Business Wire

Alexion Reaches Funding Agreement with NICE and NHS England for Strensiq® (asfotase alfa) for Patients with Pediatric-onset Hypophosphatasia (HPP)

Del

Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) today announced that it has reached a national funding agreement with the National Institute for Health and Care Excellence (NICE) and the National Health Service (NHS) England based on a Managed Access Agreement (MAA), which provides access to Strensiq® (asfotase alfa) for patients in England with pediatric-onset hypophosphatasia (HPP), regardless of their current age. The funding agreement was announced today in a positive final evaluation determination (FED) issued by the NICE Highly Specialised Technologies (HST) Evaluation Committee to recommend Strensiq according to the MAA.

The MAA has been developed in collaboration between physician thought-leaders, patient groups, NHS England, and Alexion. The MAA ensures access to Strensiq for infants, children and adult patients with pediatric-onset HPP who experience the most disabling symptoms and are expected to benefit most from therapy.

“It is a success that patients with HPP in England who meet the criteria of the Managed Access Agreement will have access to Strensiq, which is the only treatment for this severely debilitating and often life-threatening disease,” said Lindsay Weaver, Chief Executive, Children Living with Inherited Metabolic Diseases (CLIMB). “We are relieved that NICE, NHS England and Alexion have reached an agreement that benefits patients with pediatric-onset HPP most in need of treatment. We will be following the progress of the agreement, which involves the collection of robust data, to ensure continued access for patients.”

HPP is an ultra-rare metabolic disease characterized by defective bone formation that can lead to weakness and deformity of bones, fractures and other skeletal abnormalities, as well as complications such as profound muscle weakness, severe pain, seizures in perinatal/infantile forms of HPP, and respiratory failure potentially leading to premature death in infants.1-5

“We worked diligently and constructively with NICE, NHS England, advocates and physicians, and are extremely pleased that we were able to reach an agreement to make Strensiq available to patients with pediatric-onset HPP in England who are most in need of treatment,” said Ludwig Hantson, Chief Executive Officer of Alexion. "The decision to provide access to Strensiq is an important milestone for patients and their families."

Strensiq is approved in the European Union as a long-term enzyme replacement therapy in patients with pediatric-onset HPP. Strensiq is also approved in the United States for the treatment of patients with perinatal-, infantile- and juvenile-onset HPP, as well as in Japan and other countries. Alexion is currently progressing local funding processes for Strensiq in additional countries worldwide.

About Hypophosphatasia (HPP)

HPP is a genetic, chronic, progressive, and potentially life-threatening ultra-rare metabolic disease that can affect people of all ages. HPP is characterized by defective bone mineralization that can lead to weakness and deformity of bones, fractures and other skeletal abnormalities, as well as systemic complications such as profound muscle weakness, muscle, bone and joint pain, seizures in perinatal/infantile forms of HPP, and respiratory failure leading to premature death in infants. The signs, symptoms and severity of HPP can vary from patient to patient, and because of the progressive nature of the disease, new symptoms can appear at any age and symptoms can worsen over time, causing significant disability.1-5 HPP is traditionally classified by the age of the patient at the onset of symptoms of the disease, with perinatal-, infantile- and juvenile-onset HPP (also known as pediatric-onset HPP) defined by the onset of the first symptom prior to 18 years of age.1

HPP can have devastating consequences for patients at any stage of life.1 In a natural history study, infants who had their first symptom of HPP within the first 6 months of life had high mortality, with an overall mortality rate of 73 percent at 5 years.7 In these patients, mortality was primarily due to respiratory failure.1,7 In patients surviving and those with juvenile-onset HPP, long-term clinical sequelae include recurrent and non-healing fractures, profound muscle weakness, debilitating pain, and the requirement for ambulatory assistive devices such as wheelchairs, wheeled walkers, and canes.1,4

HPP is caused by mutations in the gene encoding an enzyme known as tissue non-specific alkaline phosphatase (TNSALP). This enzyme plays a critical role in the proper mineralization of bones.1,2

About Strensiq ® (asfotase alfa)

Strensiq (asfotase alfa) is a highly innovative bone-targeted enzyme replacement therapy that treats the underlying cause of HPP by replacing the missing TNSALP enzyme. In clinical studies of patients with HPP who had their first symptom prior to the age of 18, treatment with Strensiq improved overall survival in infants, enhanced bone mineralization and improved height, weight, and mobility.6,8

STRENSIQ IMPORTANT SAFETY INFORMATION

Hypersensitivity reactions, including anaphylaxis, have been reported in STRENSIQ-treated patients. Signs and symptoms consistent with anaphylaxis included difficulty breathing, choking sensation, nausea, periorbital edema, and dizziness. These reactions have occurred within minutes after subcutaneous administration of STRENSIQ and can occur in patients on treatment for more than one year. Other hypersensitivity reactions have also been reported in STRENSIQ-treated patients, including vomiting, fever, headache, flushing, irritability, chills, skin erythema, rash, pruritus and oral hypoesthesia. If a severe hypersensitivity reaction occurs, discontinue STRENSIQ treatment and initiate appropriate medical treatment. Consider the risks and benefits of re-administering STRENSIQ to individual patients following a severe reaction. If the decision is made to re-administer the product, monitor patients for a reoccurrence of signs and symptoms of a severe hypersensitivity reaction.

Localized lipodystrophy, including lipoatrophy and lipohypertrophy, has been reported at injection sites after several months in patients treated with STRENSIQ. Advise patients to follow proper injection technique and to rotate injection sites.

Patients with HPP are at increased risk for developing ectopic calcifications. In clinical trials, 14 cases (14%) of ectopic calcification of the eye, including the cornea and conjunctiva, and the kidneys (nephrocalcinosis) were reported. There was insufficient information to determine whether or not the reported events were consistent with the disease or due to STRENSIQ. No visual changes or changes in renal function were reported. Ophthalmology examinations and renal ultrasounds are recommended at baseline and periodically during treatment with STRENSIQ to monitor for signs and symptoms of ophthalmic and renal ectopic calcifications and for changes in vision or renal function. The most common adverse reactions (≥ 10%) are injection site reactions, lipodystrophy, ectopic calcifications and hypersensitivity reactions.

Please click here for the full Prescribing Information.

About Alexion

Alexion is a global biopharmaceutical company focused on developing and delivering life-transforming therapies for patients with devastating and rare disorders. Alexion is the global leader in complement inhibition and has developed and commercializes the first and only approved complement inhibitor to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two life-threatening ultra-rare disorders. In addition, Alexion’s metabolic franchise includes two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D). Alexion is advancing its rare disease pipeline with highly innovative product candidates in multiple therapeutic areas. This press release and further information about Alexion can be found at: www.alexion.com

[ALXN-G]

Forward-Looking Statements

This news release contains forward-looking statements, including statements related to potential medical benefits of Strensiq® (asfotase alfa) for hypophosphatasia (HPP). Forward-looking statements are subject to factors that may cause Alexion’s results and plans to differ from those expected, including, for example, risks and uncertainties of drug development, decisions of regulatory authorities regarding the adequacy of our research, marketing approval or material limitations on the marketing of Strensiq for HPP, delays, interruptions or failures in the manufacture and supply of our products and our product candidates, delays in establishing commercial infrastructure for Strensiq for HPP, the possibility that results of clinical trials are not predictive of safety and efficacy results of Strensiq in broader or different patient populations, the possibility that clinical trials of our product candidates could be delayed, the adequacy of our pharmacovigilance and drug safety reporting processes, the risk that third party payers (including governmental agencies) will not reimburse or continue to reimburse for the use of our products at acceptable rates or at all, risks regarding government investigations, including investigations of Alexion by the SEC and DOJ, the risk that anticipated regulatory filings are delayed, the risk that estimates regarding the number of patients with Strensiq and observations regarding the natural history of patients with Strensiq are inaccurate, risks related to potential disruptions to our business as a result of leadership changes, and a variety of other risks set forth from time to time in Alexion's filings with the Securities and Exchange Commission, including but not limited to the risks discussed in Alexion's Quarterly Report on Form 10-Q for the period ended March 31, 2017 and in Alexion's other filings with the SEC. Alexion does not intend to update any of these forward-looking statements to reflect events or circumstances after the date hereof, except when a duty arises under law.

References

1.   Rockman-Greenberg C. Hypophosphatasia. Pediatr Endocrinol Rev. 2013; 10 (suppl 2):380-388.
2. Whyte MP. Hypophosphatasia: nature’s window on alkaline phosphatase function in humans. In: Bilezikian JP, Raisz LG, Martin TJ, eds. Principles of Bone Biology. Vol 1. 3rd ed. San Diego, CA: Academic Press; 2008:1573-1598.
3. Whyte MP, Greenberg CR, Salman N, et al. Enzyme-replacement therapy in life-threatening hypophosphatasia. N Engl J Med. 2012; 366(10):904-913.
4. Seshia SS, Derbyshire G, Haworth JC, Hoogstraten J. Myopathy with hypophosphatasia. Arch Dis Child. 1990; 65(1):130-131.
5. Baumgartner-Sigl S, Haberlandt E, Mumm S, et al. Pyridoxine-responsive seizures as the first symptom of infantile hypophosphatasia caused by two novel missense mutations (c.677T>C, p.M226T; c.1112C>T, p.T371I) of the tissue-nonspecific alkaline phosphatase gene. Bone. 2007; 40(6):1655-1661.
6. Strensiq Summary of Product Characteristics. Alexion Pharmaceuticals.
7. Whyte MP, Leung E, Wilcox W, et al. Hypophosphatasia: a retrospective natural history study of the severe perinatal and infantile forms. Poster presented at the 2014 Pediatric Academic Societies and Asian Society for Pediatric Research Joint Meeting, Vancouver, B.C., Canada, May 5, 2014. Abstract 752416.
8.

Whyte MP, Rockman-Greenberg C, Onzono K, et al. Asfotase Alfa Treatment Improves Survival for Perinatal and Infantile Hypophosphatasia. The Journal of Clinical Endocrinology & Metabolism 2016; 101: 334-342.

Contact information

Alexion:
Media
Kim Diamond, +1 475-230-3775
Executive Director, Corporate Communications
or
Lauren Cettier, +41 (0)44 457 4323
Director, External Communications, EMEA
or
Investors
Elena Ridloff, CFA, +1 475-230-3601
Vice President, Investor Relations
or
Catherine Hu, +1 475-230-3599
Director, Investor Relations

Om Business Wire

Business Wire
Business Wire
24 Martin Lane
EC4R 0DR London

+44 20 7626 1982http://www.businesswire.co.uk

Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.

Følg saker fra Business Wire

Registrer deg med din epostadresse under for å få de nyeste sakene fra Business Wire på epost fortløpende. Du kan melde deg av når som helst.

Siste saker fra Business Wire

Madison Realty Capital Provides $64.0 Million Construction Financing for 200 Kent Avenue Development in Williamsburg, Brooklyn17.11.2017 16:20Pressemelding

Madison Realty Capital (MRC) announced the closing of a $64.0 million construction loan for 200 Kent Avenue, a 117,326 square foot mixed-use development located in Williamsburg, Brooklyn. The retail portion of the property, which will include over 600 feet of frontage, will be anchored by a popular national grocery chain. Overall, the project will offer 50,101 square feet of retail space, 22,055 square feet of office and restaurant space, and 45,170 square feet of parking. “We continue to establish MRC as the one-stop shop for financing transitional real estate, including ground-up development deals like 200 Kent Avenue,” said Josh Zegen, Co-Founder and Managing Principal of MRC. “In this case, we’re working with a repeat MRC borrower who understands our ability to execute efficiently, given our firm’s up-to-the minute knowledge of local market conditions and non-bureaucratic approach to

Ncardia Announces Completion of €10.5M Investment Round17.11.2017 12:31Pressemelding

Ncardia, an emerging drug discovery and development stem cell technology company whose mission is to deliver cardiac and neural solutions based on its best-in-class human induced pluripotent stem cell (iPSC)-derived technology, today announced the completion of a €10.5 million series B financing round. The round was led by Épimède, a Belgium venture capital firm. Ncardia is a privately-held company with operations in Europe and the US, that produces and commercializes high-quality, fully-functional human iPSC-derived cardiovascular and neuronal cell types. Using its cell products, Ncardia develops and commercializes assay services for drug safety and efficacy testing. Additionally the company has built up a strong portfolio of patents covering the use of stem cell models for these applications. Stefan Braam, CEO of Ncardia, commented: “At Ncardia, important progress h

Double Win for Vocalink at the 2017 Payments Awards17.11.2017 09:35Pressemelding

Vocalink, a Mastercard company, was last night awarded with two prestigious awards - The Overall Winner and the Payments Infrastructure of the Year Award, for its leading technology and expertise, and in recognition for a milestone year that saw Vocalink’s solutions transform the way people and businesses move money across the globe. 2017 saw Vocalink deliver ground-breaking immediate payments solutions both in the US and Thailand as well as enabling access to the UK payments industry through their payments gateway solution PayPort. The business also saw the launch of the new image based cheque clearing system, creating a robust and more efficient system in the UK. RTP ® in the US: This week, Vocalink announced the launch of RTP® (Real-Time Payments) in the US on behalf of The Clearing House; one of the most comprehensive real-tim

Envion AG: Start-up from the Heart of Berlin Has Pioneered Decentralized Mobile Mining by Combining Blockchain with Regenerative Energy17.11.2017 09:00Pressemelding

Envion AG has created a technology for the first truly mobile data-center that uses low-priced local energy to mine a broad spectrum of cryptocurrencies (Bitcoin, Ethereum, etc.). By harvesting locally available clean energy right at the source, envion can operate at lower costs than competitors and at the same time reduces the CO2 footprint of the blockchain industry. Envion aims at decentralizing the highly-concentrated mining market (China holds 80% in Bitcoin mining) and at bringing control of the market back to the users. That’s why envion gives 100% of its mining profits back to its community. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20171117005011/en/ Envion - World's Most Profitable Standard of Self-Expanding Crypto Infrastructure (Photo: Business Wire) Current challenges in the energy

Chugai's HEMLIBRA® Receives the World's First Regulatory Approval from FDA for Hemophilia A with Inhibitors16.11.2017 23:30Pressemelding

Chugai Pharmaceutical Co., Ltd. (TOKYO:4519) announced today that the U.S. Food and Drug Administration (FDA) has approved the bispecific antibody emicizumab (US product name: HEMLIBRA®) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A (congenital factor VIII deficiency) with factor VIII inhibitors. This represents the first regulatory approval for HEMLIBRA® around the world. The US Biologics License Application (BLA) was submitted by Genentech, a member of Roche Group. “We are pleased that HEMLIBRA, a biopharmaceutical created by Chugai scientists based on their exceptional idea, has received its first regulatory approval,” said Chugai’s President & COO, Tatsuro Kosaka. “HEMLIBRA is a first in class biopharmaceutical which is an advancement on the current limited treatment options available for pat

Pacific Drilling Receives Approval of First Day Motions16.11.2017 22:08Pressemelding

Pacific Drilling S.A. (OTCPink: PACDQ) (“Pacific Drilling” or the “Company”) today announced that the United States Bankruptcy Court for the Southern District of New York has granted the relief requested by the Company in certain first day motions related to ordinary course business activities, subject to certain modifications at the request of the Court, the United States Trustee and stakeholders. The approved motions give us the authority to, among other things, continue to pay employee wages and benefits without interruption, to utilize our current cash management system, and to pay certain foreign and critical vendors for goods and services provided prior to the petition date. All vendors will be paid in full and in cash on normal payment terms for all goods and services provided on or after the petition date. Paul Reese, Chief Executive Officer of Pacific Drilling, said, “With the

I vårt presserom finner du alle våre siste saker, kontaktpersoner, bilder, dokumenter og annen relevant informasjon om oss.

Besøk vårt presserom