Alexion Release Confirms FDA Approves Strensiq™ (asfotase alfa) for Treatment of Patients with Perinatal-, Infantile- and Juvenile-Onset Hypophosphatasia (HPP)

Loading...
Loading...
Alexion Pharmaceuticals, Inc.
ALXN
announced today that the U.S. Food and Drug Administration (FDA) has approved Strensiq™ (asfotase alfa) for the treatment of patients with perinatal-, infantile- and juvenile-onset hypophosphatasia (HPP). Strensiq, an innovative enzyme replacement therapy (ERT), is the first therapy approved in the U.S. for the treatment of patients with HPP, a genetic, chronic, and progressive ultra-rare metabolic disease in which patients experience devastating effects on multiple systems of the body, leading to debilitating or life-threatening complications.1 This Smart News Release features an interactive multimedia capsule. View the full release here: http://www.businesswire.com/news/home/20151023005892/en/ "The FDA approval of Strensiq brings a highly innovative treatment to patients who, until now, have had no effective therapy to treat this ultra-rare genetic metabolic disease that causes premature death in infants and devastating consequences in those who survive," said David Hallal, Chief Executive Officer of Alexion. "We are pleased that the label includes a survival benefit in infants, substantial bone healing, and improvements in growth and mobility in patients with HPP who had symptoms prior to the age of 18 and were treated with Strensiq. We look forward to rapidly bringing this life-transforming therapy to patients with HPP and their physicians in the United States." "Asfotase alfa is an important advance for many patients with HPP, their families, and the medical community because it can effectively replace in the skeleton the deficient enzyme called tissue non-specific alkaline phosphatase," said Michael Whyte, M.D., lead clinical trial investigator and Medical-Scientific Director of the Center for Metabolic Bone Disease and Molecular Research at Shriners Hospital for Children in St. Louis. "Without treatment, many newborns and infants with HPP fail to develop a normal rib cage and die from respiratory failure, and young children with HPP can suffer from rickets and muscle weakness. In clinical studies, 97 percent of severely affected newborns or infants were alive at age 1 year with asfotase alfa treatment compared to 42 percent of historical control patients. Treatment with asfotase alfa, now for up to seven years, often markedly improved overall health. In young children with HPP, now treated for five years with asfotase alfa, significant corrections of the skeletal complications were documented, and all had better mobility and function -- most achieving the normal range for healthy peers. I am more than gratified by this progress." HPP is characterized by low alkaline phosphatase (ALP) activity and defective bone mineralization that can lead to deformity of bones and other skeletal abnormalities, as well as systemic complications such as profound muscle weakness, seizures, pain, and respiratory failure leading to premature death in infants.1-5 HPP is an ultra-rare disease, which is defined as a disease that affects fewer than 20 patients per one million in the general population.6 "Today is a defining moment for the HPP community, which now has an approved therapy for the first time. It is my hope that patients and their families will benefit from improved awareness of HPP, faster diagnosis, and better outcomes now that there is an approved and effective treatment," said Deborah Sittig, President and Founder of Soft Bones. Alexion will offer support to patients with HPP through its OneSource™ program. OneSource provides each patient and family with personalized support from a dedicated Alexion nurse case manager, who can help patients understand their insurance benefits, receive reimbursement assistance, and provide education support such as in-home injection training. Through OneSource, patients and families can obtain further information regarding third-party foundations and co-pay assistance programs, which help patients meet out-of-pocket expenses related to the treatment of HPP. For uninsured patients who have no access to insurance, the Alexion Access Foundation, a charitable entity, provides Strensiq free of charge for patients. Patients, caregivers, and healthcare providers in the U.S. can now call 1-888-765-4747 to speak with a OneSource nurse case manager. Alexion will now begin serving patients with HPP in the U.S., with Strensiq becoming available commercially by October 27, 2015. The FDA approved Strensiq under Priority Review, and had granted Breakthrough Therapy designation for Strensiq. With this approval, the FDA also issued a Rare Pediatric Disease Priority Review Voucher, which confers priority review to a subsequent drug application that would not otherwise qualify for priority review. The rare pediatric disease review voucher program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases. Strensiq is also approved in the European Union, Japan, and Canada. Clinical Data7 The approval of Strensiq in the U.S. was based on data from four clinical trials and supporting extension trials comprising patients with perinatal-, infantile- and juvenile-onset HPP who received treatment with Strensiq for up to 6.5 years. In patients (ages 1 day to 6.5 years) with perinatal/infantile-onset HPP, treatment with Strensiq resulted in a significant survival benefit compared to historical control patients with similar clinical characteristics. At week 48, the Kaplan-Meier estimate of overall survival was 97 percent for treated patients (n=68) compared to 42 percent for historical control patients (n=48). In addition, estimated invasive ventilator-free survival was 96 percent for treated patients (n=54) compared to 31 percent for historical control patients (n=48). Study results also demonstrated substantial improvements in the skeletal manifestations of HPP, as assessed by the Radiographic Global Impression of Change (RGI-C) scale, and improvements in height and weight, as measured by z-scores, in patients treated with Strensiq. In patients (ages 6 to 12 years) with juvenile-onset HPP, treatment with Strensiq resulted in significant improvements in the skeletal manifestations of HPP at 24 weeks, as measured by RGI-C, compared to historical controls. Importantly, by month 54, 100 percent of Strensiq-treated juvenile-onset patients were responders to treatment (n=8), as measured by substantial bone healing, compared to 6 percent of patients in the historial control group (n=32) at last assessment. In addition, patients treated with Strensiq had improvements in height and weight, as measured by z-scores, compared with untreated historical controls, as well as improvements in gait and mobility. By 4 years of treatment, 100 percent of patients assessed (n=6) achieved the 6 Minute Walk Test within the normal range for age-, sex- and height-matched peers, whereas no patients were in the normal range at baseline. The most commonly reported adverse events observed in clinical trials were injection site reactions. Other common adverse reactions included lipodystrophy, ectopic calcifications, and hypersensitivity reactions. Conference Call Alexion will host a conference call/webcast on Monday, October 26, 2015, at 8:30 a.m. ET to discuss the FDA approval. To participate in this call, dial (866) 433-3833 (USA) or (704) 908-0448 (international), confirmation code 60248704, shortly before 8:30 a.m. ET. A replay of the call will be available for a limited period following the call, beginning at 7:30 p.m. ET. The replay number is (855) 859-2056 (USA) or (404) 537-3406 (international), confirmation code 60248704. The audio webcast can be found on the Investor page of Alexion's website at: http://ir.alexionpharm.com.
Loading...
Loading...
Posted In: NewsFDAPress Releases
Benzinga simplifies the market for smarter investing

Trade confidently with insights and alerts from analyst ratings, free reports and breaking news that affects the stocks you care about.

Join Now: Free!

Loading...