Scienza e Tecnologia
Pharming announces the publication of data from a compassionate use programme of RUCONEST® in COVID-19 patients in a peer-reviewed journal
Given this was a small case series, the outcomes were retrospectively compared to a matched control population of 15 patients. Baseline characteristics, admission laboratory parameters and treatments administered were similar in both groups. Both groups received standard of care as well as experimental therapies including antiviral and anti-cytokine directed medications. However, 8/15 (53%) patients in the control population required mechanical ventilation and 4 of these patients died, compared to only 1 (20%) requiring mechanical ventilation and no deaths in the RUCONEST group. Overall, treatment with, in total five normal dose equivalents of RUCONEST over 48h was well-tolerated.
The full publication is available on the company's website www.pharming.com
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 attacks of angio-edema in patients with hereditary angioedema (HAE) in Europe ; for adults, adolescents and children from two years of age, in the US, for adults and adolescents and in 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 PI3Kδ 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.
Sijmen de Vries, CEO: T: +31 71 524 7400
Susanne Embleton , Investor Relations Manager: +31 71 524 7400/investor@pharming.com
Victoria Foster Mitchell / Mary Whittow
T: +44 203 727 1000
Leon Melens
T: +31 6 53 81 64 27
E: pharming@lifespring.nl