Alexion Reaches Funding Agreement with NICE and NHS England for Strensiq® (asfotase alfa) for Patients with Pediatric-onset Hypophosphatasia (HPP)
5.7.2017 11:00 | Business Wire
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.
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
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.
|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.|
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.
Kim Diamond, +1 475-230-3775
Executive Director, Corporate Communications
Lauren Cettier, +41 (0)44 457 4323
Director, External Communications, EMEA
Elena Ridloff, CFA, +1 475-230-3601
Vice President, Investor Relations
Catherine Hu, +1 475-230-3599
Director, Investor Relations
For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.
Tilaa tiedotteet sähköpostiisi
Haluatko tietää asioista jo ennen kuin ne uutisoidaan? Kun tilaat tiedotteemme tältä julkaisijalta, saat ne sähköpostiisi yhtä aikaa suomalaisen median kanssa. Tilauksen voit halutessasi perua milloin tahansa.
Lue lisää julkaisijalta Business Wire
Boehringer Ingelheim starts clinical study on interchangeability between its adalimumab biosimilar candidate and HUMIRA®27.7.2017 16:00 | Tiedote
Boehringer Ingelheim announced today that the first patient has been enrolled into its VOLTAIRE-X interchangeability study. The goal of the study is to demonstrate that BI 695501 is interchangeable with the U.S.-marketed formulation of Humira®* 40mg/0.8mL. This is the first study in the U.S. to investigate an interchangeability designation for an adalimumab biosimilar candidate. The study will compare the pharmacokinetics and clinical outcomes between patients receiving Humira® continuously, versus those who switch repeatedly between Humira® and BI 695501, Boehringer Ingelheim’s adalimumab biosimilar candidate.1 The study will also assess safety, immunogenicity and efficacy.1 “We are pleased that the first patient has now been enrolled in VOLTAIRE-X, and look forward to continued recruitment and patient follow-up,” said Ivan Blanarik, Senior Vice President and Head of
Acxiom Launches Connected Spaces27.7.2017 15:30 | Tiedote
Today, Acxiom® (Nasdaq: ACXM), in collaboration with Adobe, announced the launch of Connected Spaces, a global solution aimed at delivering more relevant omnichannel experiences to customers. Connected Spaces is designed to revolutionise customer experience and business returns for retail, travel and leisure locations such as airports, malls, sports stadiums, concert arenas and resorts. A pioneering example is Heathrow airport. Heathrow is able to deliver more relevant communications to customers in real-time as part of its overall engagement strategy and is already generating exceptional results including an increase in spend of 20-25 percent for engaged customers. Common to airports, malls and the like is a high footfall of consumers who intend to visit multiple concession, franchise, or subcontracted brands operating in their environments. This can translate to a relative
Watch BizWireTV: Helix Brings DNA Kits to the Public and Michael Kors Gets Ready to Buy Jimmy Choo27.7.2017 15:08 | Tiedote
On the latest BizWireTV, catch the latest Quick Biz Hits. Also see what’s happening in the startup world with the Accelerator Report, featuring the VC Watch and this week’s Startup Standout. This Smart News Release features multimedia. View the full release here: http://www.businesswire.com/news/home/20170727005456/en/ BizWireTV is hosted by Jordyn Rolling (Photo: Business Wire) Now you can watch BizWireTV, and the latest breakthroughs in tech from the biggest brands, on any screen you want by downloading the new app through the Apple TV and iPhone App Store and Google Play for Android devices. Top of the Wire Helix launches first online consumer marketplace for DNA-powered products that offer insights on ancestry, entertainment, family, fitness, health and nutrition. Watch BizWireTV to
European Commission Grants Orphan Drug Designation to Allena Pharmaceuticals’ Investigational Therapy for the Treatment of Primary Hyperoxaluria27.7.2017 14:05 | Tiedote
Allena Pharmaceuticals, Inc., a specialty biopharmaceutical company dedicated to bringing first in class, specific, non-absorbed, oral enzyme therapeutics to patients with serious renal, urologic and orphan diseases, announced today that the European Commission has granted orphan drug designation to Allena’s investigational product ALLN-177, Bacillus subtilis oxalate decarboxylase, for the treatment of primary hyperoxaluria (PH). The orphan designation was granted to Allena Pharmaceuticals Ireland Limited, a subsidiary of Allena Pharmaceuticals, Inc. Allena’s lead compound ALLN-177, is being developed to treat patients with severe hyperoxaluria, a condition characterized by markedly elevated urinary oxalate excretion. PH, a type of severe hyperoxaluria, is a rare genetic disorder caused by endogenous overproduction of oxalate by the liver that can result in kidney stone disease, kidney
Subscription Period for Superior Industries’ Tender of UNIWHEELS AG’s Shares to Close Monday, July 31, 201727.7.2017 09:00 | Tiedote
Superior Industries International, Inc. (NYSE:SUP), one of the world’s largest manufacturers of aluminum wheels, reiterated today that the subscription period for the tender offer to acquire all remaining outstanding shares of UNIWHEELS AG (“UNIWHEELS”) will close Monday, July 31, 2017. On June 30, 2017, Superior announced it had commenced a tender offer to acquire the remaining 954,920 shares held by the public shareholders, which represents approximately 7.7% of the total outstanding shares of UNIWHEELS, for cash consideration of 247.87zl per share. Superior acquired the other approximately 92.3% of the outstanding shares through a tender offer that was settled on May 30, 2017, where the public shareholders also received cash consideration of 247.87zl per share. Following the settlement of the tender offer, Superior will finalize the proceedings to delist UNIWHEELS’ common stock from
Ipsen Delivers Strong Sales Growth of 18.8%1 in the First Half of 2017 and Upgrades Its Guidance for Full Year 201727.7.2017 08:00 | Tiedote
Regulatory News: Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-driven biopharmaceutical group, today announced financial results for the first half of 2017. H1 2017 Highlights Strong operating performance of 18.8%1 Group sales growth and Core Operating Income margin improvement by 1.2 points to 26.2% of sales Specialty Care sales growth of 23.1%1 reflects continued Somatuline® momentum and includes contribution of key new products Cabometyx® and Onivyde® Consumer Healthcare sales growth of 1.3%1 including contribution of newly acquired products Upgraded full year 2017 guidance of Specialty Care sales growth greater than 24.0%1, slight growth1 of Consumer Healthcare sales, and Core Operating Income margin greater than 25.0% o
Uutishuoneessa voit lukea tiedotteitamme ja muuta julkaisemaamme materiaalia. Löydät sieltä niin yhteyshenkilöidemme tiedot kuin vapaasti julkaistavissa olevia kuvia ja videoita. Uutishuoneessa voit nähdä myös sosiaalisen median sisältöjä. Kaikki STT Infossa julkaistu materiaali on vapaasti median käytettävissä.Tutustu uutishuoneeseemme