ABIONYX Pharma Receives FDA Orphan Drug Designation (ODD) for CER-001 for the Treatment of LCAT Deficiency Presenting as Kidney Dysfunction and/or Ophthalmologic Disease
29 Mars 2022 - 6:34PM
Business Wire
- ODD follows positive results in two compassionate use
cases
- Published data from compassionate use cases demonstrated for
the first time that bio-HDL therapy can reduce lipid deposits in
the kidney, slow the decline of kidney function while eliminating
the need for dialysis, beneficially remodel lipoproteins, and
improve visual impairment due to corneal lipid deposits
- Orphan designation provides a new strategy for clinical
development of bio-HDL in kidney diseases and ophthalmologic
diseases in the US
Regulatory News:
ABIONYX Pharma (FR0012616852 – ABNX – PEA PME
eligible) (Paris:ABNX), a new generation biotech company
dedicated to the discovery and development of innovative therapies
for patients, today announced that the U.S. Food and Drug
Administration (FDA) has granted orphan-drug designation (ODD) to
the Bio-HDL CER-001 for the treatment of lecithin-cholesterol
acyltransferase (LCAT) deficiency. The designation covers both
partial LCAT deficiency, presenting as Fisheye Disease, and
complete LCAT deficiency presenting with renal symptoms and corneal
opacities. Progression of LCAT deficiency, for which there is no
approved treatment, can ultimately lead to renal failure requiring
dialysis or kidney transplant, and/or to complete corneal
opacification requiring transplant.
The European Medicines Administration (EMA) granted ODD status
to CER-001 for the treatment of LCATdeficiency in July 2021.
Positive clinical results from CER-001 in LCAT disease have
previously been published. In the Annals of Internal Medicine in
March 2021, a case study of a patient who was about to undergo
dialysis due to the rapid decline in renal function was described.
The patient was able to avoid the need for dialysis during her
treatment with CER-001 and in addition, lipid deposits in her
corneas which had caused significant visual blurring, improved with
treatment. The improvement in visual function was still observed
after 1 year of follow-up. A second case was described in the
Journal of Internal Medicine in November 2021 and showed that
CER-001 reduced glomerular lipid deposits and slowed the patient’s
decline in renal function. Furthermore, CER-001 remodeled his
plasma lipoproteins by reducing the level of LpX, large abnormal
lipid complexes known to be renally toxic.
“We are pleased to have received ODD for CER-001 just eight
months after the ODD in Europe and one week after the first
positive clinical results in COVID-19. These OD designations from
the FDA underscore the importance of bringing this important
therapeutic option to patients with LCAT Deficiency both as a
kidney disease and as an ophthalmic disease,” commented Cyrille
TUPIN, CEO of ABIONYX Pharma. “We look forward to presenting new
clinical results in the coming months and providing additional
insight into the potential of our Bio-HDL therapy platform in both
kidney diseases and ophthalmologic diseases. The ODD paves the way
for ABIONYX Pharma to launch a new strategic clinical development
of the bio-HDL in kidney diseases and ophthalmologic diseases in
the US.”
Orphan-drug designation is granted by the FDA to a drug or
biologic intended to treat a rare disease or condition, which
generally includes a disease or condition that affects fewer than
200,000 individuals in the U.S. Supporting the development and
evaluation of new treatments for rare diseases is a key priority
for the FDA. The designation is granted based on the mechanism of
action of the drug or biologic taken into consideration with the
pathogenesis of the disease or condition, its course and prognosis
as well as the availability of treatments and/or resistance to
available treatments.
Orphan drug designation qualifies sponsors for incentives
including: tax credits for qualified clinical trials, exemption
from user fees, and a potential for seven years of market
exclusivity after approval.
Next financial press release :
Annual Results, April 28th 2022
About the Bio-HDL CER-001
CER-001 is the first-in-class bio-HDL mimetic that directly
targets a key underlying metabolic defect of LCAT deficiency. The
bio-HDL is one of the most advanced biomedicines and is a potential
novel treatment for kidney diseases, sepsis or COVID-19, but also
for ophthalmologic diseases involving lipid abnormalities. These
abnormalities could be modified by pharmacological agents that
increase plasma ApoA-I and HDL levels, but more importantly
increase the number of functional HDL. The antiinflammatory
properties and/or the increase in the Reverse Cholesterol Transport
(RCT) of CER-001 can prevent decline in kidney function and improve
vision in LCAT patients. Bio-HDL as a biomedicine was found to be
completely safe and very well tolerated by more than 600 patients
involved in all previous clinical studies.
About ABIONYX Pharma
ABIONYX Pharma is a new generation biotech company that aims to
contribute to health through innovative therapies in indications
where there is no effective or existing treatment, even the rarest
ones. Thanks to its partners in research, medicine,
biopharmaceuticals and shareholding, the company innovates on a
daily basis to propose drugs for the treatment of renal and
ophthalmological diseases, or new HDL vectors used for targeted
drug delivery.
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NewCap Investor relations Louis-Victor Delouvrier
abionyx@newcap.eu +33 (0)1 44 71 98 53
NewCap Media relations Arthur Rouillé abionyx@newcap.eu
+33 (0)1 44 71 00 15
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