Pharming plans submission Rhucin BLA to US FDA end 2010
(Thomson Reuters ONE) -
Leiden, The Netherlands, August 25, 2010. Biotech company Pharming Group NV
("Pharming" or "the Company") (NYSE Euronext: PHARM) today announced that it
intends to submit the Biologic License Application (BLA) to the US Food and Drug
Administration (FDA) to obtain marketing approval for Rhucin® for the treatment
of acute angioedema attacks in patients with Hereditary Angioedema (HAE).
Following pre-BLA discussions with the FDA, Pharming is preparing the BLA
dossier for submission towards the end of this year but no later than January
2011.
The BLA will be based on the data of the European Marketing Authorization
Application (MAA), on which the European Medicines Agency's Committee for
Medicinal Products for Human Use (CHMP) adopted a positive opinion in June of
this year. The BLA will be updated with patient data collected since the
completion of the MAA package and will include additional analyses requested by
the FDA. The BLA dossier will include data on over 500 administrations in over
150 patients, demonstrating the safety and efficacy of Rhucin for the treatment
of HAE attacks.
To further strengthen Rhucin's competitive profile, Pharming is preparing to
initiate a Phase IIIB/IV study. In this global multicenter randomized
placebo-controlled study, focusing on "time to onset of relief" of HAE symptoms,
50 patients will either receive 50U/kg Rhucin or placebo.
"Following the positive opinion from the European Medicines Agency in June this
year, the BLA submission will be the next significant milestone in the
development of Rhucin. It demonstrates our commitment to provide global access
to this innovative highly effective and safe replacement therapy for HAE
patients", said Dr. Rienk Pijpstra, Chief Medical Officer. "The study will
further emphasize Rhucin's benefits for HAE patients such as rapid onset of
relief and excellent response rates."
More detailed information on clinical studies can shortly be found on
www.clinicaltrials.gov. More information on the BLA procedure can be found on
www.fda.gov. The Company will provide further information on the Rhucin BLA
review at appropriate stages during the process.
List of used abbreviations
BLA Biologic License Application
CHMP European Medicines Agency's Committee for Medicinal Products for Human
Use
FDA US Food and Drug Administration
HAE Hereditary Angioedema
MAA Marketing Authorization Application
Rhucin Rhucin® in non-EU territories, Ruconest(TM) in European Economic Area
Biologic License Application (BLA)
In the USA, biological products are approved for marketing under the provisions
of the Public Health Service (PHS) Act. The Act requires a firm which
manufactures a biologic for sale in interstate commerce to hold a license for
the product. To commercialize the new biological product in the USA, the FDA
needs to approve a Biologics License Application (BLA). A BLA is a submission
that contains specific information on the manufacturing processes, chemistry,
pharmacology, clinical pharmacology and the medical affects of the biologic
product. If the information provided meets FDA requirements, the application is
approved and a license is issued allowing the company to market the product.
Background on Hereditary Angioedema
HAE is a genetic disorder caused by a shortage of C1 inhibitor activity.
Approximately one in 30,000 individuals suffers from HAE and has an average of
seven acute attacks per year. HAE attacks that are untreated can last up to five
days. The disease is characterized by acute attacks of painful swelling of soft
tissues (edema), including regions of the skin, the intestine, and the mouth and
throat. If the soft tissue of the throat is involved, an attack of angioedema
can be fatal. In addition to the life-threatening nature of the disease, quality
of life for individuals with the disease may be seriously impaired.
About Pharming Group NV
Pharming Group NV is developing innovative products for the treatment of genetic
disorders, specialty products for surgical indications, and nutritional
products. On June 24 2010, the European Medicines Agency adopted a positive
opinion for Ruconest(TM) (Rhucin in non-EU territories) for the treatment of
angioedema attacks. Market Authorization in the European Economic Area is
therefore expected to be granted in September 2010. The product is also under
development for follow-on indications, i.e. antibody-mediated rejection (AMR)
and delayed graft function (DGF) following kidney transplantation. The
technologies of the Company include innovative platforms for the production of
protein therapeutics, including technology and processes for the purification
and formulation of these products. Additional information is available on the
Pharming website,http://www.pharming.com.
This press release contains forward looking statements that involve known and
unknown risks, uncertainties and other factors, which may cause the actual
results, performance or achievements of the Company to be materially different
from the results, performance or achievements expressed or implied by these
forward looking statements.
Contact:
Ms. Marjolein van Helmond, Pharming Group NV, T: +31 (0)71 52 47 431 or +31 (0)6
109 299 54
[HUG#1440232]
Press release (PDF):
http://hugin.info/132866/R/1440232/384592.pdf
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Source: Pharming Group N.V. via Thomson Reuters ONE
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Datum: 25.08.2010 - 14:50 Uhr
Sprache: Deutsch
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