ABIONYX Pharma Receives Positive Opinion From EMA Within the Framework of the Orphan Drug Designation Process for CER-001 for the Rare Disease LCAT Deficiency
28 Luglio 2021 - 07:30AM
Business Wire
- Culmination of new strategy in an ultra-rare kidney
disease
- New step in repositioning the product for renal and
ophthalmologic diseases
- Secured biomanufacturing of CER-001 for future
trials
Regulatory News:
ABIONYX Pharma (FR0012616852 - ABNX - PEA PME eligible),
a next-generation biotech company dedicated to the discovery and
development of innovative therapies, announced today that the
Committee for Orphan Medicinal Products (COMP) of the European
Medicines Agency (EMA) has issued a positive opinion on the
company's application for Orphan Drug Designation (ODD) for its
drug candidate CER-001, as a potential treatment for
lecithin-cholesterol acyltransferase (LCAT) deficiency clinically
characterized by hemolytic anemia and renal failure, most often
leading to renal transplantation, and corneal opacities. CER-001 is
a first-in-class bio-HDL mimetic that directly targets a key
underlying metabolic defect of LCAT deficiency.
The culmination of a new strategy to use CER-001 in an
ultra-rare renal disease
This positive opinion from the COMP of the EMA for an orphan
drug designation for bio-HDL is of strategic importance for the
development of our product. This designation offers an important
advantage by providing access to the centralized marketing
authorization procedure as well as 10 years of market exclusivity
in the European Community from the date of obtaining a marketing
authorization. This positive opinion is also a recognition of the
need for innovative treatments for patients with LCAT deficiency, a
serious and lifelong disease. Several nATU applications have been
received necessitating the decision to secure biomanufacturing to
produce clinical batches for patient use and a European regulatory
filing. CER-001 is the only bio-HDL to receive this important
designation in Europe and the first potential disease-modifying
treatment for patients with LCAT deficiency.
Orphan drug designation in the European Union (EU) is granted by
the European Commission on the basis of a positive opinion issued
by the COMP of the EMA. To obtain this designation, a drug
candidate must be intended to treat a severely debilitating or
life-threatening condition that affects fewer than 5 in 10,000
people in the EU, and there must be sufficient clinical or
non-clinical data to suggest that the investigational drug can
produce clinically relevant results. Orphan drug designation by the
EMA provides companies with certain benefits and incentives,
including assistance with clinical protocols, differentiated
assessment procedures for health technology assessments in certain
countries, access to a centralized marketing authorization
procedure valid in all EU member states, reduced regulatory fees,
and 10 years of market exclusivity from the time a marketing
authorization is granted.
New step in repositioning the product for renal and
ophthalmologic diseases
The prevalence of LCAT deficiency is estimated to be less than 1
in 1,000,000. Several named compassionate use programs (nATUs) are
evaluating the efficacy, safety, tolerability and compliance of
CER-001 in patients with LCAT deficiency in Europe.
As LCAT deficiency also affects the cornea, this orphan
designation paves the way for clinical development in
ophthalmology, as announced in the context of expanding the
potential of CER-001's innovation and its pleiotropic action as a
natural recombinant HDL.
In addition to nATUs, ABIONYX Pharma is currently conducting a
Phase 2a clinical trial evaluating the efficacy, safety,
pharmacokinetics and pharmacodynamics of CER-001 treatment in
adults with sepsis at high risk of Acute Kidney Injury, a disease
affecting more than 2 million people worldwide.
Securing the bioproduction of CER-001
As anticipated, the company had decided to launch purchases in
view of the scarcity in the supply of raw materials and on the
production deadlines of the various inputs for the manufacture of
its bio-HDL due to COVID 19. Therefore, the company, after having
signed its strategic production agreement, has secured the
bioproduction of CER-001 vials for its various upcoming trials.
CER-001 is now one of the most advanced biomedicines in France, and
ABIONYX Pharma controls the entire value chain of its biomedicine,
from patents in renal and ophthalmologic diseases to its
bioproduction, through the various clinical stages. The company
targets a marketing application for CER-001 in LCAT deficiency as
soon as possible.
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 Nicolas Merigeau abionyx@newcap.eu
+33 (0)1 44 71 94 98
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