SOURCE: Pharming Group N.V.

August 25, 2010 09:06 ET

Pharming plans submission Rhucin BLA to US FDA end 2010

LEIDEN, NETHERLANDS--(Marketwire - 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™ 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™ (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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Contact Information

  • Contact:

    Ms. Marjolein van Helmond
    Pharming Group NV
    T: +31 (0)71 52 47 431 or
    +31 (0)6 109 299 54