Takeda receives fda approval to expand the use of hyqvia® to treat primary immunodeficiency in children
Cambridge, mass.--(business wire)--takeda (tse:4502/nyse:tak) today announced that the u.s. food and drug administration (fda) has approved a supplemental biologics license application (sbla) to expand the use of hyqvia to treat primary immunodeficiency (pi) in children 2-16 years old. hyqvia is the only subcutaneous immune globulin (scig) infusion that can be administered once a month – every three or four weeks – and was first approved in the u.s. in 2014 for the treatment of pi in adults. pi is an umbrella term to describe a group of more than 400 disorders that affect the body's immune system, increasing susceptibility to infection.1 children living with pi and their families face distinct challenges as they are more likely to get frequent and serious infections that can impact their school attendance and ability to participate in social activities.2 while immune globulin (ig) infusions can be effective at protecting against infections associated with a pi diagnosis, some therapy options may not be optimal for all children and their families. for example, intravenous route of administration may be difficult for some children and the frequency of some subcutaneous therapies may be challenging for some families’ schedules.2 “families of children living with primary immunodeficiency may feel overwhelmed by their child’s chronic medical needs. when it comes to treatment, having choices can mean a great deal to families,” said jorey berry, president and chief executive officer of the immune deficiency foundation. “the approval of this new pi treatment for children 2 to 16 years old offers an alternative for health care providers and families who might prefer a less frequent treatment option that can be administered subcutaneously at home, after appropriate training, or in an infusion center.” the fda approval of hyqvia for the treatment of pi in pediatric patients was based on evidence from a pivotal, prospective, open-label, non-controlled phase 3 clinical trial that included 44 pi patients between the ages of 2 and 16. data were analyzed when all subjects completed 12 months of participation (one year of observation) in the trial. the data showed no clinically meaningful differences in trough immunoglobulin g (igg) levels across age groups. during the 12-month trial period, hyqvia was shown to be efficacious with respect to the occurrence of acute serious bacterial infections (asbis), a primary endpoint. the mean asbi rate per year was 0.04 and was statistically significantly lower (with an upper 1-sided 99% confidence interval of 0.21, p5% of patients in the clinical trials were: local adverse reactions including pain, erythema, edema, and pruritus, and systemic adverse reactions including, headache, antibody formation against recombinant human hyaluronidase (rhuph20), fatigue, nausea, pyrexia, and vomiting. drug interactions passive transfer of antibodies may transiently interfere with the immune responses to live attenuated virus vaccines (e.g., measles, mumps, rubella, and varicella). use in specific populations pregnancy: limited human data are available on the use of hyqvia during pregnancy. the effects of antibodies to the recombinant human hyaluronidase on the human embryo or fetal development are unknown. it is not known whether hyqvia can cause fetal harm when administered to a pregnant woman or if it can affect reproductive capacity. hyqvia should be given to a pregnant woman only if clearly needed. for full u.s. prescribing information, please visit: https://www.shirecontent.com/pi/pdfs/hyqvia_usa_eng.pdf about primary immunodeficiency primary immunodeficiency is not a single condition; it’s a group of more than 400 rare, chronic disorders that disrupt the body’s immune system from functioning properly.1 these conditions are often inherited; however, some of the disorders are also caused by genetic and environmental factors.1 in the united states, pi affects about 1 in 1,200 people.1 people with pi often face increased susceptibility to infections, repeated infections, and infections that are difficult to treat.1 about takeda takeda is a global, values-based, r&d-driven biopharmaceutical leader headquartered in japan, committed to discover and deliver life-transforming treatments, guided by our commitment to patients, our people and the planet. takeda focuses its r&d efforts on four therapeutic areas: oncology, rare genetics and hematology, neuroscience, and gastroenterology (gi), with expertise in immune and inflammatory diseases. we also make targeted r&d investments in plasma-derived therapies and vaccines. we are focusing on developing highly innovative medicines that contribute to making a difference in people’s lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative r&d engine and capabilities to create a robust, modality-diverse pipeline. our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries and regions. for more information, visit https://www.takeda.com. important notice for the purposes of this notice, “press release” means this document, any oral presentation, any question and answer session and any written or oral material discussed or distributed by takeda pharmaceutical company limited (“takeda”) regarding this release. this press release (including any oral briefing and any question-and-answer in connection with it) is not intended to, and does not constitute, represent or form part of any offer, invitation or solicitation of any offer to purchase, otherwise acquire, subscribe for, exchange, sell or otherwise dispose of, any securities or the solicitation of any vote or approval in any jurisdiction. no shares or other securities are being offered to the public by means of this press release. no offering of securities shall be made in the united states except pursuant to registration under the u.s. securities act of 1933, as amended, or an exemption therefrom. this press release is being given (together with any further information which may be provided to the recipient) on the condition that it is for use by the recipient for information purposes only (and not for the evaluation of any investment, acquisition, disposal or any other transaction). any failure to comply with these restrictions may constitute a violation of applicable securities laws. the companies in which takeda directly and indirectly owns investments are separate entities. in this press release, “takeda” is sometimes used for convenience where references are made to takeda and its subsidiaries in general. likewise, the words “we”, “us” and “our” are also used to refer to subsidiaries in general or to those who work for them. these expressions are also used where no useful purpose is served by identifying the particular company or companies. forward-looking statements this press release and any materials distributed in connection with this press release may contain forward-looking statements, beliefs or opinions regarding takeda’s future business, future position and results of operations, including estimates, forecasts, targets and plans for takeda. without limitation, forward-looking statements often include words such as “targets”, “plans”, “believes”, “hopes”, “continues”, “expects”, “aims”, “intends”, “ensures”, “will”, “may”, “should”, “would”, “could” “anticipates”, “estimates”, “projects” or similar expressions or the negative thereof. these forward-looking statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those expressed or implied by the forward-looking statements: the economic circumstances surrounding takeda’s global business, including general economic conditions in japan and the united states; competitive pressures and developments; changes to applicable laws and regulations, including global health care reforms; challenges inherent in new product development, including uncertainty of clinical success and decisions of regulatory authorities and the timing thereof; uncertainty of commercial success for new and existing products; manufacturing difficulties or delays; fluctuations in interest and currency exchange rates; claims or concerns regarding the safety or efficacy of marketed products or product candidates; the impact of health crises, like the novel coronavirus pandemic, on takeda and its customers and suppliers, including foreign governments in countries in which takeda operates, or on other facets of its business; the timing and impact of post-merger integration efforts with acquired companies; the ability to divest assets that are not core to takeda’s operations and the timing of any such divestment(s); and other factors identified in takeda’s most recent annual report on form 20-f and takeda’s other reports filed with the u.s. securities and exchange commission, available on takeda’s website at: https://www.takeda.com/investors/sec-filings/ or at www.sec.gov. takeda does not undertake to update any of the forward-looking statements contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule. past performance is not an indicator of future results and the results or statements of takeda in this press release may not be indicative of, and are not an estimate, forecast, guarantee or projection of takeda’s future results. medical information this press release contains information about products that may not be available in all countries, or may be available under different trademarks, for different indications, in different dosages, or in different strengths. nothing contained herein should be considered a solicitation, promotion or advertisement for any prescription drugs including the ones under development. references __________________________ 1 immune deficiency foundation. about primary immunodeficiencies. accessed may 11, 2021. www.primaryimmune.org/about-primary-immunodeficiencies. 2 ballow, m., heimall, j., epland, k., leiding, j., perez, e., riedl, m., & younger, e. m. patient & family handbook for primary immunodeficiency diseases. immune deficiency foundation;2019. p.199-204. 3 hyqvia [immune globulin infusion 10% (human) with recombinant human hyaluronidase] u.s. prescribing information. us-hyq-0557v1.0 04/23
