Pharming Announces FDA Acceptance for Review of Supplemental BLA for RUCONEST for Prophylaxis of Hereditary Angioedema Attacks

Leiden, the Netherlands – 17 Jan 2018

Pharming Group N.V. (Euronext Amsterdam: PHARM) announced today that the U.S. Food and Drug Administration (FDA) has accepted for review Pharming’s supplemental Biologics License Application (sBLA) for RUCONEST® [Recombinant Human C1 Esterase Inhibitor/ conestat alfa] for routine prophylaxis to prevent attacks in adult and adolescent patients with hereditary angioedema (HAE). The FDA has indicated that the sBLA is sufficiently complete to permit a substantive review and has set an action date of September 21, 2018.

RUCONEST® is currently approved for the treatment of acute attacks in adult and adolescent patients with HAE. If approved for this new indication, RUCONEST® would become the first C1 inhibitor therapy that would be approved for both acute treatment and prophylaxis of HAE attacks.

Pharming Group N.V.

Sijmen de Vries, CEO
Tel: +31 71 524 7400

Robin Wright, CFO
Tel: +31 71 524 7400

FTI Consulting, London, UK:

Julia Phillips/ Victoria Foster Mitchell
Tel: +44 203 727 1136

LifeSpring Life Sciences Communication, Amsterdam, The Netherlands:

Leon Melens
Tel: +31 6 53 81 64 27

This press release of Pharming Group N.V. and its subsidiaries (“Pharming”, the “Company” or the “Group”) 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 commercialize 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.

RUCONEST® (C1 esterase inhibitor [recombinant]) is indicated for the treatment of acute attacks in adult and adolescent patients with hereditary angioedema (HAE). Effectiveness in clinical studies was not established in HAE patients with laryngeal attacks.

• RUCONEST® (C1 esterase inhibitor [recombinant]) is contraindicated in:
  • Patients with a history of allergy to rabbits or rabbit-derived products.
  • Patients with a history of life-threatening immediate hypersensitivity reactions to C1 esterase inhibitor preparations, including anaphylaxis.
• Hypersensitivity: Severe hypersensitivity reactions may occur. Should symptoms occur, discontinue RUCONEST and administer appropriate treatment. Because hypersensitivity reactions may have symptoms similar to HAE attacks, treatment methods should be carefully considered.
• Thromboembolic Events: Serious arterial and venous thromboembolic (TE) events have been reported at the recommended dose of plasma-derived C1 esterase inhibitor products in patients with risk factors. Risk factors may include the presence of an indwelling venous catheter/access device, prior history of thrombosis, underlying atherosclerosis, use of oral contraceptives or certain androgens, morbid obesity, and immobility. Monitor patients with known risk factors for TE events during and after RUCONEST administration.
• Intravenous Use: RUCONEST is for intravenous use after reconstitution only. No more than 2 doses should be administered within a 24-hour period.
• Pregnancy and Nursing: RUCONEST has not been studied in pregnant women; therefore, it should only be used during pregnancy if clearly needed. Advise patients to notify their physician if they are breastfeeding or plan to breastfeed.
• Adverse reactions: The serious adverse reaction in clinical studies of RUCONEST was anaphylaxis.
• Common adverse reactions: The most common adverse reactions (incidence ≥2%) were headache, nausea, and diarrhea.

Please see Full Prescribing Information for RUCONEST® as applicable for various jurisdictions:

FDA: RUCONEST® / EMA: RUCONEST®

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 authorization.

RUCONEST® is commercialized by Pharming in Algeria, Andorra, Austria, Bahrain, Belgium, France, Germany, Ireland, Jordan, Kuwait, Lebanon, Luxembourg, Morocco, the Netherlands, Oman, Portugal, Qatar, Syria, Spain, Switzerland, Tunisia, the United Arab Emirates, the United Kingdom, the United States of America and Yemen.

RUCONEST® is distributed by Swedish Orphan Biovitrum AB (publ) (SS: SOBI) in the other EU countries, and in Azerbaijan, Belarus, Georgia, Iceland, Kazakhstan, Liechtenstein, Norway, Russia, Serbia and Ukraine.

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 Megapharm.

RUCONEST® is also being investigated in a Phase II clinical trial for the treatment of HAE in young children (2-13 years of age) and evaluated for various additional follow-on indications.

Pharming’s technology platform includes a unique, GMP-compliant, validated process for the production of pure recombinant human proteins that has proven capable of producing

industrial quantities of high quality recombinant human proteins in a more economical and less immunogenetic way compared with current cell-line based methods. Leads for enzyme replacement therapy (“ERT”) for Pompé and Fabry’s diseases are being optimized at present, with additional programs not involving ERT also being explored at an early stage at present.

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. Pre-clinical development and manufacturing will take place to global standards at CSIPI 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.

Pharming has declared that the Netherlands is its “Home Member State” pursuant to the amended article 5:25a paragraph 2 of the Dutch Financial Supervision Act.

Additional information is available on the Pharming website: www.pharming.com