working to
advance its clinical development stage products that target
aberrant purine metabolism and xanthine oxidase to decrease or
inhibit production of uric acid. At XORTX, we are dedicated to
developing medications to improve the quality of life and future
health of patients with kidney disease. Additional information on
XORTX is available at www.xortx.com.
For more
information, please contact: |
|
|
|
Allen Davidoff, CEO |
Nick Rigopulos, Director of Communications |
adavidoff@xortx.com or +1 403 455 7727 |
nick@alpineequityadv.com or +1 617 901 0785 |
|
|
Media Inquiries, OIipriya Das, PhD, MSc |
|
olipriya.das@russopartnersllc.com or +1 409 365 3641 |
|
|
|
References:
- Wiley C., Kamat S., Stelhorn R.,
Blais J., Analysis of nationwide date to determine the incidence
and diagnosis of autosomal dominant polycystic kidney disease in
the USA, Kidney Disease, 5(2): 107-117, 2019
- Bergmann C., Guay-Woodford L.M.,
Harris P.C., Horie S., Peters D.J., Torres V.E., Polycystic Kidney
Disease, Nat Rev Dis Primers. 4(1): 50, 2018
-
https://pkdcure.org/living-with-pkd/chronic-pain-management/
- Gimpel C., Bergmann C., Bockenhauer
D., et al., International consensus statement of the diagnosis and
management of autosomal dominant polycystic kidney disease in
children and young people, Nat Rev Nephrol 15(11):713-726,
2019
Neither the TSX Venture Exchange nor Nasdaq has
approved or disapproved the contents of this news release. No stock
exchange, securities commission or other regulatory authority has
approved or disapproved the information contained herein.
Forward Looking Statements
This press release
contains express or implied forward-looking statements pursuant to
U.S. Federal securities laws. These forward-looking statements and
their implications are based on the current expectations of the
management of XORTX only, and are subject to a number of factors
and uncertainties that could cause actual results to differ
materially from those described in the forward-looking statements.
Except as otherwise required by law, XORTX undertakes no obligation
to publicly release any revisions to these forward-looking
statements to reflect events or circumstances after the date hereof
or to reflect the occurrence of unanticipated events. More detailed
information about the risks and uncertainties affecting XORTX is
contained under the heading “Risk Factors” in XORTX’s Registration
Statement on Form F-1 filed with the SEC, which is available on the
SEC’s website, www.sec.gov (including any documents forming a part
thereof or incorporated by reference therein), as well as in our
reports, public disclosure documents and other filings with the
securities commissions and other regulatory bodies in Canada, which
are available on www.sedarplus.ca.
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