BioCryst Announces Approval of ORLADEYO® (berotralstat) in United Arab Emirates

Company selects NewBridge Pharmaceuticals as regional distributor in Gulf Cooperation Council

RESEARCH TRIANGLE PARK, N.C., Sept. 09, 2021 (GLOBE NEWSWIRE) — BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the Ministry of Health and Prevention (MOHAP) in the United Arab Emirates (UAE) has granted marketing authorization for oral, once-daily ORLADEYO® (berotralstat) for the prevention of recurrent attacks in patients with hereditary angioedema (HAE) 12 years and older. To support commercialization efforts in the UAE, BioCryst has entered into a supply and distribution agreement with NewBridge Pharmaceuticals (NewBridge), which also covers the Gulf Cooperation Council (GCC) and Iraq.

“As the first targeted oral, once-daily treatment, ORLADEYO provides an important new treatment option for patients and physicians,” said Henrik Balle Boysen, executive vice president and chief operating officer of HAE International, a global non-profit network of patient associations dedicated to improving the lives of people with HAE. “While there is still more work to be done to raise awareness to support earlier diagnosis and treatment, the approval of ORLADEYO is an important advancement for HAE patients in the UAE.”

“With many prevalent rare diseases in the MENA region, I am personally inspired, and we at NewBridge are proud, to be part of this partnership with BioCryst for the UAE and a number of other markets in the GCC. This partnership supports our mission by providing access to an important new therapy for HAE patients in a hope that we can help ease their suffering and support them to live better lives,” said Joe Henein, president and chief executive officer of NewBridge Pharma.

“NewBridge is the right partner for BioCryst as they share our vision to bring innovative medicines to patients living with rare diseases,” said Charlie Gayer, chief commercial officer of BioCryst. “With experience across regulatory, medical and commercial, and strong local relationships with key stakeholders, NewBridge will help accelerate our efforts to bring ORLADEYO to patients across the globe by providing a much-needed new option to HAE patients in the UAE.”

NewBridge Pharmaceuticals, headquartered in Dubai, UAE, is a regional specialty company with a comprehensive pharmaceutical platform of services and expertise, established to bridge the access gap and partner with global pharma and biotech companies to in-license and commercialize U.S. Food and Drug Administration or European Medicines Agency approved innovative therapeutics that address unmet medical needs into the Middle East and North Africa (MENA) regions.

 

About ORLADEYO® (berotralstat)

ORLADEYO® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.

U.S. Indication and Important Safety Information

 

INDICATION
ORLADEYO® (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.

 

Limitations of use
The safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been established. ORLADEYO should not be used for the treatment of acute HAE attacks. Additional doses or dosages of ORLADEYO higher than 150 mg once daily are not recommended due to the potential for QT prolongation.

 

IMPORTANT SAFETY INFORMATION
An increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-daily dosage and was concentration dependent.

The most common adverse reactions (≥10% and higher than placebo) in patients receiving ORLADEYO were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease.

A reduced dosage of 110 mg taken orally once daily with food is recommended in patients with moderate or severe hepatic impairment (Child-Pugh B or C) and in patients taking chronically administered P-glycoprotein (P-gp) or breast cancer resistance protein (BCRP) inhibitors (eg, cyclosporine).

Berotralstat is a substrate of P-gp and BCRP. P-gp inducers (eg, rifampin, St. John’s wort) may decrease berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. The use of P-gp inducers is not recommended with ORLADEYO.

ORLADEYO at a dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a narrow therapeutic index that are predominantly metabolized by CYP2D6 or CYP3A4, appropriate monitoring and dose titration is recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor. Appropriate monitoring and dose titration is recommended for P-gp substrates (eg, digoxin) when coadministering with ORLADEYO.

The safety and effectiveness of ORLADEYO in pediatric patients <12 years of age have not been established.

There are insufficient data available to inform drug-related risks with ORLADEYO use in pregnancy. There are no data on the presence of berotralstat in human milk, its effects on the breastfed infant, or its effects on milk production.

To report SUSPECTED ADVERSE REACTIONS, contact BioCryst Pharmaceuticals, Inc. at 1-833-633-2279 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see full Prescribing Information.

 

About BioCryst Pharmaceuticals

BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily ORLADEYO® (berotralstat) is approved in the United States, the European Union, Japan, the United Arab Emirates and the United Kingdom. BioCryst has several ongoing development programs including BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva, and galidesivir, a potential treatment for Marburg virus disease and Yellow Fever. RAPIVAB® (peramivir injection) has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan and Korea. Post-marketing commitments for RAPIVAB are ongoing. For more information, please visit the company’s website at www.biocryst.com.

 

Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding BioCryst’s plans and expectations for ORLADEYO. These statements involve known and unknown risks, uncertainties and other factors which may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: the ongoing COVID-19 pandemic, which could create challenges in all aspects of BioCryst’s business, including without limitation delays, stoppages, difficulties and increased expenses with respect to BioCryst’s and its partners’ development, regulatory processes and supply chains, negatively impact BioCryst’s ability to access the capital or credit markets to finance its operations, or have the effect of heightening many of the risks described below or in the documents BioCryst periodically files with the Securities and Exchange Commission; BioCryst’s ability to successfully implement its commercialization plans for, and to commercialize, ORLADEYO, which could take longer or be more expensive than planned; the commercial viability of ORLADEYO, including its ability to achieve market acceptance; the results of our partnership with NewBridge may not meet our current expectations; the FDA, MOHAP or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay, or withdraw market approval for products and product candidates; BioCryst’s ability to successfully manage its growth and compete effectively; risks related to the international expansion of BioCryst’s business; and actual financial results may not be consistent with expectations, including that revenue, operating expenses and cash usage may not be within management’s expected ranges.  Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause the actual results to differ materially from those contained in BioCryst’s forward-looking statements.

Brukinsa® (zanubrutinib) capsules, for oral use

Name Brukinsa® (zanubrutinib) capsules
Description

Brukinsa® (zanubrutinib) is a Bruton’s tyrosine kinase (BTK) inhibitor.

Indication Indicated for the treatment of adults with mantle cell lymphoma (MCL) who have received at least one prior treatment for their cancer.
 Strengths

80 mg capsules
Recommended dose:

  • The recommended dose of Brukinsa is 160 mg taken orally twice daily or 320 mg taken orally once daily.
 Regulatory Status

EMA approved in 2021; USFDA approved in 2019
Registered in Saudi Arabia and UAE

 Business Partner BeiGene

BeiGene and NewBridge Pharmaceuticals Announce Approval in Saudi Arabia of BRUKINSA® (Zanubrutinib) for the Treatment of Patients with Relapsed or Refractory Mantle Cell Lymphoma

BeiGene and NewBridge Pharmaceuticals are working together to rapidly advance BRUKINSA in the Middle East and North African (MENA) region

CAMBRIDGE, Mass. & BEIJING & DUBAI, United Arab Emirates–(BUSINESS WIRE)– BeiGene (NASDAQ: BGNE; HKEX: 06160), a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide, and NewBridge Pharmaceuticals, a specialty company in the Middle East and North Africa (MENA) regions established to bridge the access gap by partnering with global pharma and biotech companies, today announced that BRUKINSA® (zanubrutinib) has received approval from the Saudi Food and Drug Authority (SFDA) for the treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy. BeiGene and NewBridge Pharmaceuticals are working together to bring BRUKINSA to healthcare providers and people living with MCL in Saudi Arabia and other MENA markets following regulatory approvals.

“Non-Hodgkin’s lymphoma is a leading cause of cancer incidence and mortality in Saudi Arabia, representing a significantly unmet need for those living with diseases, such as MCL. BRUKINSA is a next-generation BTK inhibitor designed to improve tolerability issues often associated with the class and has demonstrated efficacy in clinical trials for patients with relapsed or refractory MCL,” said Dr. Ahmad Absi, Hematology Section Head at the National Guard Health Affairs in Jeddah, Kingdom of Saudi Arabia.

“We are delighted that MCL patients in Saudi Arabia will now have access to BRUKINSA, a potentially best-in-class BTK inhibitor. Driven by our belief—Cancer has no borders. Neither do we, BeiGene is committed to expanding access to high-impact medicines for all patients who need them. With approval in Saudi Arabia and in the United Arab Emirates earlier this year, we are working with NewBridge Pharmaceuticals to bring BRUKINSA to more patients in the MENA region,” commented Mohammed Al-Kapany, Senior Director of New Markets in MENA at BeiGene.

“The approval of BRUKINSA in Saudi Arabia represents an important milestone in our ongoing collaboration with BeiGene. With this differentiated BTK inhibitor now approved in two markets in the MENA region, we are one step closer to reaching patients living with MCL with new treatment options,” remarked Joe Henein, President and CEO of NewBridge Pharmaceuticals.

The recommended dose of BRUKINSA is either 160 mg twice daily or 320 mg once daily, taken orally with or without food. The dose may be adjusted for adverse reactions and reduced for patients with severe hepatic impairment and certain drug interactions.

 

About Mantle Cell Lymphoma (MCL)

Non-Hodgkin’s lymphoma (NHL) is the third most common cancer in Saudi Arabia,1 with approximately 1,700 new cases in 2020.2 MCL is rare form of NHL, accounting for five percent of all cases. It develops in the outer edge of a lymph node called the mantle zone. Mantle cell lymphoma occurs more often in men than in women. It is usually diagnosed in people in their early 60s.3 MCL has a poor prognosis, with a median survival of three to four years, and is often diagnosed at a later stage of disease.4

 

About BRUKINSA

BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.

BRUKINSA is approved in the following indications and regions:

  • For the treatment of mantle cell lymphoma (MCL) in adult patients who have received at least one prior therapy (United States, November 2019)*;
  • For the treatment of MCL in adult patients who have received at least one prior therapy (China, June 2020)**;
  • For the treatment of chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) in adult patients who have received at least one prior therapy (China, June 2020)**;
  • For the treatment of relapsed or refractory MCL (United Arab Emirates, February 2021);
  • For the treatment of Waldenström’s macroglobulinemia (WM) in adult patients (Canada, March 2021);
  • For the treatment of adult patients with WM who have received at least one prior therapy (China, June 2021)**;
  • For the treatment of MCL in adult patients who have received at least one prior therapy (Canada, July 2021);
  • For the treatment of MCL in adult patients who have received at least one prior therapy (Chile, July 2021);
  • For the treatment of adult patients with MCL who have received at least one previous therapy (Brazil, August 2021);
  • For the treatment of adult patients with WM (United States, August 2021);
  • For the treatment of adult patients with marginal zone lymphoma (MZL) who have received at least one anti-CD20-based regimen (United States, September 2021)*;
  • For the treatment of adult patients with MCL who have received at least one previous therapy (Singapore, October 2021);
  • For the treatment of MCL in patients who have received at least one prior therapy (Israel, October 2021);
  • For the treatment of adult patients with WM who have received at least one prior therapy, or in first line treatment for patients unsuitable for chemo-immunotherapy (Australia, October 2021);
  • For the treatment of adult patients with MCL who have received at least one prior therapy (Australia, October 2021);
  • For the treatment of adult patients with MCL who have received at least one previous therapy (Russia, October 2021); and
  • For the treatment of adult patients with MCL who have received at least one previous therapy (Saudi Arabia, November 2021).

To date, more than 20 marketing authorization applications have been submitted for BRUKINSA for various indications.

* This indication was approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

** This indication was approved under conditional approval. Complete approval for this indication may be contingent upon results from ongoing randomized, controlled confirmatory clinical trials.

IMPORTANT U.S. SAFETY INFORMATION FOR BRUKINSA (ZANUBRUTINIB)

Warnings and Precautions

Hemorrhage

Fatal and serious hemorrhagic events have occurred in patients with hematological malignancies treated with BRUKINSA monotherapy. Grade 3 or higher hemorrhage including intracranial and gastrointestinal hemorrhage, hematuria and hemothorax have been reported in 3.4% of patients treated with BRUKINSA monotherapy. Hemorrhage events of any grade occurred in 35% of patients treated with BRUKINSA monotherapy.

Bleeding events have occurred in patients with and without concomitant antiplatelet or anticoagulation therapy. Co-administration of BRUKINSA with antiplatelet or anticoagulant medications may further increase the risk of hemorrhage.

Monitor for signs and symptoms of bleeding. Discontinue BRUKINSA if intracranial hemorrhage of any grade occurs. Consider the benefit-risk of withholding BRUKINSA for 3-7 days pre- and post-surgery depending upon the type of surgery and the risk of bleeding.

Infections

Fatal and serious infections (including bacterial, viral, or fungal) and opportunistic infections have occurred in patients with hematological malignancies treated with BRUKINSA monotherapy. Grade 3 or higher infections occurred in 27% of patients, most commonly pneumonia. Infections due to hepatitis B virus (HBV) reactivation have occurred.

Consider prophylaxis for herpes simplex virus, pneumocystis jiroveci pneumonia and other infections according to standard of care in patients who are at increased risk for infections. Monitor and evaluate patients for fever or other signs and symptoms of infection and treat appropriately.

Cytopenias

Grade 3 or 4 cytopenias, including neutropenia (26%), thrombocytopenia (11%) and anemia (8%) based on laboratory measurements, developed in patients treated with BRUKINSA monotherapy. Grade 4 neutropenia occurred in 13% of patients, and Grade 4 thrombocytopenia occurred in 3.6% of patients.

Monitor complete blood counts regularly during treatment and interrupt treatment, reduce the dose, or discontinue treatment as warranted. Treat using growth factor or transfusions, as needed.

Second Primary Malignancies

Second primary malignancies, including non-skin carcinoma, have occurred in 14% of patients treated with BRUKINSA monotherapy. The most frequent second primary malignancy was non-melanoma skin cancer, reported in 8% of patients. Other second primary malignancies included malignant solid tumors (4.0%), melanoma (1.7%) and hematologic malignancies (1.2%). Advise patients to use sun protection and monitor patients for the development of second primary malignancies.

Cardiac Arrhythmias

Atrial fibrillation and atrial flutter were reported in 3.2% of patients treated with BRUKINSA monotherapy. Patients with cardiac risk factors, hypertension, and acute infections may be at increased risk. Grade 3 or higher events were reported in 1.1% of patients treated with BRUKINSA monotherapy. Monitor signs and symptoms for atrial fibrillation and atrial flutter and manage as appropriate.

Embryo-Fetal Toxicity

Based on findings in animals, BRUKINSA can cause fetal harm when administered to a pregnant woman. Administration of zanubrutinib to pregnant rats during the period of organogenesis caused embryo-fetal toxicity including malformations at exposures that were 5 times higher than those reported in patients at the recommended dose of 160 mg twice daily. Advise women to avoid becoming pregnant while taking BRUKINSA and for 1 week after the last dose. Advise men to avoid fathering a child during treatment and for 1 week after the last dose.

If this drug is used during pregnancy, or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential hazard to a fetus.

Adverse reactions

The most common adverse reactions, including laboratory abnormalities, in ≥ 30% of patients who received BRUKINSA (N = 847) included decreased neutrophil count (54%), upper respiratory tract infection (47%), decreased platelet count (41%), hemorrhage (35%), decreased lymphocyte count (31%), rash (31%) and musculoskeletal pain (30%).

Drug Interactions

CYP3A Inhibitors: When BRUKINSA is co-administered with a strong CYP3A inhibitor, reduce BRUKINSA dose to 80 mg once daily. For coadministration with a moderate CYP3A inhibitor, reduce BRUKINSA dose to 80 mg twice daily.

CYP3A Inducers: Avoid coadministration with moderate or strong CYP3A inducers.

Specific Populations

Hepatic Impairment: The recommended dose of BRUKINSA for patients with severe hepatic impairment is 80 mg orally twice daily.

Please see full U.S. Prescribing Information at www.beigene.com/PDF/BRUKINSAUSPI.pdf and Patient Information at www.beigene.com/PDF/BRUKINSAUSPPI.pdf.

BeiGene Oncology

BeiGene is committed to advancing hematology, immuno-oncology and targeted therapies in order to bring impactful and affordable medicines to patients across the globe. We have a growing R&D team of approximately 2,300 colleagues dedicated to advancing more than 90 clinical trials involving more than 13,000 patients and healthy subjects. Our expansive portfolio is directed by a predominantly internalized clinical development team supporting trials in more than 40 countries or regions. We currently market three medicines discovered and developed in our labs: BTK inhibitor BRUKINSA in the United States, China, Canada, Australia and additional international markets; and non-FC-gamma receptor binding anti-PD-1 antibody tislelizumab and PARP inhibitor pamiparib in China. BeiGene has a high quality, innovative science and medicine organization and is a leader in China with a large oncology focused commercial team.

BeiGene also partners with innovative companies who share our goal of developing therapies to address global health needs. We commercialize a range of oncology medicines in China licensed from Amgen and Bristol Myers Squibb. We also plan to address greater areas of unmet need globally through our collaborations including with Amgen, Bio-Thera, EUSA Pharma, Mirati Therapeutics, Seagen, and Zymeworks. BeiGene has also entered into a collaboration with Novartis granting Novartis rights to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.

 

About BeiGene

BeiGene is a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide. With a broad portfolio of more than 40 clinical candidates, we are expediting development of our diverse pipeline of novel therapeutics through our own capabilities and collaborations. We are committed to radically improving access to medicines for two billion more people by 2030. BeiGene has a growing global team of over 7,700 colleagues across five continents. To learn more about BeiGene, please visit www.beigene.com and follow us on Twitter at @BeiGeneGlobal.

 

About NewBridge Pharmaceuticals

NewBridge Pharmaceuticals is a regional specialty company with a comprehensive pharmaceutical platform of services and expertise, established to bridge the access gap and partner with global pharma and biotech companies to in-license and commercialize U.S. Food and Drug Administration or European Medicines Agency approved innovative therapeutics that address unmet medical needs into the Middle East and North Africa (MENA) regions.

For more information, please visit www.nbpharma.com.

 

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding plans for development and commercialization of BRUKINSA in Saudi Arabia, MENA and other markets, the potential commercial opportunity for BRUKINSA, plans for making BRUKINSA accessible to patients in Saudi Arabia, MENA and other markets, the potential for BRUKINSA to be a best-in-class BTK inhibitor and to provide improved clinical benefits to patients, and BeiGene’s plans, commitments, aspirations and goals under the headings “BeiGene Oncology” and “About BeiGene”. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeiGene’s ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; BeiGene’s ability to achieve commercial success for its marketed medicines and drug candidates, if approved; BeiGene’s ability to obtain and maintain protection of intellectual property for its medicines and technology; BeiGene’s reliance on third parties to conduct drug development, manufacturing and other services; BeiGene’s limited experience in obtaining regulatory approvals and commercializing pharmaceutical products and its ability to obtain additional funding for operations and to complete the development and commercialization of its drug candidates and achieve and maintain profitability; the impact of the COVID-19 pandemic on the BeiGene’s clinical development, regulatory, commercial, and other operations, as well as those risks more fully discussed in the section entitled “Risk Factors” in BeiGene’s most recent quarterly report on Form 10-Q as well as discussions of potential risks, uncertainties, and other important factors in BeiGene’s subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeiGene undertakes no duty to update such information unless required by law.

 

References:

  1. Rauf MS, Akhtar S, Maghfoor I. Changing trends of adult lymphoma in the Kingdom of Saudi Arabia – comparison of data sources. Asian Pac J Cancer Prev. 2015;16(5):2069-72. doi: 10.7314/apjcp.2015.16.5.2069. PMID: 25773852.
  2. Globocan 2020. Available at https://gco.iarc.fr/today/data/factsheets/populations/682-saudi-arabia-fact-sheets.pdf. Accessed November 2021.
  3. Lymphoma Research Foundation. Understanding Mantle Cell Lymphoma. Available at https://lymphoma.org/wp-content/uploads/2018/10/MantleCellLymphomaFact-Sheet.pdf. Accessed October 2021.
  4. Philip J. Bierman, James O. Armitage, in Goldman’s Cecil Medicine (Twenty Fourth Edition), 2012.

View source version on businesswire.comhttps://www.businesswire.com/news/home/20211114005288/en/

Source Article: BeiGene

Pharming signs agreement with NewBridge Pharmaceuticals for the commercialization of RUCONEST® in the Middle East and North Africa

Pharming Group N.V. (“Pharming” or “the Company”) (Euronext Amsterdam: PHARM/Nasdaq: PHAR) announces it has entered into an exclusive licence agreement with
NewBridge Pharmaceuticals (“NewBridge”) for the distribution of RUCONEST® (conestat alfa) in the
Middle East and North Africa (“MENA”).
NewBridge, headquartered in Dubai, United Arab Emirates, is a regional specialty company, with a
comprehensive pharmaceutical platform of services and expertise, established to bridge the access gap
and partner with global pharma and biotech companies to in-license and commercialize US Food and
Drug Administration (FDA) or European Medicines Agency (EMA) approved innovative therapeutics that
address unmet medical needs into MENA.
Under the terms of the agreement, NewBridge will work closely with Pharming to provide access for
RUCONEST® for the treatment of acute hereditary angioedema (“HAE”) in MENA. NewBridge will be
responsible for the named patient supply and, where applicable, marketing of RUCONEST® in the
region.
RUCONEST® is a plasma-free recombinant human C1 esterase inhibitor (“rhC1INH”) protein
replacement therapy approved for the treatment of acute attacks of HAE in adults and children aged
two years and over.
RUCONEST® is approved by the FDA and EMA and commercialised in over 20
countries.
HAE is a rare genetic condition characterized by recurrent, unpredictable episodic swellings of mucosal
or cutaneous sites, causing pain, disfigurement, and disability which last for hours, and occasionally,
several days. For patients, the disorder is disabling and can be fatal if not treated.

Sijmen de Vries, Chief Executive Officer, Pharming Group commented:
“Pharming is committed to supporting patients with HAE, along with their caregivers, as they live with
this debilitating disease. We are therefore delighted to enter into this agreement with NewBridge to
ensure access to RUCONEST® in new geographies. NewBridge’s extensive experience in the Middle East
and North Africa, along with their strategic focus in rare diseases, make them an ideal partner for
Pharming in the region. We look forward to continuing to expand the global reach of RUCONEST®, in
line with our growth strategy, to serve HAE patients with unmet medical needs.”

 

About RUCONEST®
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 NewBridge Pharmaceuticals
NewBridge Pharmaceuticals FZ LLC (NBP) is a privately owned company, established and headquartered
in Dubai, United Arab Emirates since 2010 with an extended physical reach across the Middle East and
North African countries (MENA).
NBP is a regional specialty pharmaceutical organization with a comprehensive platform of services and
in-depth local expertise, focusing on licensing and commercializing USFDA & EMA approved innovative
therapeutics that address unmet medical needs in the MENA Region, and offering a one-stop solution
to access multiple geographies to companies wanting to expand their presence in the MENA region.

 

About Pharming Group N.V.
Pharming Group N.V. is a global, commercial stage biopharmaceutical company developing innovative
protein replacement therapies and precision medicines for the treatment of rare diseases and unmet
medical needs.
The flagship of our portfolio is our recombinant human C1 esterase inhibitor (rhC1INH) franchise. C1INH
is a naturally occurring protein that down regulates the complement and contact cascades in order to
control inflammation in affected tissues.
Our lead product, RUCONEST®, is the first and only plasma-free rhC1INH protein replacement therapy.
It is approved for the treatment of acute hereditary angioedema (HAE) attacks. We are commercializing
RUCONEST® in the United States, the European Union and the United Kingdom through our own sales
and marketing organization, and the rest of the world through our distribution network.
In addition, we are investigating the clinical efficacy of rhC1INH in the treatment of further indications,
including pre-eclampsia, acute kidney injury, and severe pneumonia as a result of COVID-19 infections.
Furthermore, we are leveraging our transgenic manufacturing technology to develop next-generation
protein replacement therapies, most notably for Pompe disease, which is currently in preclinical
development.

 

Forward-looking Statements
This press release contains forward-looking statements, including with respect to timing and progress
of Pharming’s preclinical studies and clinical trials of its product candidates, Pharming’s clinical and
commercial prospects, Pharming’s ability to overcome the challenges posed by the COVID-19 pandemic
to the conduct of its business, and Pharming’s expectations regarding its projected working capital
requirements and cash resources, which statements are subject to a number of risks, uncertainties and
assumptions, including, but not limited to the scope, progress and expansion of Pharming’s clinical trials
and ramifications for the cost thereof; and clinical, scientific, regulatory and technical developments. In
light of these risks and uncertainties, and other risks and uncertainties that are described in Pharming’s
2020 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2020 filed
with the U.S. Securities and Exchange Commission, the events and circumstances discussed in such
forward-looking statements may not occur, and Pharming’s actual results could differ materially and
adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of
the date of this press release and are based on information available to Pharming as of the date of this
release.

 

References
1. Ruconest Summary of Product Characteristics. Available at:
https://www.medicines.org.uk/emc/product/8580/smpc#gref
2. Longhurst H, et al. Lancet 2012;379(9814):474-481.

the CEO of NewBridge Pharmaceuticals Interview with CNBC

Watch CNBC Arabia’s episode “Made in UAE” Documentary featuring Joe Henein, President and CEO of NewBridge Pharmaceuticals, and his colleagues talk about NewBridge’s Journey on its 10th year anniversary, highlighting the UAE’s support for entrepreneurship and diversity on the link below

https://www.youtube.com/watch?v=osXyHOa_Fno&t=325s

 

 

CAPHOSOL® CAPHOSOL DISPERSIBLE®

Description

Caphosol® is indicated for the treatment and prevention of oral mucositis, a complication of cancer treatment (including radiation and chemotherapy). It contains a high concentration of phosphate and calcium ions to lubricate the mucosa of the mouth, tongue and oropharynx and help maintain the integrity of the oral cavity. Caphosol® is available as ampules or in dispersible form.

Latuda

Name LATUDA® (Lurasidone)
Description LATUDA® is a once-daily oral atypical antipsychotic. In addition to its well-demonstrated efficacy, LATUDA® with its favorable metabolic safety profile may be a solution for unmet medical needs of schizophrenia patients treated with current antipsychotics.
Indication LATUDA® is indicated for the treatment of schizophrenia in adults aged 18 years and over.
Strengths

18.5 mg, 37 mg and 74 mg film coated tablets

Regulatory Status

FDA approved in October 2010

EMA approved in March 2014

Latuda® tablets are registered in UAE and KSA.

Business Partner

Sunovion Pharmaceuticals

 

Guardant360

Description

Guardant360® assay is a liquid biopsy that provides fast, accurate and comprehensive genomic results from a simple blood draw. Test results are available in approximately seven days upon receipt in the US laboratory.

The test is done using circulating tumor DNA (ctDNA), which is produced when tumors shed small pieces or their genetic material into the bloodstream.

Guardant360® assay examines a panel of 74 genes (specifications) to identify any alterations in the tumor DNA.

Since its launch in 2014, Guardant360® assay has been ordered by more than 6,000 oncologists over 100,000 times for patients with advanced cancer to help select treatment.

Use and indication
  • Advanced stage – solid tumor -cancer patient with a solid tumor, whose tissue biopsy is insufficient for genetic testing.
  • Advanced stage cancer patient who wants to identify targeted therapy options while avoiding an invasive repeated tissue biopsy.
To know more about Guardant360® assay please visit the https://www.guardanthealthamea.com or click on this link