Pharming receives Orphan Drug Designation from the European Commission for leniolisib for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) - Seite 3

Leniolisib was recently in-licensed from Novartis and is being studied in a registration-enabling Phase II/III trial which is currently enrolling patients in clinical sites in the US and Europe. To date, leniolisib has proven to be safe and well tolerated in healthy subjects as well as APDS patients during the first-in-human trial and the ongoing open label extension trial.

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 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 for adults. The product is available on a named-patient basis in other territories where no marketing authorisation application was yet submitted.

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 developed 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 the commercialisation of RUCONEST in the Republic of China and the future development of new products.