LEIDEN, Netherlands, April 30, 2020 /PRNewswire/ -- Pharming announces that it has received formal European Commission approval to treat acute hereditary angioedema (HAE) attacks in children with RUCONEST®. Following the positive opinion and recommendation from the European Medicine Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) on the extension of the indication for RUCONEST® received on 26 March 2020.
-- Pharming has received a formal European Commission (EC) decision on the
approval of a new RUCONEST® license extension in Europe
-- European Commission decision was received six weeks earlier than
-- The extension of the indication means that RUCONEST® is now approved
for the treatment of acute angioedema attacks in adults, adolescents,
and children (aged two years and above) with hereditary angioedema (HAE)
due to C1 esterase inhibitor deficiency in Europe
Pharming Group N.V. today announces that the European Commission has approved an extension in the indication of RUCONEST®'s (conestat alfa) Marketing Authorisation to include the treatment of acute angioedema attacks in children with hereditary angioedema (HAE). This marketing authorisation expands the age range of Pharming's lead product, RUCONEST®, a recombinant analogue of human C1 esterase inhibitor. RUCONEST® was previously approved for adults and adolescents in Europe.
The European Commission's decision allows children aged two years and older to be treated with RUCONEST® for acute angioedema attacks. In the European Union, RUCONEST® has been approved for this indication in adults since 2010 and in adolescents since 2016.
HAE is a rare disease caused by a deficiency of the C1 esterase inhibitor protein and is characterised by spontaneous and recurrent episodes of swelling (edema attacks) of the skin in different parts of the body, as well as in the airways and internal organs. Edema of the throat, nose or tongue is particularly dangerous and potentially life-threatening and can lead to obstruction of the airway passages.
The C1 esterase inhibitor protein is required to control the 'complement' and 'contact' systems, collections of proteins in the blood that fight against infection and cause inflammation. Patients with low levels of this protein have excessive activity of these two systems, which leads to the symptoms of angioedema. The active substance in RUCONEST®, conestat alfa, is a copy of the C1 esterase inhibitor protein and works in the same way as the natural human protein. When it is given during an angioedema attack, RUCONEST® stops this excessive activity, helping to relieve the patient's symptoms.
Sijmen de Vries, Chief Executive of Pharming, said:
"We are pleased to receive approval from the European Commission and to be able to offer RUCONEST® as a treatment for acute HAE attacks in all patients aged two years and above. As we expand our distribution network in Europe following the reacquisition of RUCONEST®-licensed territories in December 2019, we are seeing increasing demand for the product in the treatment of HAE. This approval allows us to treat the most vulnerable patients and further demonstrates the safety and efficacy of RUCONEST®."
Paediatric study results
The open-label, single arm, Phase II clinical trial was designed in agreement with the EMA as part of a Paediatric Investigation Plan (PIP) to assess the pharmacokinetic, safety and efficacy profiles of RUCONEST® at a dose of 50 U/kg in HAE patients aged 2-13 years in support of the indication for treatment of HAE attacks in children.
A total of 20 children with HAE were treated for 73 HAE attacks at a dose of 50 U/kg (up to a maximum of 4200 U). The study reported clinically meaningful relief of symptoms assessed using a visual analogue scale (VAS) completed by the patient (assisted by their parent). The median time to onset of relief was 60 minutes (95% confidence interval: 60-653), and the median time to minimal symptoms was 123 minutes (95% confidence interval: 120-126). Only 3/73 (4%) attacks were treated with a second dose of RUCONEST®.
RUCONEST® was generally safe and well-tolerated in the study. No patients withdrew from the study due to adverse events. There were no related serious adverse events, hypersensitivity reactions, or neutralising antibodies detected.
Hereditary Angioedema (HAE) is a rare genetic disorder. The condition is caused by a deficiency of the C1 esterase inhibitor protein, which is normally present in blood and helps control inflammation (swelling) and parts of the immune system. Because defective C1-Inhibitor does not adequately perform its regulatory function, a biochemical imbalance can occur and produce unwanted peptides that induce the capillaries to release fluids into surrounding tissue, thereby causing swelling or edema.
HAE is characterised by spontaneous and recurrent episodes of swelling (edema attacks) of the skin in different parts of the body, as well as in the airways and internal organs. Edema of the skin usually affects the extremities, the face, and the genitals. Patients suffering from this kind of edema often withdraw from their social lives because of the disfiguration, discomfort and pain these symptoms may cause. Almost all HAE patients suffer from bouts of severe abdominal pain, nausea, vomiting and diarrhoea caused by swelling of the intestinal wall.
Edema of the throat, nose or tongue is particularly dangerous and potentially life-threatening and can lead to obstruction of the airway passages. Although there is currently no known cure for HAE, it is possible to treat the symptoms associated with angioedema attacks. HAE affects about 1 in 10,000 to 1 in 50,000 people worldwide. Experts believe that a lot of patients are still seeking the right diagnosis: although HAE is (in principle) easy to diagnose, it is frequently identified very late or not discovered at all. The reason HAE is often misdiagnosed is because the symptoms are similar to those of many other common conditions such as allergies or appendicitis. By the time it is diagnosed correctly, the patient has often been through a long-lasting ordeal.
RUCONEST® (recombinant C1 esterase inhibitor) is indicated for the treatment of acute attacks in adult and adolescent patients with hereditary angioedema (HAE).
RUCONEST® contains C1 esterase inhibitor at 50 U/kg. When administered at the onset of HAE attack symptoms at the recommended dose, RUCONEST® may help to return a patient's C1 esterase inhibitor levels to normal range and relieve the symptoms of an HAE attack with a low recurrence of symptoms within 24 hours.
The most common side effect of RUCONEST® (seen in between 1 and 10 patients in 100) is nausea. For the full list of all side effects reported with RUCONEST®, see the package leaflet. RUCONEST® must not be used in patients with known or suspected allergy to rabbits. For the full list of restrictions, see the package leaflet.
RUCONEST® is the only recombinant C1 esterase inhibitor worldwide. RUCONEST® is approved by the US Food and Drug Administration (FDA) for the treatment of acute attacks in adult and adolescent patients with HAE since July 2014.
About Pharming Group N.V.
Pharming is a specialty pharmaceutical company developing innovative products for the safe, effective treatment of rare diseases and unmet medical needs. Pharming's lead product, RUCONEST® (conestat alfa) is a recombinant human C1 esterase inhibitor approved for the treatment of acute Hereditary Angioedema ("HAE") attacks in patients in Europe, the US, Israel and South Korea. The product is available on a named-patient basis in other territories where it has not yet obtained marketing authorisation.
RUCONEST® is commercialised by Pharming in the US and in Europe, and the Company holds all other commercialisation rights in other countries not specified below. In some of these other countries distribution is made in association with the HAEi Global Access Program (GAP). RUCONEST® is distributed in Argentina, Colombia, Costa Rica, the Dominican Republic, Panama, and Venezuela by Cytobioteck, in South Korea by HyupJin Corporation and in Israel by Kamada.
RUCONEST® is also being evaluated for various additional indications. Pharming's technology platform includes a unique production process that has proven capable of producing industrial quantities of pure high quality recombinant human proteins in a more economical and less immunogenic way compared with current cell-line based methods.
Leads for enzyme replacement therapy ("ERT") for Pompe and Fabry's diseases are also being produced and optimised respectively at present.
Pharming has recently in-licensed leniolisib from Novartis, a small molecule and selective PI3K inhibitor, which is in a registrational study for activated PI3K-delta syndrome (APDS), a rare form of Primary Immunodeficiency.
Pharming has a long term partnership with the China State Institute of Pharmaceutical Industry ("CSIPI"), a Sinopharm company, for joint global development of new products, starting with recombinant human Factor VIII for the treatment of Haemophilia A. Preclinical development and manufacturing will take place to global standards at CSIPI and its affiliates and are funded by CSIPI. Clinical development will be shared between the partners with each partner taking the costs for their territories under the partnership.
This press release of Pharming Group N.V. and its subsidiaries ("Pharming", the "Company") may contain forward-looking statements including without limitation those regarding Pharming's financial projections, market expectations, developments, partnerships, plans, strategies and capital expenditures.
The Company cautions that such forward-looking statements may involve certain risks and uncertainties, and actual results may differ. Risks and uncertainties include without limitation the effect of competitive, political and economic factors, legal claims, the Company's ability to protect intellectual property, fluctuations in exchange and interest rates, changes in taxation laws or rates, changes in legislation or accountancy practices and the Company's ability to identify, develop and successfully commercialise new products, markets or technologies.
As a result, the Company's actual performance, position and financial results and statements may differ materially from the plans, goals and expectations set forth in such forward-looking statements. The Company assumes no obligation to update any forward-looking statements or information, which should be taken as of their respective dates of issue, unless required by laws or regulations.
For further public information, contact:
Sijmen de Vries, CEO: T: +31 71 524 7400
Susanne Embleton, Investor Relations Manager: T: +31 71 524 7400
Victoria Foster Mitchell / Mary Whittow, T: +44 203 727 1000
LifeSpring Life Sciences Communication, Amsterdam, The Netherlands
Leon Melens, Tel: +31 6 53 81 64 27