SHANGHAI, March 29, 2024 /PRNewswire/ -- Alebund
Pharmaceuticals ("Alebund" or the "Company"), an integrated
biopharmaceutical company focusing on developing innovative
therapies for the treatment of renal diseases and related chronic
conditions, announced today that the US Food and Drug
Administration (FDA) has granted Orphan Drug Designation (ODD) to
AP303 for the treatment of autosomal dominant polycystic kidney
disease (ADPKD).
"We are very excited that AP303 has been granted Orphan Drug
Designation by the FDA", Dr. Gavin
Xia, Co-founder, Chairman, and CEO of Alebund commented. "It
is an important milestone for Alebund as well as our efforts to
address the significant unmet medical needs with ADPKD. We look
forward to advancing AP303 to improve future treatment options for
patients in need."
AP303, a novel drug candidate developed in-house by Alebund,
has demonstrated a meaningful improvement
of renal survival in an ADPKD mice model. AP303 has
completed its first-in-human study in
healthy subjects in Australia and is ready for a Phase II trial in
ADPKD. The prevalence of ADPKD is reported to be between 1 in 400
and 1 in 1,000 live births; it is an important cause of end-stage
kidney disease (ESKD) requiring renal replacement therapy
[1]. This Orphan Drug Designation reflects the need for
more new treatment options and the potential for AP303 to address
the pressing needs of people living with ADPKD.
The US FDA's ODD is a special status granted to support the
development and evaluation of potential new medicines intended for
the treatment, diagnosis, or prevention of rare diseases or
disorders that affect fewer than 200,000 people in the US. The
designation provides incentives to advance the development of
treatments for rare diseases, including marketing exclusivity, tax
credits for clinical research costs, and exemption from
prescription drug user fees.
About ADPKD
ADPKD is the most common monogenic kidney disease and one of the
leading causes of end-stage kidney disease in adults worldwide. Its
course is characterized by the development and inexorable expansion
of multiple cysts scattered throughout the kidney parenchyma. ADPKD
is most caused by mutations in the
genes PKD1 (in 78% of disease pedigrees) or PKD2 (in 15% of disease
pedigrees). Treatment recommendation for patients with ADPKD
includes anti-hypertensive treatment, dietary restrictions, and
pharmacotherapy for a limited percentage of suitable patients. More
broadly applicable treatment options with fewer side effects are
needed to effectively slow the decline of kidney function and
improve kidney outcomes in patients with ADPKD.
About Alebund Pharmaceuticals
Alebund is a biopharmaceutical company jointly incubated by a
group of industry leaders in the field of nephrology in
Shanghai in 2018. Alebund focuses
on the discovery and development of novel therapies primarily for
kidney diseases and their complications, as well as other chronic
conditions. Alebund has built a diversified and balanced pipeline
of drug candidates targeting a range of renal diseases, including
chronic kidney disease (CKD)/dialysis complications, IgA
nephropathy, diabetic kidney disease, and autosomal dominant
polycystic kidney disease (ADPKD). Alebund's pipeline comprises
both small-molecule and biological assets, in which many programs
are in the clinical stage.
[1] Bergmann, C., Guay-Woodford, L.M., Harris, P.C. et
al. Polycystic kidney disease. Nat Rev Dis
Primers 4, 50 (2018).
https://doi.org/10.1038/s41572-018-0047-y
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SOURCE Alebund Pharmaceuticals