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

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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

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