Business Wire

Emmaus Life Sciences Receives Validation From European Medicines Agency on Marketing Authorization Application for Sickle Cell Disease Treatment Xyndari (oral glutamine)

Jaa

Emmaus Life Sciences, Inc. (Emmaus) announced today that the company’s Marketing Authorization Application (MAA) for Xyndari has been fully validated and is now under assessment by the European Medicines Agency (EMA) for the treatment of sickle cell disease.

“Sickle cell disease patients in the European Union have not had access to new therapies for almost 20 years,” said Yutaka Niihara, MD, MPH, CEO and chairman of Emmaus. “The submission of this application reflects our continued commitment to provide treatment options for this painful and life-threatening disease to as many patients as possible.”

The MAA is supported by data from the company's phase 3 randomized, double-blind, placebo-controlled, multi-center clinical trial of 230 patients ages 5 to 58 years old with sickle cell disease who had two or more painful crises within 12 months prior to enrollment. Patients who were treated with Xyndari over a 48-week period experienced fewer crisis episodes compared to patients who received a placebo (median 3 vs. median 4), fewer hospitalizations for sickle cell pain (median 2 vs. median 3), and fewer days in the hospital (median 6.5 days vs. median 11 days). Study patients on Xyndari also had fewer occurrences of acute chest syndrome, a life-threatening complication of sickle cell disease (8.6 percent vs. 23.1 percent). A sickle cell crisis was defined as a visit to an emergency room/medical facility for sickle cell disease-related pain treated with a parenterally administered narcotic or ketorolac; the occurrence of chest syndrome, priapism, and splenic sequestration were also considered sickle cell crises. The most common adverse reactions (incidence >10 percent) in clinical studies were constipation, nausea, headache, abdominal pain, cough, pain in extremity, back pain and chest pain.

Xyndari for the treatment of sickle cell disease will be reviewed by the EMA under the centralized licensing procedure for all 28 member states of the European Union, Norway and Iceland. Xyndari has also received orphan designation and an approved paediatric investigation plan (PIP) from the EMA.

Outside the United States, this treatment is currently available through an early access program based on a named patient use for sickle cell disease patients that have exhausted other treatment options. This program is administered by myTomorrows and provides physicians in the EU, Turkey, Middle East and South America with the possibility to prescribe oral L-glutamine to eligible patients prior to marketing authorization.

The U.S. Food and Drug Administration (FDA) approved Endari™ (L-glutamine oral powder) for the treatment of sickle cell disease, specifically to reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older, on July 7, 2017. Endari is currently available by prescription to patients with sickle cell disease in the U.S.

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.

About Endari™

Indication

Endari is indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients five years of age and older.

Important Safety Information

The most common adverse reactions (incidence >10 percent) in clinical studies were constipation, nausea, headache, abdominal pain, cough, pain in extremities, back pain and chest pain.

Adverse reactions leading to treatment discontinuation included one 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 five 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 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, M.D., 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.

Forward-Looking Statements

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.

Contact information

for Emmaus Life Sciences
Lori Teranishi, 510-290-6160
lteranishi@iq360inc.com

Tietoja julkaisijasta

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 ensimmäisten joukossa? Kun tilaat mediatiedotteemme, saat ne sähköpostiisi välittömästi julkaisuhetkellä. Tilauksen voit halutessasi perua milloin tahansa.

Lue lisää julkaisijalta Business Wire

Incyte Announces Positive Interim Data from Phase 2 Trial of Pemigatinib, Its Selective FGFR Inhibitor, in Patients with Cholangiocarcinoma21.10.2018 13:45Tiedote

Incyte Corporation (Nasdaq:INCY) announces updated data from its ongoing Phase 2 FIGHT-202 trial evaluating pemigatinib (INCB54828), its selective fibroblast growth factor receptor (FGFR) inhibitor, in patients with advanced/metastatic or surgically unresectable cholangiocarcinoma (bile duct cancer) who failed at least one previous treatment. In patients with FGFR2 translocations who were followed for at least eight months, interim study results demonstrated an overall response rate (ORR) of 40 percent, the primary endpoint, and a median progression free survival (PFS) of 9.2 months, a key secondary endpoint. These results are being presented at the European Society for Medical Oncology (ESMO) 2018 Congress in Munich, Germany in a poster presentation on Sunday, October 21 from 12:45 p.m. CEST to 1:45 p.m. CEST (6:45 a.m. ET to 7:45 a.m. ET). (Location: Hall A3 – Poster Area Networking Hub; Abstract #756P) “We are pleased to share updated interim results from our ongoing FIGHT-202 trial

Servier and Taiho Oncology Announce Phase III LONSURF® Study Has Met Primary and Secondary Endpoints Demonstrating Prolonged Overall Survival and Progression-Free Survival in Patients with Refractory Metastatic Gastric Cancer21.10.2018 12:10Tiedote

Servier and Taiho Oncology, Inc. (U.S.), a subsidiary of Taiho Pharmaceutical Co., Ltd. (Japan), jointly announced today clinical data from the pivotal Phase III TAS-102 Gastric Study (TAGS) evaluating LONSURF® (trifluridine/tipiracil, TAS-102) versus placebo and best supportive care in patients with heavily pre-treated metastatic gastric cancer who have progressed or are intolerant to previous lines of therapy. The study met its primary endpoint of prolonged overall survival (OS) and secondary endpoint measures of progression-free survival (PFS) consistently supported the OS results, as well as continuing to demonstrate the predictable safety and tolerability profile of trifluridine/tipiracil. Data from TAGS was presented by Dr. Hendrik-Tobias Arkenau, Executive Medical Director of the Sarah Cannon Research Institute UK and an investigator for TAGS, at the ESMO 2018 Congress in Munich, Germany during an oral session (Abstract #LBA25). The study results were simultaneously published in

Phase 3 Data on Filgotinib in Biologic-Experienced Rheumatoid Arthritis to Be Presented at 2018 ACR/ARHP Annual Meeting21.10.2018 01:30Tiedote

Gilead Sciences, Inc. (Nasdaq: GILD) and Galapagos NV (Euronext & NASDAQ: GLPG) today announced detailed results from the Phase 3 FINCH 2 clinical trial of filgotinib, an investigational, selective JAK1 inhibitor, in adults with moderately-to-severely active rheumatoid arthritis and prior inadequate response or intolerance to biologic agents. The data, which are being presented as a late-breaking poster at the 2018 American College of Rheumatology/Association of Rheumatology Health Professionals (ACR/ARHP) Annual Meeting in Chicago, suggest filgotinib has a potential role in addressing important unmet needs in the treatment of rheumatoid arthritis. Positive efficacy data from FINCH 2 were previously announced in September 2018. The data show statistically significant improvements in the proportion of patients achieving a range of clinical efficacy endpoints, including the proportion of patients achieving American College of Rheumatology 20 percent (ACR20, primary endpoint), 50 percent

Manchester United and True Religion Launch Denim Range19.10.2018 20:37Tiedote

Manchester United (NYSE:MANU) and luxury denim brand True Religion have collaborated to launch a range of premium club branded denim wear. A first for the club, the new global partnership with the iconic American denim brand will see a range of men’s & women’s co-branded styles go on sale beginning 26th October in the club’s Megastore as well as online via United Direct and truereligion.com & eu.truereligion.com. The exclusive collection features jeans, shirts and jackets, including a highly desirable limited edition denim jacket embroidered with the club’s crest. Fans will have the opportunity to win a selection of clothing from the new range by visiting www.manutd.com/truereligion. Manchester United’s Group Managing Director, Richard Arnold, comments: “True Religion is a well-known, established name in fashion, creating unique designs without compromising on quality. The range we have collaborated on includes the same attention to detail and craftsmanship that has made True Religion

Arch Insurance Announces Strategic Leadership Changes19.10.2018 16:10Tiedote

Arch Insurance today announced that Matt Shulman will assume the newly created role of CEO, Arch Insurance North America, effective January 1, 2019. In this role, he will lead Arch Insurance’s operations in the United States and Canada. He will report to Nicolas Papadopoulo, Chairman and CEO of Arch Worldwide Insurance Group. Mr. Shulman, who has more than 20 years of experience in the insurance industry, has been with Arch Insurance since 2009 and has served as the President and CEO of Arch Insurance Europe since 2016. “Matt brings significant U.S. and international experience to this role. Under his leadership, together with our senior team, Arch Insurance will continue to enhance our value proposition to our customers through a robust, diversified product portfolio, creative solutions and excellent service,” Mr. Papadopoulo said. Arch Insurance has also created a new organizational structure with three Chief Underwriting Officers (CUO) dedicated to specific lines of business. These

Takeda to Present Results from Phase 3 ALTA-1L Trial Highlighting Intracranial Efficacy of ALUNBRIG® (brigatinib) Versus Crizotinib in First-Line Advanced ALK+ Non-Small Cell Lung Cancer19.10.2018 15:00Tiedote

Takeda Pharmaceutical Company Limited (TSE: 4502) today announced that intracranial efficacy data from the Phase 3 ALTA-1L (ALK in Lung Cancer Trial of BrigAtinib in 1 st Line) trial showed improved intracranial progression-free survival (PFS) and intracranial objective response rate (ORR) with ALUNBRIG (brigatinib) compared to crizotinib among anaplastic lymphoma kinase-positive (ALK+) non-small cell lung cancer (NSCLC) patients. Data for these secondary endpoints will be presented in a poster discussion at the European Society for Medical Oncology (ESMO) 2018 Congress on Friday, October 19 at 2:00 p.m. CET in Munich, Germany. These results further support ALUNBRIG as a potential treatment for adults with ALK+ locally advanced or metastatic NSCLC who had not received a prior ALK inhibitor. ALUNBRIG is currently not approved as first-line therapy for advanced ALK+ NSCLC. “ALK+ NSCLC often spreads to the brain, so having options that can clearly demonstrate efficacy both in the brain an

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