ABIONYX Pharma Announces a New Compassionate Access Authorization (CAA) for CER-001 in the Rare Disease LCAT Deficiency or Norum Disease in Europe
10 Julho 2023 - 1:00PM
Business Wire
- 4th CAA patient in Europe in the rare disease LCAT
deficiency also known as Norum disease
- Scientific consistency and coherence of clinical data from
first 3 patients
- Treatment safety and tolerability data as positive as
ever
- Constructive discussions with European regulatory
authorities to define a fast-track clinical development pathway for
CER-001 for patients with this rare disease
- Treatment of LCAT deficiency or Norum disease presents a
major medical challenge, given the disease's constant progression
and rapid renal decline even following kidney grafting
Regulatory News:
ABIONYX Pharma, (FR0012616852 - ABNX - PEA PME eligible),
a new generation biotech company dedicated to the discovery and
development of innovative therapies based on the world's only
natural recombinant ApoA-I, announces that the company has been
granted a new Compassionate Access Authorization (CAA) in Europe
for CER-001 in the rare, untreated kidney disease LCAT
Deficiency.
This is the fourth patient suffering from LCAT deficiency to be
granted Compassionate Access to CER-001, in development by ABIONYX
Pharma, in Europe. The four patients suffering from this rare
disease, which has a major impact on quality of life and on
lifespan, represent very different stages in the evolution of the
disease, for which CER-001 may prove effective, whether or not the
patients are transplanted, and whatever their age.
CER-001 would represent a unique therapeutic breakthrough for
patients who need to be diagnosed as early as possible to avoid
dialysis, followed by one or more transplants during their
lifetime. The early administration of CER-001 in young patients
with Norum disease could have a real beneficial effect on these
patients, transforming their lives and, above all, delaying the end
of their lives, while very significantly reducing the management of
their disease, whether with dialysis or by one or more
transplants.
The safety and tolerability data for this treatment are as
positive as ever, with no adverse events reported for any of the
patients.
Constructive discussions have been initiated with the European
regulatory authorities to define a pathway for the rapid
advancement of CER-001's clinical development in the patient
population affected by this rare disease.
The treatment of LCAT deficiency or Norum disease is a major
medical challenge, given the constant progression of the disease,
leading to rapid renal decline and often premature death, and the
total absence of approved treatments to slow or modify the
progression of the disease.
ABIONYX Pharma continues to receive and fulfill Compassionate
Access requests from hospitals around the world. The
biomanufacturing of the latest and subsequent batches of CER-001 to
the highest GMP quality standards will enable these requests to be
met, and the clinical results of the first patients will be
communicated exclusively via scientific publication.
About LCAT deficiency or Norum disease
LCAT deficiency is a rare degenerative disease affecting around
1 in 1,000,000 newborns. It is characterized by renal failure and
hemolytic anemia, sometimes by corneal opacities, and biochemically
by severely reduced HDL-cholesterol levels. It is caused by
mutations in the LCAT gene, whose enzymatic activity catalyzes the
formation of cholesterol esters in lipoproteins. Accumulation of
unesterified cholesterol in the body, notably in the cornea, red
blood cells and kidneys, leads to severe degeneration and premature
death. There are currently no treatment options to alter the course
of LCAT deficiency, known as Norum disease after the Norwegian
physician Kaare Norum who identified and studied it in the 1960s.
To date there are no approved treatments for this disease.
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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version on businesswire.com: https://www.businesswire.com/news/home/20230710845791/en/
NewCap Investor relations Louis-Victor Delouvrier Nicolas
Fossiez abionyx@newcap.eu +33 (0)1 44 71 98 53
NewCap Media relations Arthur Rouillé abionyx@newcap.eu
+33 (0)1 44 71 94 94
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