LEIDEN, NETHERLANDS--(Marketwire - September 03, 2009) - Biotech company Pharming
Group NV ("Pharming" or "the Company") (NYSE Euronext: PHARM) today
announced that it has submitted the Marketing Authorization
Application (MAA) for Rhucin for the treatment of acute attacks of
Hereditary Angioedema (HAE) to the European Medicines Agency (EMEA).
In this new MAA, Pharming has followed up on and addressed concerns
raised by the CHMP during the former evaluation procedure in 2007. In
particular, the size of the clinical database has been significantly
expanded, additional clinical efficacy and safety data have been
collected and analyzed, and an analysis of immunogenicity data after
(repeat) treatment with Rhucin has been completed.
Efficacy of Rhucin was demonstrated in two randomized controlled
studies and is supported by four open label studies, including the
successful treatment of potentially life-threatening laryngeal
attacks. In the placebo controlled clinical studies a total of 70
patients were randomized to Rhucin (100 U/kg or 50 U/kg body weight)
or placebo control treatment. Patients with active Rhucin treatment
responded rapidly to treatment, with statistically significant and
clinically relevant shorter time to onset of relief and time to
minimal symptoms compared to placebo. No relapses of attacks were
recorded. Superiority over placebo was also consistently supported by
exploratory endpoints, sensitivity analyses and subgroup analyses.
Additional data on 155 attacks in 79 patients collected in the
ongoing open-label studies furthermore confirmed Rhucin's efficacy.
The updated safety dataset comprising 405 administrations in 164
subjects, includes 14 patients with at least five repeat
administrations, Rhucin continued to be well tolerated, the adverse
event profile observed in the controlled studies, was similar to that
of placebo, and there were no significant infusion site reactions
such as pain. The immunogenicity analysis of 621 plasma samples
covering 217 treatments in 119 patients showed a reassuring
immuno-safety profile, without evidence for induction of neutralizing
antibodies against Rhucin, nor for induction of allergies.
"The MAA re-submission is a significant milestone in the development
of Rhucin and demonstrates our commitment to provide a highly
effective therapy for patients living with the burden of Hereditary
Angioedema", said Dr. Bruno Giannetti, Chief Operations Officer. "We
believe that our new MAA submission addresses all issues that
remained after the previous CHMP opinion in 2008. We are convinced
that our current clinical database adequately supports the efficacy
and safety of Rhucin, in single and repeated use and in all types of
acute attacks including laryngeal attacks. Our next focus will be the
admission of a Biologic License Application (BLA) for Rhucin in the
USA and we will request a pre-BLA meeting by the end of 2009."
Upon receipt of the MAA, the EMEA will validate the dossier and may
require additional data, information or clarification before the
start of evaluation of the scientific content of the dossier. Once
the application is validated, the EMEA starts the Centralised
Procedure for scientific evaluation of the MAA by the Committee for
Medicinal Products for Human Use (CHMP) according to a standard
timetable. At day 120, the CHMP produces a List of Questions to be
answered within 3 months by the Company. More information on this
procedure can be found on www.emea.europa. The Company will provide
further information on progress of the review of the Rhucin MAA
dossier at appropriate stages during the process.
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, ageing diseases, specialty products for
surgical indications, intermediates for various applications and
nutritional products. Pharming has two products in late stage
development - Rhucin® for Hereditary Angioedema and human Lactoferrin
for use in food products and one product in early stage clinical
development - Prodarsan® for Cockayne Syndrome. The advanced
technologies of the Company include innovative platforms for the
production of protein therapeutics, technology and processes for the
purification and formulation of these products, as well as technology
in the field of DNA repair (via DNage). 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
Sijmen de Vries, Pharming Group NV, T: +31 (0)71 52 47 400
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