Alexion Pharmaceutic...
NasdaqGM:ALXN
$
182,50
$
+
$3,05 (1,70%)
182,50
$
+$3,05 (1,70%)
End-of-day quote: 07/20/2021
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Alexion Pharmaceuticals Company Info
EPS Growth 5Y
0,00%
Market Cap
$40,34 B
Long-Term Debt
$0,00 B
Short Interest
0,00%
Annual earnings
N/A
Dividend
$0,00
Dividend Yield
0,00%
Founded
1992
Industry
Country
ISIN Number
Website
Analyst Price Target
There are currently no price targets available for this stock.
In the last five quarters, Alexion Pharmaceuticals’s Price Target has risen from $177,38 to $177,38 - a 0,00% increase.
Top growth stocks in the health care sector (5Y.)
What does Alexion Pharmaceuticals do?
Alexion Pharmaceuticals, Inc. and its subsidiaries operate as a biopharmaceutical company worldwide.
The company focuses on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines.
The company has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), as well as the...
Alexion Pharmaceuticals, Inc. and its subsidiaries operate as a biopharmaceutical company worldwide.
The company focuses on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines.
The company has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), as well as the first and only approved complement inhibitor to treat anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD) in patients who are anti-aquaporin-4 (AQP4) antibody positive.
The company also has two enzyme replacement therapies and the first and only approved therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D). With the acquisition of Portola Pharmaceuticals, Inc. (Portola) in July 2020, the company added the first and only approved Factor Xa inhibitor reversal agent for patients treated with rivaroxaban or apixaban when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding.
In addition to the company’s marketed therapies, it has a pipeline resulting from internal innovation and business development. The company focuses its research efforts on novel molecules and targets in the complement cascade and development efforts on the core therapeutic areas of hematology, nephrology, neurology, metabolic disorders, cardiology, ophthalmology and acute care.
Marketed Products
ULTOMIRIS (ALXN1210/ravulizumab-cwvz)
ULTOMIRIS is an innovative, long-acting C5 inhibitor discovered and developed by the company that works by inhibiting the C5 protein in the terminal complement cascade. In clinical studies, ULTOMIRIS demonstrated rapid, complete, and sustained reduction of free C5 levels.
In December 2018, ULTOMIRIS was approved by the U.S. Food and Drug Administration (FDA) as a new treatment option for adult patients with PNH in the U.S.
ULTOMIRIS was approved as a new treatment option for adult patients with PNH by Japan's Ministry of Health, Labour, and Welfare (MHLW) in June 2019. ULTOMIRIS was approved by the European Commission (EC) in July 2019 as a treatment for adult patients with PNH with hemolysis with clinical symptoms indicative of high disease activity, and also for adult patients who are clinically stable after having been treated with SOLIRIS for at least the past six months.
In October 2019, the FDA approved the use of ULTOMIRIS as a treatment for adult and pediatric (one month of age or older) patients with aHUS to inhibit complement-mediated thrombotic microangiopathy (TMA).
In June 2020, the EC approved ULTOMIRIS for the treatment of adults and children with a body weight of 10kg or above with aHUS who are complement inhibitor treatment-naïve or have received SOLIRIS (eculizumab) for at least three months and have evidence of response to eculizumab.
In September 2020, ULTOMIRIS was approved by Japan's MHLW as a new treatment option for adult and pediatric patients with aHUS.
In October 2020, ULTOMIRIS 100 mg/mL formulation was approved by the FDA for the treatment of adult patients with PNH and for adults and pediatric (one month of age or older) patients with aHUS to inhibit complement-mediated TMA. In November 2020, ULTOMIRIS 100 mg/mL formulation was approved by the EC for the treatment of PNH and aHUS. The 100 mg/mL formulation is a higher concentration formulation of ULTOMIRIS than the formulation initially approved in December 2018. ULTOMIRIS 100 mg/mL reduces average annual infusion times by approximately 60 percent compared to ULTOMIRIS 10 mg/mL while delivering safety and efficacy consistent with the ULTOMIRIS 10 mg/mL formulation.
SOLIRIS (eculizumab)
SOLIRIS is an innovative C5 inhibitor discovered and developed by the company that works by inhibiting the C5 protein in the terminal complement cascade. SOLIRIS is a humanized monoclonal antibody that effectively blocks terminal complement activity at the doses currently prescribed.
SOLIRIS is approved for the treatment of PNH and aHUS in pediatric and adult patients in the U.S., Europe, Japan and in several other countries. The company is sponsoring multinational registries to gather information regarding the natural history of patients with PNH and aHUS, and the longer-term outcomes during anti-C5 treatment.
In 2017, the FDA and EC regulatory authorities approved SOLIRIS for the treatment of gMG in adults who are anti-acetylcholine receptor (AChR) antibody-positive. Additionally, in 2017, the MHLW in Japan approved SOLIRIS as a treatment for patients with gMG who are AChR antibody-positive and whose symptoms are difficult to control with high-dose intravenous immunoglobulin therapy or plasmapheresis (PLEX).
In June 2019, SOLIRIS became the first FDA-approved treatment option for adult patients with NMOSD who are AQP4 auto antibody positive. In August 2019, the EC approved SOLIRIS as the first treatment in Europe for NMOSD in adults who are AQP4 antibody-positive with a relapsing course of the disease. In November 2019, the Japanese MHLW approved SOLIRIS as a treatment for the prevention of relapse in patients with AQP4 antibody-positive NMOSD, including Neuromyelitis Optica.
STRENSIQ (asfotase alfa)
STRENSIQ, a targeted enzyme replacement therapy, is the first and only approved therapy for patients with HPP and is designed to directly address underlying causes of HPP by aiming to restore the genetically defective metabolic process, thereby preventing or reversing the severe and potentially life-threatening complications in patients with HPP. STRENSIQ is approved in the U.S. for patients with perinatal-, infantile- and juvenile-onset HPP, in Europe for the treatment of patients with pediatric-onset HPP, and in Japan for the treatment of patients with HPP. The company is sponsoring a multinational registry to gather information regarding the natural history of patients with HPP and the longer-term outcomes during STRENSIQ treatment.
KANUMA (sebelipase alfa)
KANUMA, a recombinant form of the human LAL enzyme, is the only enzyme-replacement therapy that is approved for the treatment for patients with LAL-D. KANUMA is approved in the U.S. for the treatment of patients with LAL-D, in Europe for long-term enzyme replacement therapy in patients with LAL-D, and in Japan for the treatment of patients with LAL-D. The company is sponsoring a multinational registry to gather information regarding the natural history of patients with LAL-D and the longer-term outcomes during KANUMA treatment.
ANDEXXA (coagulation factor Xa - [recombinant] inactivated-zhzo)
ANDEXXA is approved by the FDA as a reversal agent for patients treated with rivaroxaban or apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding. ANDEXXA was approved under the FDA’s Accelerated Approval Pathway, and received conditional marketing authorization in the European Union (EU) based on the change from baseline in anti-Factor Xa activity in healthy volunteers and in patients through the ANNEXXA-4 trial demonstrating hemostatic efficacy. Continued approval for this indication is contingent upon post-marketing study results that verify that clinical benefit is conferred to patients.
Clinical Development Programs
In addition to the company’s ongoing development programs, the company holds a minority interest in and an option to acquire Caelum Biosciences (Caelum), a biotechnology company that is developing CAEL-101 for light chain (AL) amyloidosis. CAEL-101 is a chimeric monoclonal antibody (mAb) designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis, a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. A Phase Ia/Ib study for CAEL-101 has been completed. Following discussions with the FDA, a Phase II trial for CAEL-101 commenced during the first quarter of 2020. The trial met its primary objectives, supporting the safety and tolerability of CAEL-101 and confirmed the dose and regimen to be adopted for the Phase III studies.
In September 2020, the company and Caelum announced the initiation of the Cardiac Amyloid Reaching for Extended Survival (CARES) program. This includes two parallel Phase III trials to evaluate the survival benefits of CAEL-101. Dosing is underway in the two parallel Phase 3 studies; one in patients with Mayo stage IIIa disease and one in patients with Mayo stage IIIb disease.
ULTOMIRIS (ALXN1210/ravulizumab-cwvz)
ULTOMIRIS is an innovative, long-acting C5 inhibitor discovered and developed by the company that works by inhibiting the C5 protein in the terminal complement cascade. In clinical studies, ALXN1210 demonstrated rapid, complete, and sustained reduction of free C5 levels.
Intravenous (IV)
In January 2019, the company announced that the Phase III, global, single arm, multicenter study evaluating the safety and efficacy of ALXN1210 administered by IV infusion every 8 weeks to adult patients with aHUS who had never been treated with a complement inhibitor (inhibitor-naïve patients) met its primary objective. A second Phase III, single arm, multicenter study to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmaco-dynamics (PD) of ALXN1210 administered by IV infusion every 8 weeks in inhibitor-naïve pediatric patients (including adolescents) with aHUS is ongoing.
In March 2019, the company initiated a Phase III double-blind, placebo-controlled, multicenter study to evaluate the safety and efficacy of ALXN1210 in adult patients for the treatment of gMG. Additionally, in December 2019, the company initiated a Phase III, single arm, open-label, multicenter study to evaluate the safety and efficacy of ALXN1210 in adult patients with NMOSD.
In March 2020, the company initiated a Phase III, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of ALXN1210 in patients with amyotrophic lateral sclerosis (ALS).
In May 2020, following the FDA's acceptance of the company’s investigational new drug (IND) application, the company initiated a Phase III open-label, randomized, controlled clinical trial of ALXN1210 in adult patients with coronavirus (COVID-19), who are hospitalized with severe COVID-19 requiring mechanical ventilation. The trial is investigating the role of terminal complement inhibition in managing patients with severe COVID-19. In January 2021, the company paused further enrollment in this study due to lack of efficacy, pending further analysis of the data. This decision was made based on the recommendation of an independent data monitoring committee (IDMC), following their review of data from a pre-specified interim analysis.
In August 2020, the company submitted an application to the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) to register the ULTOMIRIS 100mg/ml formulation. The 100 mg/mL formulation is a higher concentration formulation of ULTOMIRIS than the formulation initially approved in December 2018. ULTOMIRIS 100 mg/mL reduces average annual infusion times by approximately 60 percent compared to ULTOMIRIS 10 mg/mL while delivering safety and efficacy consistent with the ULTOMIRIS 10 mg/mL formulation.
In December 2020, the company initiated a Phase III, multicenter study to evaluate the safety and efficacy of ALXN1210 in hematopoietic stem cell transplantation associated thrombotic microangiopathy (HSCT-TMA).
In addition to aHUS, NMOSD, gMG, ALS, COVID-19 and HSCT-TMA, the company plans to initiate a Phase III study of ALXN1210 in complement mediated thrombotic microangiopathy (CM-TMA); a proof of concept basket study in renal indications, including Lupus Nephritis (LN) and Immunoglobulin A Nephropathy (IgAN); and a Phase II/III study in dermatomyositis (DM), a rare autoimmune inflammatory myopathy characterized by chronic inflammation and degeneration of muscle and skin.
Subcutaneous (SC) Delivery
In March 2019, the company initiated a PK-based Phase III study of ALXN1210 delivered subcutaneously once per week to PNH patients to support regulatory approval submissions in both PNH and aHUS. In June 2020, the company announced that the ongoing study met its primary objective of pharmacokinetic-based non-inferiority of ULTOMIRIS SC versus IV ULTOMIRIS at Day 71. Pending completion of the study and collection of required 12-month safety data, the company expects to file for approval in the U.S. and E.U. for the SC formulation and device combination in PNH and aHUS in the third quarter of 2021.
ALXN1720
ALXN1720 is a novel humanized bi-specific minibody that binds selectively and with high affinity to C5 and to albumin. ALXN1720 is designed for subcutaneous administration as a concentrated formulation for the treatment of disease states involving dysregulated terminal complement activity. In September 2019, the company initiated a Phase I healthy volunteer study of ALXN1720 to assess its safety and tolerability. This trial was paused due to the COVID-19 pandemic, but was re-initiated in August 2020. This trial was paused again in fourth quarter 2020 due to a second wave of COVID-19 and the company expects to re-initiate it in the second quarter of 2021. Additionally, pending successful completion of the Phase I healthy volunteer study, the company plans to initiate ALXN1720 trials in gMG and DM.
ALXN1820
ALXN1820 is a bispecific minibody binding to properdin and albumin and is a first-in-class therapeutic antagonist of properdin, with potent, selective activity, attractive PK-PD, subcutaneous bioavailability and safety. In December 2020, the company submitted a Clinical Trial Application to Human Research Ethics Committees in Australia.
Approval was received and notification was sent to Australia’s Therapeutic Goods Administration for ALXN1820 in December 2020. In the first quarter of 2021, the company initiated a healthy volunteers Phase I study. Early preclinical data for ALXN1820 indicate the potential for convenient, weekly, self-administered subcutaneous dosing. There are multiple potential indications for ALXN1820 across a number of therapeutic areas, including hematology, pulmonology, nephrology and dermatology, where properdin is believed to play an important role.
ALXN1830 (SYNT001)
ALXN1830 is a humanized monoclonal antibody that is designed to inhibit the interaction of the neonatal Fc receptor (FcRn) with Immunoglobulin G (IgG) and IgG immune complexes and has the potential to improve treatment in a number of rare IgG-mediated diseases. The company initiated a Phase I study of a SC formulation of ALXN1830 in healthy volunteers in December 2019.
The company re-initiated a Phase II trial of the IV formulation in Warm Autoimmune Hemolytic Anemia (WAIHA) in early 2020. Due to the COVID-19 pandemic, the company discontinued the Phase II trial in WAIHA and the Phase I healthy volunteer study. A new Phase I healthy volunteer study is planned to be initiated in the first quarter of 2021, while the Phase II studies in WAIHA and gMG exclusively with the SC formulation are planned to start in the second half of 2021.
ALXN1840 (WTX101)
ALXN1840, an innovative product candidate that addresses the underlying cause of Wilson disease, is a first-in-class oral copper-binding agent with a unique mechanism of action and the ability to access and mobilize copper from tissue.
In February 2020, the company completed enrollment in a Phase III study of ALXN1840 for the treatment of Wilson disease. The company expects top-line results from this Phase III study in the first half of 2021.
ALXN1850
ALXN1850 is an Enzyme Replacement Therapy replacing deficient alkaline phosphatase (ALP) activity and targets ALP substrates to improve bone mineralization and ameliorate systemic manifestations of the disease. It is a next generation HPP therapy that is designed to be provide higher activity, higher bioavailability, and longer half-life than STRENSIQ (asfotase alfa). These improvements may result in significant benefit for HPP patients, including potentially lower, less frequent doses, improved efficacy and lower injection volumes when compared to STRENSIQ.
In November 2020, the company submitted an IND for ALXN1850 to the FDA and received approval to proceed with a Phase I study in HPP patients. The company plans to initiate the Phase I study in the second quarter of 2021. ALXN1850 is designed for subcutaneous administration.
ALXN2040 (danicopan/ACH-4771)
ALXN2040 is an oral Factor D inhibitor designed to treat diseases associated with dysregulation of the complement alternative pathway. ALXN2040 as an add on therapy to anti-C5 for PNH patients with clinically evident extravascular hemolysis (EVH) has received orphan drug and breakthrough therapy designation by the FDA and both orphan and PRIME designation by the European Medicines Agency (EMA). Two Phase II studies of ALXN2040 as an oral add-on therapy for PNH-EVH are ongoing, and a Phase III trial for PNH-EVH was initiated, with dosing of the first patient in the first quarter of 2021. Two Phase II studies previously initiated evaluating the safety, efficacy, pharmacokinetics, and pharmaco-dynamics of ALXN2040 in patients with C3G have been discontinued and the company is no longer pursuing ALXN2040 as a treatment for C3G. The company plans to develop ALXN2050 in a variety of orphan renal indications, including C3G.
The company plans to initiate a Phase II study of ALXN2040 in Geographic Atrophy, a chronic and progressive degeneration of the portion of the retina responsible for central and color vision and leading to permanent loss of visual acuity, in the second half of 2021.
ALXN2040 was added to the pipeline as a result of the Achillion (Achillion Pharmaceuticals, Inc.) acquisition.
ALXN2050 (ACH-5228)
ALXN2050 is an oral Factor D inhibitor designed to treat diseases associated with dysregulation of the complement alternative pathway. ALXN2050 is in a Phase II trial as a potential monotherapy treatment for PNH and is being evaluated for development in other alternative pathway-mediated rare diseases. The company has paused additional enrollment in the Phase II study of ALXN2050 monotherapy that is underway in PNH patients, pending the receipt of further Phase I data (expected in the second quarter of 2021) that will allow for dose escalation in the Phase II study. Additionally, the company plans to initiate proof-of-concept trials of ALXN2050 in patients with various renal diseases in the first half of 2021.
ALXN2050 was added to the pipeline as a result of the Achillion acquisition.
ALXN2060 (AG10)
In September 2019, the company entered into an agreement with Eidos Therapeutics, Inc. (Eidos), through which the company obtained an exclusive license to develop and commercialize AG10 in Japan for transthyretin amyloidosis (ATTR). AG10 is an orally administered small molecule in development designed to target the root cause of ATTR by stabilizing transthyretin (TTR) in the blood. Eidos is studying AG10 in a Phase III clinical trial in patients with ATTR cardiomyopathy (ATTR-CM) and a Phase III clinical trial in patients with ATTR polyneuropathy (ATTR-PN), excluding Japan.
In the fourth quarter 2020, the company initiated a single arm AG10 Phase III study in ATTR-CM in Japan. The Japan Phase III study data for ATTR-CM is expected to bridge to the global randomized controlled Phase III study being conducted by Eidos, and serve as the basis for seeking regulatory approval to commercialize AG10 in Japan.
ALXN2070 (ANDEXXA)
The acquisition of Portola added Portola’s commercialized medicine, ANDEXXA, for which additional clinical trials are being conducted to obtain full regulatory approvals and to expand the approved indications. As noted above, ANDEXXA has obtained accelerated approval in the U.S. and conditional marketing authorization in the EU based on the change from baseline in anti-Factor Xa activity in healthy volunteers, and in patients through the ANNEXA-4 trial. Full approval in the U.S. and EU for current indications requires completion of ANNEXA-I, a Phase IV randomized controlled clinical trial currently underway evaluating the safety and efficacy of ANDEXXA versus standard of care in patients presenting with acute intracranial hemorrhage while taking an oral Factor Xa inhibitor. A Japan J-New Drug Application filing is planned for the first quarter 2021. A U.S. supplemental Biologics License Application (sBLA) submission for a label expansion for the reversal of edoxaban and enoxaparin associated bleeds occurred in the fourth quarter of 2020.
Additionally, the company plans to initiate a Phase II study in 2021 for the reversal of anticoagulation in patients taking apixaban, rivaroxaban, edoxaban, or enoxaparin who require urgent surgery.
ALXN2075 (Cerdulatinib)
ALXN2075 (Cerdulatinib) is a small molecule dual spleen tyrosine kinase and janus kinase (SYK/JAK) inhibitor in development for the treatment of hematological malignancies. The company is reviewing preliminary clinical data and considering Follicular Lymphoma as a next step in the program. A Phase I/IIa study is ongoing for the treatment of hematological malignancies. ALXN2075 was added to the pipeline portfolio as a result of the acquisition of Portola.
SOLIRIS (eculizumab)
SOLIRIS is an innovative C5 inhibitor discovered and developed by the company that works by inhibiting the C5 protein in the terminal complement cascade. SOLIRIS is a humanized monoclonal antibody that effectively blocks terminal complement activity at the doses currently prescribed.
In June 2020, Japan’s MHLW granted SAKIGAKE designation for SOLIRIS in Guillain-Barré syndrome (GBS). Results from the Japanese eculizumab trial for GBS (JET-GBS study) suggested the potential efficacy and safety of SOLIRIS as a treatment for GBS.
Complement activation may play a role in the pathophysiology of GBS. The company plans to initiate a Phase III study of SOLIRIS in GBS in Japan in the first half of 2021.
Manufacturing
The company has various agreements with Lonza Group AG and its affiliates (Lonza) to provide cell bank and drug substance through 2030.
Sales and Marketing
The company has established a commercial organization to support the sale of its products in the U.S., Europe, Japan, Latin America, the Asia Pacific countries, and other territories.
Customers
The company’s customers are primarily consisted of distributors, pharmacies, hospitals, hospital buying groups, and other healthcare providers. In some cases, the company also sells its products to governments and government agencies.
The company’s net product sales to three customers, AmerisourceBergen Corporation, McKesson Corporation, and Cardinal Health, Inc., each accounted for more than 10.0% of total revenues for the year ended December 31, 2020 and on a combined basis, accounted for approximately 47.4%.
Intellectual Property Rights
SOLIRIS Exclusivity
With respect to SOLIRIS, the company owns an issued U.S. patent that covers the eculizumab composition of matter that will expire in 2021, taking into account patent term extension. The company also owns other issued U.S. patents that cover the composition, use and formulation of eculizumab, which expire in 2027. SOLIRIS also benefits from orphan drug exclusivity for treating gMG until 2024 and for treating NMOSD until 2026 (orphan drug exclusivity for SOLIRIS for treating PNH and aHUS in the U.S. previously expired).
In Europe, SOLIRIS is protected by orphan drug exclusivity through late 2023 for aHUS, until 2027 for gMG and until 2029 for NMOSD (orphan drug exclusivity for SOLIRIS for treating PNH in Europe previously expired). In Japan, the company owns an issued patent that covers the eculizumab composition of matter and will expire in 2027. SOLIRIS is also protected in Japan by orphan drug exclusivity until 2027 for gMG and until 2029 for NMOSD (orphan drug exclusivity for SOLIRIS for treating PNH in Japan previously expired). In addition to the foregoing patent and regulatory protections, the company owns other patents and pending patent applications that are directed to various aspects of eculizumab and which may provide additional protection for SOLIRIS in the U.S., Europe, Japan and other countries.
In January 2019, the Opposition Division of the European Patent Office determined, following multi-party opposition proceedings, to revoke the company’s European patent No. 2359834, which relates to the formulation of SOLIRIS. This decision is under appeal.
While the company has the patent rights to SOLIRIS as detailed above, in August 2019, the U.S. Patent and Trademark Office instituted inter partes review (IPR) of three of the company’s patents that relate to SOLIRIS. In May 2020, the company entered into a Confidential Settlement and License Agreement with Amgen Inc. (Amgen) to settle the three IPRs (Settlement Agreement). Pursuant to the Settlement Agreement, the company and Amgen have terminated each of the pending IPRs, and effective March 1, 2025 (or an earlier date in certain circumstances), the company grants to Amgen (and its affiliates and certain partners) a non-exclusive, royalty-free, license under the U.S. patents and patent applications related to eculizumab and various aspects of the eculizumab product that the company markets and sells under the tradename SOLIRIS.
ULTOMIRIS Exclusivity
With respect to ULTOMIRIS, the company owns issued U.S., European and Japanese patents that cover the composition of matter, use and formulation of ravulizumab that will expire in 2035. ULTOMIRIS is also protected by orphan drug exclusivity for treating PNH in the U.S. until 2025 and in Japan until 2029. ULTOMIRIS is also protected in Japan by orphan drug exclusivity for treating PNH until 2029. In addition to the foregoing patent and regulatory protections, the company owns other patents and pending patent applications that are directed to various aspects of ULTOMIRIS and which may provide additional protection for ULTOMIRIS in the U.S., Europe, Japan and other countries.
STRENSIQ Exclusivity
With respect to STRENSIQ, the company owns an issued U.S. patent that covers the asfotase alfa composition of matter that will expire in 2029, including patent term restoration. STRENSIQ is also protected in the U.S. by orphan drug exclusivity until 2022. In Europe, the company owns two issued patents that cover the asfotase alfa composition of matter and these will expire in 2025 and 2028. Additionally, the company has received supplementary protection certificates that extend the patent protection until 2030 in many European countries. STRENSIQ is also protected in Europe by orphan drug exclusivity until 2027. In Japan, STRENSIQ is protected by an issued patent that covers the asfotase alfa composition of matter until 2028 and by orphan drug exclusivity until 2025. In addition to the foregoing patent and regulatory protections, the company owns other patents and pending patent applications that are directed to various aspects of STRENSIQ and which may provide additional protection for STRENSIQ in the U.S., Europe, Japan and other countries.
KANUMA Exclusivity
With respect to KANUMA, the company owns issued patents in the U.S., Europe and Japan that cover the composition of matter and/or methods of using the product to treat LAL-D that will expire in 2031. The company maintained the European patent in an opposition proceeding that was favorably resolved in 2017. An exclusively licensed composition of matter patent that has been extended to 2026 via supplementary protection certificates further protects KANUMA in certain European countries. KANUMA also is protected by orphan drug exclusivity until 2022, 2027 and 2026 in the U.S., Europe and Japan, respectively.
ANDEXXA/ONDEXXYA Exclusivity
With respect to ANDEXXA, the company owns an issued U.S. patent that covers the andexanet alfa composition of matter and has an expiration in 2030. A pending application for patent term extension could extend the expiration to 2032. ANDEXXA is also protected in the U.S. by orphan drug exclusivity until 2025. In Europe, the company owns two issued patents that cover the andexanet alfa composition of matter and have expirations in 2028.
Additionally, the company has applied for supplementary protection certificates that could extend the patent protection until 2033 in many European countries. In Japan, the company owns an issued patent that covers the andexanet alfa composition of matter and has an expiration in 2028. In addition to the foregoing patent and regulatory protections, the company owns other patents and pending patent applications that are directed to various aspects of ANDEXXA/ONDEXXYA and which may provide additional protection for ANDEXXA/ONDEXXYA in the U.S., Europe, Japan and other countries.
Investigational Compounds
The company also owns the U.S. and foreign patents and patent applications that protect its investigational compounds and product candidates.
Government Regulation
The company’s five approved products are regulated by the FDA as biologics. The company must register each controlled clinical trial, other than Phase I trials, on a website administered by the National Institutes of Health (NIH).
The company and its third party manufacturers are required under regulations to ensure that all of its processes, methods, and equipment are compliant with GCP, GLP, cGMP and pharmacovigilance rules.
In addition to participating in the Medicaid Drug Rebate Program, federal law requires manufacturers like the company to participate in the Public Health Service’s 340B drug pricing program in order for federal funds to be available for the manufacturer’s drugs under Medicaid and Medicare Part B.
However, ‘orphan drugs’ i.e., those designated under section 526 of the Federal Food, Drug, and Cosmetic Act, such as certain of the company’s products that have received market authorization (as identified above), are exempted from the ceiling price requirements for these eligible entities added by PPACA (the Patient Protection and Affordable Care Act, as amended by the Health Care and Education Reconciliation Act of 2010) (except for certain children’s hospitals).
To participate, the company is required to enter into an Federal Supply Schedule (FSS) contract and other agreements with the Department of Veterans Affairs (VA) for its products, which qualify as ‘covered drugs’. Under these agreements, the company must make its products available to the ‘Big Four’ federal agencies the VA, the Department of Defense (DoD), the Public Health Service (including the Indian Health Service), and the Coast Guard at pricing that is capped pursuant to a statutory federal ceiling price, or FCP, formula set forth in Section 603 of the Veterans Health Care Act of 1992 (VHCA).
In addition, pursuant to regulations issued by the DoD to implement Section 703 of the National Defense Authorization Act for Fiscal Year 2008, each of the company’s covered drugs is listed on an agreement with the Defense Health Agency (DHA) under which the company has agreed to honor the ‘Big Four’ pricing for the company’s products when they are dispensed to TRICARE beneficiaries by TRICARE retail network pharmacies.
PPACA contains several provisions that have an impact on the company’s business. In addition, most healthcare providers who prescribe and dispense the company’s products and research institutions with whom the company collaborates for its sponsored clinical trials are subject to privacy and security requirements under HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act (HITECH), and its implementing regulations.
The company is also subject to the U.S. Foreign Corrupt Practices Act (FCPA), the U.K. Bribery Act (U.K. Bribery Act), and other anti-corruption laws and regulations pertaining to its financial relationships and interactions with foreign government officials.
Research and Development
During the year ended December 31, 2020, the company incurred research and development expenses of $1,002.9.
History
Alexion Pharmaceuticals, Inc. was founded in 1992. The company was incorporated in 1992 under the laws of the state of Delaware.
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