LUND, Sweden, Sept. 16, 2021 /PRNewswire/ -- Camurus (NASDAQ
STO: CAMX) today announced that the US Food and Drug Administration
(FDA) has granted orphan drug designation (ODD) for the company's
investigational medicinal product, octreotide subcutaneous depot
(CAM2029), for the treatment of autosomal dominant polycystic liver
disease.
Polycystic liver disease (PLD) is a rare, genetic, and chronic
disorder characterized by progressive growth of cysts in the liver
which can cause severe symptoms and result in an impaired quality
of life of patients. There is today no approved pharmacological
treatment available for PLD.
"The orphan drug designation for CAM2029 is a significant
milestone in our efforts to develop an effective pharmacological
treatment for people with polycystic liver disease and a
recognition of the importance of our overall development program
for CAM2029," says Dr. Fredrik
Tiberg, CEO and Head of R&D at
Camurus. "CAM2029 is designed to address a
significant unmet medical need in this population and could
potentially become the first approved pharmacological treatment for
PLD in the US market," he added.
Camurus has also recently received a Safe-to-Proceed letter from
the FDA for the start of a randomized placebo-controlled Phase 2/3
study designed to assess efficacy and safety of CAM2029 in patients
with symptomatic PLD.
Orphan drug designation qualifies the drug developer for a
variety of development incentives and the prospect of seven years
of market exclusivity, if approved. The FDA's ODD program is
designed to advance the development of drugs for the treatment of
rare diseases affecting fewer than 200.000 people in the US. More
information about FDA rare diseases and the ODD program is
available on www.fda.org.
For more information
Fredrik Tiberg, President &
CEO
Tel. +46 (0)46 286 46 92
fredrik.tiberg@camurus.com
Fredrik Joabsson, Chief Business Development Officer
Tel. +46 (0)70 776 17 37
ir@camurus.com
About polycystic liver disease
Polycystic liver disease (PLD) is a rare genetic and chronic
disorder characterized by progressive growth of multiple (> 10)
fluid-filled cysts in the liver, which can cause severe symptoms
such as abdominal pain and discomfort, shortness of breath
(dyspnea), indigestion (dyspepsia), gastro-esophageal reflux, and
limited mobility. Rare complications are hepatic cyst hemorrhage,
infection or rupture.1-3 Age and gender contribute to
disease severity; increasing age is positively associated with both
cyst sizes and numbers, and women are highly overrepresented among
symptomatic patients.4-6 Most patients with
PLD are diagnosed in their 30s after reporting a sudden and
accelerated increase of abdominal girth, together with other
PLD-related symptoms.5 There is currently no approved
pharmacological treatment for PLD, but growing scientific evidence
has shown that somatostatin analogues, such as octreotide, are
effective in slowing cyst growth and fluid secretion in the liver
and that they may also help reduce liver
volume.7-8
About CAM2029
CAM2029 is a ready-to-use, long-acting subcutaneous depot of
octreotide under development for treatment of three rare diseases;
acromegaly, gastroenteropancreatic neuroendocrine tumors (GEP-NET),
and polycystic liver disease (PLD). CAM2029 has been successfully
evaluated in four completed clinical Phase 1 and 2 studies and is
being assessed in two ongoing pivotal Phase 3 studies in acromegaly
and a pivotal Phase 3 study in GEP-NET. A Phase 2/3 study in
patients with PLD is under preparation. CAM2029 is developed for
enhanced octreotide exposure and easy self-administration by
patients using a prefilled pen injector. CAM2029 has been granted
orphan designation in the EU for the treatment of acromegaly and in
the US for the treatment of PLD.
About Camurus
Camurus is a Swedish science-led biopharmaceutical company
committed to developing and commercializing innovative and
differentiated medicines for the treatment of severe and chronic
conditions. New drug products with best-in-class potential are
conceived based on the company's proprietary
FluidCrystal® drug delivery technologies and its
extensive R&D expertise. Camurus' clinical pipeline includes
products for the treatment of cancer, endocrine diseases, pain and
addiction, which are developed in-house and in collaboration with
international pharmaceutical companies. The company's shares are
listed on Nasdaq Stockholm under the ticker CAMX. For more
information, visit www.camurus.com.
References[AB1]
- Abu-Wasel, B., et al., Pathophysiology, epidemiology,
classification and treatment options for polycystic liver diseases.
World J Gastroenterol, 2013. 19(35): p.
5775-86.
- Perugorria, M.J., et al., Polycystic liver diseases:
advanced insights into the molecular mechanisms. Nat Rev
Gastroenterol Hepatol, 2014. 11(12): p.
750-61.
- Neijenhuis, M.K., et al., Impact of liver volume on
polycystic liver disease-related symptoms and quality of life.
United European Gastroenterol J, 2018. 6(1): p.
81-88.
- Van Keimpema L., et al. .
Patients with isolated polycystic liver disease referred to liver
centres: clinical characterization of 137 cases. Liver
international : official journal of the International Association
for the Study of the Liver.
2011;31(1):92-8.
- van Aerts RMM, et al. . Clinical management of polycystic
liver disease. J Hepatol. 2018;68(4):827-37.50. van Aerts RMM,
Kievit W, de Jong ME, Ahn C, Bañales JM, Reiterová J, et al.
Severity in polycystic liver disease is associated with aetiology
and female gender: Results of the International PLD Registry. Liver
international : official journal of the International Association
for the Study of the Liver.
2019;39(3):575-82.
- Gevers T. J. G., et al. Liver Int. 2015
May;35(5):1607-14.. doi:
10.1111/liv.12726;
- isani A., et al. Clin Gastroenterol Hepatol. 2016
Jul;14(7):1022-1030. doi:
10.1016/j.cgh.2015;
- van Aerts R., et al., Gastroenterology. 2019
Aug;157(2):481-491.
doi:10.1053/j.gastro.2019.04.018
This information, Camurus AB is obliged to make public pursuant
to the EU Market Abuse Regulation. The information was submitted
for publication, through the agency of the managing director, at
11:00 am CET on 16 September 2021.
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