Business Wire

ADDMEDICA Receives FDA-Approval for Orphan Drug Siklos®, First and Sole Hydroxyurea-Based Treatment* for Paediatric Patients with Sickle Cell Anaemia.

Jaa

ADDMEDICA has announced that the US Food and Drug Administration (FDA) has approved Siklos® (hydroxyurea tablets) in paediatric patients, two years of age and older, suffering from sickle cell anaemia.

Approval was obtained under a priority review and orphan drug designation.

Particularly suitable for paediatric populations, Siklos® offers dosing options to facilitate adjustment based on the patient’s weight to obtain the best efficacy/tolerance ratio for each child throughout the growth years.

FDA-approval of Siklos® was granted based on efficacy and safety data collected in a cohort study, Escort-HU, which included several hundred child and adult patients treated with Siklos® in Europe.

According to François Anger, President of ADDMEDICA:

“FDA-approval of Siklos® is a major breakthrough in the treatment of American children with sickle cell anaemia. European patients have been benefitting from Siklos®, the only form of hydroxyurea indicated in paediatric care in the world, since 2007. This successful outcome can also be attributed to the patients who agreed to participate in the Escort-HU study and the physicians who allowed it to happen. With the new FDA-approval, ADDMEDICA consolidates its leadership in the treatment of sickle cell anaemia by continuing to invest in the development and availability of this essential therapeutic product in new countries.”

Siklos® will be marketed in the United States by Medunik USA Inc.

“We’re pleased to partner with ADDMEDICA to distribute this orphan drug which is recognised as the reference drug treatment in sickle cell anaemia. It will improve the health and quality of life of hundreds of children with the disease in the United States,” said Éric Gervais, Executive Vice-President of Medunik. “The launch of Siklos® in the next few months establishes our footprint in the United States in orphan drug therapies and is the latest in a series of significant milestones for the company.”

About sickle cell anaemia

Sickle cell anaemia is a particularly disabling disease, whose most common acute manifestations are painful vaso-occlusive crises in the bones, lungs (acute chest syndrome) and most vascularised organs, and strokes. It may be life-threatening, especially in the case of acute chest syndrome.

The disease is caused by abnormal haemoglobin molecules causing red blood cells to become stiff and sickle-shaped under certain conditions whence the name of sickle-cell anaemia.

These sickled red blood cells tend to pile together, creating clots, and blocking the flow of blood through vessels and thus causing vaso-occlusive crises.

The disease is accompanied by varying degrees of anaemia and chronic complications may appear that affect various organs, e.g. hepatic or renal impairment and retarded growth.

The clinical expression of the disease and its evolution are highly variable among individuals.

Painful vaso-occlusive crises are the most common cause of hospitalisation in sickle cell anaemia patients, and therefore represent a very high cost for society.

Hydroxyurea is currently recognised as the reference drug treatment for this disease.

Approximately 100,000 persons suffer from sickle cell anaemia in the USA compared with approximately 60,000 in Europe. In both cases, it is considered to be a rare disease (less than 5 cases per 10,000 inhabitants)

About ADDMEDICA

Founded in 2005, ADDMEDICA is a pharmaceutical company based in France, focused on developing and marketing medical products for rare diseases and unmet medical needs. ADDMEDICA’s main therapeutic and development fields are

• Genetic diseases and their treatment,

• Organ transplant and tissue replacement,

• Wound healing and tissue regeneration.

ADDMEDICA developed Siklos® specifically for sickle cell anaemia. In 2007, ADDMEDICA obtained a marketing authorisation in Europe for Siklos®, the first and sole medicinal product indicated in the prevention of recurrent painful vaso-occlusive crises in sickle cell anaemia. ADDMEDICA now has FDA-approval for Silos® in the treatment of sickle cell disease in paediatric patients 2 years of age and older. Based on its expertise in the field, ADDMEDICA is pursuing the international development of Siklos®.

For more information: http://www.addmedica.com

About Medunik USA

Based in Pennsylvania, Medunik USA works to improve the health and quality of life of Americans living with rare diseases by making several orphan drug therapies available in the United States. With strategic partnerships worldwide, the company has critical experience in approval and market access processes as well as the marketing of orphan drug therapies. For more information: www.medunikusa.com.

Sources:

Concerning the disease

https://www.nhlbi.nih.gov/health/health-topics/topics/sca

https://www.orpha.net/data/patho/Pub/fr/Drepanocytose-FRfrPub125v01.pdf

Concerning Siklos® in the USA

https://www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm590096.htm

https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/208843s000lbl.pdf

Concerning Siklos® in Europe

http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/000689/human_med_001050.jsp&mid=WC0b01ac058001d125&murl=menus/medicines/medicines.jsp

Concerning Escort-HU

https://clinicaltrials.gov/ct2/show/NCT02516579

Contact information

Yucatan
Caroline Prince
cprince@yucatan.fr

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

The 2018 IDN World Report Findings Show Negative IDN Growth in 201725.9.2018 14:24Tiedote

The 2018 IDN World Report findings have been published to idnworldreport.eu. The major findings from the report are: As at December 2017, 7.5 million IDNs were registered, a drop of 14% since December 2016. A major factor in the reduction in global IDN numbers was a change of policy by the Vietnam ccTLD, which resulted in a loss of nearly 1 million .vn second level IDNs. Excluding the impact of .vn, the underlying trend is negative growth of -5%, distributed mainly across second level ccTLDs. IDNs comprise 2% of the world’s domains, a percentage that has remained fairly static since at least 2012. The data in this report include IDNs across both ccTLDs and gTLDs, both at the first and second level. Distribution patterns of IDN registrations are different from those of traditional, ASCII domain names. IDN registrations are highest in Asia and Pacific region. The script of IDNs registered throughout the world strongly correlate to the languages spoken in different countries and territori

Michael Kors Holdings Limited to Be Renamed Capri Holdings Limited25.9.2018 14:00Tiedote

Michael Kors Holdings Limited (NYSE:KORS), a global fashion luxury group, announced today that a definitive agreement has been signed to acquire all of the outstanding shares of Italian luxury fashion house Gianni Versace S.p.A. for a total enterprise value of Euro 1.83 billion or approximately US$2.12 billion. Versace, long recognized as one of the world’s leading luxury fashion companies, is synonymous with Italian glamour and style. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20180925005557/en/ L-R: Jonathan Akeroyd, Donatella Versace, John D. Idol. (Photo: Rahi Rezvani) John D. Idol, Chairman and Chief Executive Officer of Michael Kors Holdings Limited, said, “The acquisition of Versace is an important milestone for our group. Versace was founded in 1978. For over 40 years, Versace has represented the epitome of Italian fashion luxury, a testament to the brand’s timeless heritage. We are excited to have Versace as part

2018 UL International Crown to Be Staged in Incheon October 4-725.9.2018 13:00Tiedote

2018 UL International Crown will be held October 4-7 at Jack Nicklaus Golf Club Korea in Songdo, Incheon Metropolitan City. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20180925005024/en/ 2018 UL International Crown will be held October 4-7 at Jack Nicklaus Golf Club Korea in Songdo, Incheon Metropolitan City. Incheon Metropolitan City is not sparing any effort to support the event as an Ambassador Partner. The most high-profile biennial golf tournament on the LPGA Tour, the third UL International Crown to take place with the second held in Chicago in 2016. 32 players from the eight countries will compete in the four-day match-play event for the “Crown.” (Graphic: Business Wire) The most high-profile biennial golf tournament on the LPGA Tour, a first-of-its-kind team match-play competition featuring the best women golfers from the top eight countries around the world, is being played outside the United States for the first t

Abu Dhabi's Fund Agreed Friendly TOB with MINDOL25.9.2018 12:59Tiedote

Abu Dhabi Dubai STOB Series 22 Investment Limited Partnership (”AbuDhabi22”as follows) and MINDOL HOLDINGS LIMITED (”MINDOL” as follows) which is listed on the Coinsuper Exchange in Hong Kong (https://www.coinsuper.com) agreed AbuDhabi22 to get 33% of crypto currency MINDOL (MIN)(http://mindol.net) that MINDOL issued by TOB. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20180925005523/en/ The world audition of the Hollywood movie "TO DO & DIE" planned to be released worldwide in 2020! (Graphic: Business Wire) Strengthen strategic partnership with MINDOL by implementing TOB. Both of companies chose TOB as strategic approach to make a purchase at a stable price because acquisition price that AbuDhabi22 planned would be expected to significantly exceed with purchases via regular exchanges. TOB STAGE 1 has already finished and now TOB is being implemented at the purchase price of TOB STAGE 2. As a management vision for 2019, MIND

Brentwood Borough Council Stays Ahead of the Game with UniPrint/Azure Cloud Print Solution25.9.2018 12:18Tiedote

UniPrint.net announced today their successful secure cloud print deployment with Brentwood Borough Council in Essex, UK. In 2016, the Council released a report entitled “Vision for Brentwood, 2016-2019”. In it, they outlined their goals to better the lives of their constituents, and those goals included reducing costs and streamlining services. After examining their existing infrastructure, they made the decision to become the 1st UK council to move 100% of its systems and infrastructure to the cloud. “We wanted to create a very easy delivery of people’s desktops no matter where they were, whether it was a coffee shop, their home, or one of our sites,” said Tim Huggins, ICT Manager at Brentwood Borough Council. This presented a multitude of challenges, but one of the biggest was printing. “We approached our common provider,” said Huggins. “But it became quite evident very quickly that they had no solution, and we were way ahead of the game with our concepts and what we were trying to d

GSMA Reveals First Details for MWC19 Barcelona25.9.2018 10:00Tiedote

The GSMA today announced the first details of MWC19 Barcelona (formerly Mobile World Congress), including keynote speakers, prominent participating companies, programmes and events. MWC19 Barcelona will take place 25-28 February 2019, with events being staged at Fira Gran Via, Fira Montjuïc and La Farga L’Hospitalet. The GSMA expects that more than 107,000 professionals from over 200 countries and territories, representing a wide range of industry sectors, will attend MWC19 Barcelona. Under the event theme of “Intelligent Connectivity”, MWC19 Barcelona will highlight the intersection of “hyper-connectivity”, enabled by 5G and the Internet of Things (IoT), and “intelligence”, delivered by artificial intelligence (AI) and big data. In addition to the core elements of intelligent connectivity, MWC19 will explore the key topics of Content, Digital Trust, Digital Wellness and The Future. “Underpinned by 5G and AI, intelligent connectivity is a major technological force that will shape how t

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