FDA Grants Orphan Drug Designation to ADV7103 for the Treatment of Cystinuria.
25 Mars 2024 - 7:30AM
Business Wire
- After distal Renal Tubular Acidosis (dRTA) in December
2022, ADV7103 is granted "orphan drug" status in the United States
in cystinuria.
- A long-awaited milestone for the Company and its flagship
product has now been reached
- Cystinuria, a hereditary kidney disorder, is a major unmet
medical need, affecting some 30,000 patients in the U.S.
- The Company is now looking forward to signing a partnership
to pursue the development and prepare the marketing of ADV7103 in
the U.S in its two indications: cystinuria and dRTA.
Regulatory News:
Advicenne (Euronext Growth Paris ALDVI - FR0013296746), a
specialty pharmaceutical company dedicated to the development and
commercialization of innovative treatments for those suffering from
rare renal diseases, today announced that its proprietary drug
ADV7103 has been granted Orphan Drug Designation (ODD) status for
the treatment of cystinuria. This designation was issued by the US
Food and Drug Administration (US FDA). ADV7103 is one of the few
drugs to obtain orphan designation in the USA for both indications,
cystinuria and distal Renal Tubular Acidosis (dRTA).
Didier Laurens, Chief Executive Officer of Advicenne,
declared: “After receiving an initial orphan designation of
ADV7103 in dRTA in 2022, we are absolutely delighted to announce
today that we have obtained orphan drug status in cystinuria. After
months of intense discussions with the FDA, this is fantastic news
for Advicenne and a major element of value creation. I am very
proud of the teams who worked hard for this success, especially in
the USA. I am also very proud that the first clinical data obtained
in France have convinced the FDA of the benefit of ADV7103 in the
treatment of cystinuria. The treatment of this condition with
simplified alkalinization is a major unmet medical need with a very
significant market potential with more than 30,000 patients in the
USA. Obtaining this ODD in cystinuria is the powerful lever we have
been waiting for to accelerate and finalize discussions with
potential partners in the United States”.
Cystinuria is a genetic disease characterized by a buildup of
the amino acid cystine in the kidneys and bladder. These high
levels of cystine lead to the formation of cystine stones in the
kidneys, ureter, and bladder. These stones generate various
complications, such as hypertension, intense abdominal pain,
recurrent urinary tract infections, renal function impairment in up
to 70% of patients and chronic renal insufficiency. These
complications can ultimately lead to chronic renal failure.
To date, 40,000 patients suffer from cystinuria in Europe and
around 30,000 in the USA. The treatment of this pathology is based
primarily on the alkalinization of urine combined with significant
fluid intake and a low-protein diet to limit the recurring
formation of kidney stones. ADV7103, an innovative and proprietary
fixed combination of sustained-release potassium citrate and
potassium bicarbonate, has the potential to become the first major
drug in the treatment of cystinuria in North America, as well as in
Europe, where ADV7103 is also designated an orphan drug since
December 2022.
ABOUT ADVICENNE
Advicenne (Euronext: ALDVI) is a specialty pharmaceutical
company founded in 2007, specializing in the development of
innovative treatments in Nephrology. Its lead product Sibnayal® has
received Marketing Approval for distal renal tubular acidosis
(dRTA) in EU and GB. ADV7103 is currently in late-stage development
in cystinuria in Europe and in dRTA and cystinuria in the US and
Canada. Headquartered in Paris, Advicenne, listed on the Euronext
Paris stock exchange since 2017, has now been listed on Euronext
Growth Paris since its transfer on March 30, 2022. For
additional information, see: https://advicenne.com/.
Disclaimer
This press release contains certain forward-looking statements
concerning Advicenne group and its business, including its
prospects and product candidate development. Such forward-looking
statements are based on assumptions that Advicenne considers to be
reasonable. However, there can be no assurance that the estimates
contained in such forward-looking statements will be verified,
which estimates are subject to numerous risks including the risks
set forth in the 2022 Universal Registration Document filed with
the French financial market authority on April 28, 2023 (a copy of
which is available on www.advicenne.com) and to the development of
economic conditions, financial markets and the markets in which
Advicenne operates. The forward-looking statements contained in
this press release are also subject to risks not yet known to
Advicenne or not currently considered material by Advicenne. The
occurrence of all or part of such risks could cause actual results,
financial conditions, performance, or achievements of Advicenne to
be materially different from such forward-looking statements.
Advicenne expressly declines any obligation to update such
forward-looking statements.
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version on businesswire.com: https://www.businesswire.com/news/home/20240324280382/en/
Advicenne Didier Laurens, CEO +33 (0) 1 87 44 40 17
Email: investors@advicenne.com
Ulysses Communication Media relations Bruno Arabian +33
(0)6 87 88 47 26 Email: advicenne@ulysse-communication.com
Advicenne (EU:ALDVI)
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