Emmaus Life Sciences Sickle Cell Treatment Receives Positive Opinion on Pediatric Investigation Plan from European Medicines Agency
Emmaus Life Sciences announced today that the Paediatric Committee (PDCO) of the European Medicines Agency (EMA) has accepted the company's pediatric investigation plan (PIP) for its treatment of sickle cell disease. The decision allows Emmaus to proceed with the submission of a Marketing Authorization Application (MAA).
“The approval of our PIP represents a significant step forward in our efforts to bring this treatment to market in the European Union (EU),” said Yutaka Niihara, MD, MPH, Chairman and Chief Executive Officer of Emmaus Life Sciences, “We look forward to submitting our MAA in the near term and hope to provide sickle cell patients in the EU with a new treatment option once we complete the regulatory process.”
The EMA requires companies seeking to register new medicines to agree with the PDCO on a PIP that outlines a clinical development program for studying the investigational product in the pediatric population. An accepted PIP is required before a company can file an MAA for a new drug in the EU.
On July 7, 2017, the United States (U.S.) Food and Drug Administration approved Endari™ (L-glutamine oral powder), for its use in the U.S.
Emmaus has obtained both Orphan Drug designation in the U.S. and Orphan Medicinal Product designation in the E.U.
About Sickle Cell Disease
Sickle Cell Disease is an inherited blood disorder characterized by the production of an altered form of hemoglobin which polymerizes and becomes fibrous, causing red blood cells to become rigid and change form so that they appear sickle shaped instead of soft and rounded. Patients with Sickle Cell Disease suffer from debilitating episodes of sickle cell crises, which occur when the rigid, adhesive and inflexible red blood cells occlude blood vessels. Sickle cell crises cause excruciating pain as a result of insufficient oxygen being delivered to tissue, referred to as tissue ischemia, and inflammation. These events may lead to a variety of other adverse outcomes such as acute chest syndrome that requires hospitalization. Sickle Cell Disease is an orphan disease, affecting approximately 100,000 patients in the U.S. and millions worldwide with significant unmet medical needs.
Endari is indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older.
Important Safety Information
The most common adverse reactions (incidence >10%) in clinical studies were constipation, nausea, headache, abdominal pain, cough, pain in extremity, back pain, and chest pain.
Adverse reactions leading to treatment discontinuation included 1 case each of hypersplenism, abdominal pain, dyspepsia, burning sensation, and hot flash.
The safety and efficacy of Endari in pediatric patients with sickle cell disease younger than 5 years of age has not been established.
For more information, please see full Prescribing Information of Endari at www.ENDARIrx.com/Repository/Prescribing%20information.pdf
About Emmaus Life Sciences
Emmaus Life Sciences, Inc. is engaged in the discovery, development and commercialization of innovative treatments and therapies for rare diseases. The company's research on sickle cell disease was initiated by Yutaka Niihara, MD, MPH, Chairman and CEO of Emmaus, at the Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center. For more information, please visit www.emmauslifesciences.com.
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, regarding the research, development and potential commercialization of pharmaceutical products. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. Additional risks and uncertainties are described in reports filed by Emmaus Life Sciences, Inc. with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. Emmaus is providing this information as of the date of this press release and does not undertake any obligation to update any forward-looking statements as a result of new information, future events or otherwise.
for Emmaus Life Sciences
Lori Teranishi, 510-290-6160
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
Corsearch Names Diane Plaut as Global General Counsel and Head of HR23.1.2018 00:00 | Tiedote
Corsearch, a trademark and domain solutions leader, is pleased to announce that Diane Plaut has joined the company as Global General Counsel and Head of HR, reporting to President and CEO Tobias Hartmann. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20180122006527/en/ Diane Plaut, Global General Counsel and Head of HR, Corsearch (Photo: Business Wire) A long-time Corsearch Advisory Board member, client, and collaborator, Ms. Plaut brings to Corsearch over 25 years of experience in global intellectual property law, IP portfolio management, brand protection and enforcement, corporate law, privacy, and compliance. Ms. Plaut’s arrival comes as Corsearch begins a new chapter as an independent company, following its acquisition by Audax Private Equity earlier this month. “Diane is a born leader who is deeply knowledgeable about Corsearch products and services and the industry we serve,” said Mr. Hartmann. “Her passion for our compa
Bacardi to Acquire Patrón Tequila22.1.2018 23:28 | Tiedote
Family-owned Bacardi Limited, the largest privately held spirits company in the world, announced today that it is expanding its portfolio of premium spirits with a definitive agreement to acquire 100% ownership of Patrón Spirits International AG and its PATRÓN® brand, the world’s top-selling ultra-premium tequila. The transaction, which is subject to customary closing conditions, follows the successful relationship the companies have had for nearly a decade since Bacardi’s initial acquisition of a significant minority stake in Patrón in 2008. The transaction reflects an enterprise value for Patrón of US$5.1 billion and is expected to close in the first half of 2018. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20180122006459/en/ Bacardi agrees to acquire 100% ownership of Patrón Spirits International and its PATRÓN® brand, the world’s top-selling ultra-premium tequila. (Photo: Business Wire) According to the most recent IWSR
PMI’s Salary Survey Reveals that Project Management Practitioners with the PMP Certification Earn 23% More22.1.2018 19:58 | Tiedote
Project Management Institute’s (PMI) Earning Power Report reveals a significant advantage in earning potential for PMP® certified practitioners. Survey respondents holding the Project Management Professional (PMP)® certification report higher median salaries than those without a PMP certification―23% higher on average across the 37 countries surveyed. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20180122006262/en/ (Graphic: Business Wire) The Earning Power: Project Management Salary Survey is a comprehensive biennial salary survey that offers insight into the value of project management skills, experience and certifications. It is an industry-leading source of data, giving project management practitioners a greater awareness of their earning potential and helping employers, compensation committees, human resource departments and executive recruiters to better determine salary ranges. The 10th edition of the survey incorporate
ABB and City of Davos Pave the Way to Sustainable Mobility Through E-Vehicle Innovation22.1.2018 18:41 | Tiedote
For this year’s annual World Economic Forum (January 23–26, 2018), the city of Davos and ABB have teamed up in a long-term partnership to provide e-infrastructure for public and private transportation. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20180122006126/en/ ABB flash-charging station in Davos (Photo: Business Wire) Davos residents and WEF guests can experience the pioneering TOSA bus for themselves. The award-winning TOSA bus, developed by ABB in Switzerland, is already in use in Geneva and will soon be implemented in the French city of Nantes. It can recharge its battery in just 20 seconds as passengers board and disembark. In Davos, it will operate on the busy Line 1, running from the tourist center to the hospital. This is the first time TOSA technology is being used in an Alpine region. The idea is to run the system under extreme winter weather conditions; ABB and its project partners expect to gain valuable insig
Ferring enters into worldwide OmniAb® platform license agreement with Ligand22.1.2018 17:00 | Tiedote
Ferring Pharmaceuticals announced today that it has entered into a worldwide license agreement with Ligand Pharmaceuticals to use Ligand’s OmniAb platform® to discover new human monoclonal antibodies for a range of indications. Under the agreement, Ferring will use OmniAb to accelerate development solutions for unmet patient needs in reproductive medicine and women’s health, urology and gastroenterology. “Antibody-based therapies improve the quality of life and care for patients with a wide range of conditions but have not been fully investigated, in particular in the area of reproductive medicine and women’s health,” said Armin Metzger, Senior Vice President, Head of Global Pharmaceutical Research and Development, Ferring Pharmaceuticals. “This agreement expands Ferring’s growing capabilities in biologics and broadens our historical focus on peptides, opening up significant opportunities for discoveries that will help patients to live better lives.” Ligand's OmniAb® platforms produce
Calvin Klein, Inc. Announces the Spring/Summer 2018 Calvin Klein Underwear and Calvin Klein Jeans Global Advertising Campaign22.1.2018 17:00 | Tiedote
Calvin Klein, Inc., a wholly owned subsidiary of PVH Corp. [NYSE:PVH], today announced its new CALVIN KLEIN UNDERWEAR and CALVIN KLEIN JEANS global multi-media advertising campaign. The campaign represents the latest iteration of the evolution in the CALVIN KLEIN brand’s globally recognized #MYCALVINS call to action: Our Family. #MYCALVINS. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20180122005732/en/ CALVIN KLEIN, INC. ANNOUNCES THE SPRING/SUMMER 2018 Campaign Led by Kim Kardashian West, Khloé Kardashian, Kourtney Kardashian, Kendall Jenner and Kylie Jenner Shot by photographer Willy Vanderperre, the latest chapter of this new campaign features sisters Kim Kardashian West, Khloé Kardashian, Kourtney Kardashian, Kendall Jenner and Kylie Jenner. The siblings are featured wearing styles of CALVIN KLEIN UNDERWEAR that are available in stores and online now, including CALVIN KLEIN Modern Cotton and the new CALVIN KLEIN Body ran
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