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A phase II/III clinical data published in the NEJM confirms positive safety-efficacy profile for first pediatric treatment for infantile hemangioma.

PARSIPPANY, N.J., Feb. 20, 2015 /PRNewswire/ -- Pierre Fabre announced today that the results from the landmark clinical trial evaluating the first ever pediatric propranolol treatment for use in certain types of infantile hemangiomas resulted in significantly higher complete or nearly complete resolution of the target hemangioma. This data has just been published in the New England Journal of Medicine and specifically shows that the dose regimen of 3mg of propranolol base (3.4mg propranolol hydrochloride) per kilogram per day for 6 months has a significantly higher successful treatment rate than placebo (60.4% vs 3.6%; p<0.0001). The publication of these results follows recent FDA approval and EU market authorizations for Hemangeol (propranolol hydrochloride oral solution), which was developed by the Pierre Fabre Dermatologie and Pierre Fabre Research teams in partnership with the University of Bordeaux for use in infants 1 to 5 months of age with proliferating infantile hemangioma requiring systemic therapy.

"This publication is the result of 5 years' worth of development work on a pediatric formulation of propranolol and represents a wonderful recognition of the efforts of investigators and their teams spread throughout 56 centers and 16 countries. We are particularly proud to be able to offer now an effective solution to children who suffer from this potentially function-threatening, disfiguring, or ulcerated skin condition thanks to the therapeutic advancement that has been confirmed by this study," declared Dr Jean Jacques VOISARD Dermatologist, General Manager of Pierre Fabre Dermatologie.

Infantile hemangioma is the most frequent tumor in newborns. It affects 5 to 10% of newborns, aged less than 1 year, and up to 30% of premature babies. Of these tumors 12% require systemic therapy[sup.1,2,3,4]. In past decades, this therapy was based on systemic use of glucocorticoid therapy with no evolution in sight. Thus, it was Dr. Christine Leaute Labreze, a Dermatologist from Bordeaux University Hospital, France, who shared her first observations around the efficacy of propranolol in certain types of infantile hemangiomas, in a 2008 letter featured in the New England Journal of Medicine (NEJM)[sup.5].

Thus, as of 2008 the University of Bordeaux and Pierre Fabre Dermatologie engaged in a partnership whose primary objective was to develop a specific formulation of propranolol that would be safe and effective for use in infants with proliferating infantile.

The study that was published in the NEJM[sup.6] is a multicenter phase II/III randomized double blind adaptive trial in 460 babies aged 5 weeks to 5 months presenting a type of hemangioma that requires systemic treatment. This placebo controlled trial has been designed to determine the dose, the duration of treatment and the efficacy of propranolol in infantile hemangiomas. The primary outcome was defined as success or failure of trial treatment at week 24 vs baseline. A successful treatment outcome consisted of complete resolution of the target hemangioma or a nearly complete resolution, meaning minimal degree of telangiectasis, erythema, skin thickening, softtissue swelling, and distortion of anatomical landmarks as well as hemangioma evolution including changes in size and color. The results have shown that 3mg/kg/day of propranolol base for 6 months was the recommended therapeutic dose and duration of treatment with a proven efficacy. The safety was assessed for the most frequent adverse effects classified as mild or moderate in severity and included sleep disorders, respiratory disorders such as bronchitis or bronchiolitis, diarrhea, and vomiting.

These study results led to the only approved drug for Infantile Hemangioma, which fulfils an unmet medical need. We appreciate the work all study participants and the medical community, recognizing their efforts in treating Infantile Hemangiomas.

Important Safety Information

Hemangeol (propranolol hydrochloride) Oral Solution is indicated for the treatment of proliferating infantile hemangioma requiring systemic therapy.

Hemangeol is contraindicated in the following conditions: premature infants with corrected age <5 weeks; infants weighing less than 2 kg; known hypersensitivity to propranolol or any of the excipients; asthma or history of bronchospasm; heart rate <80 beats per minute, greater than first degree heart block, or decompensated heart failure; blood pressure < 50/30 mmHg; and pheochromocytoma.

Hemangeol can cause hypoglycemia in children, especially when they are not feeding regularly or are vomiting. Hypoglycemia may present in the form of seizures, lethargy, or coma. Hemangeol may cause or worsen bradycardia or hypotension and can cause bronchospasm.

Hemangeol may worsen circulatory function in patients with congestive heart failure or increase the risk of stroke in PHACE syndrome patients with severe cerebrovascular anomalies. Investigate infants with large facial infantile hemangioma for potential arteriopathy associated with PHACE syndrome prior to HEMANGEOL therapy.

Hemangeol will interfere with epinephrine used to treat serious anaphylaxis. It can also prevent the response of endogenous catecholamines to correct hypoglycemia and masks the adrenergic warning signs of hypoglycemia, particularly tachycardia, palpitations and sweating. Concomitant treatment with corticosteroids may increase the risks of hypoglycemia.

The most frequently reported adverse reactions to HEMANGEOL were sleep disorders, aggravated respiratory tract infections, diarrhea, and vomiting.

Please see Full Prescribing Information at www.hemangeol.com

About Pierre Fabre Laboratories

Pierre Fabre Laboratories is the second largest independent pharmaceutical group in France with revenue of over 2 billion Euros in 2014, 56% of which comes from international business. Pierre Fabre is present in 42 countries and its products are distributed in more than 130 countries. Through its two branches, Pierre Fabre Pharmaceuticals and Pierre Fabre Dermo-Cosmetique, Pierre Fabre's activities cover all areas of healthcare from prescription and OTC drugs to dermocosmetic treatments and natural health products. Pierre Fabre has 10,000 employees around the world, including 1400 in R&D, and devotes almost 20% of its pharmaceuticals turnover to research and development.

With its well-known brands such as Avene, A-Derma, Ducray, Glytone, Klorane, Naturactive, Rene Furterer, and Pierre Fabre Oral Care, Pierre Fabre Laboratories is the market's leading producer of dermo-cosmetics, hair care and oral care products sold in French pharmacies and drugstores. Avene is the world's best-selling dermo-cosmetics brand. For pharmaceuticals, Pierre Fabre focuses on four key treatment franchises including dermatology, oncology, neuropsychiatry, and women's health.

Created in 1983, present in 84 countries, the dermatology franchise (Pierre Fabre Dermatologie) has become a major player in dermatology over the last 30 years. The company's product portfolio covers the management of major dermatological disorders including acne, psoriasis, inflammatory dermatitis, fungal infections, and alopecia. Thanks to the Group's pharmaceutical expertise, our dermatology franchise is fully committed to the absolute requirement of quality, efficacy and safety of its drugs, research into the pharmaceutical forms best suited to dermatology, and to partnering with dermatologists.

To find out more, please visit us at www.pierre-fabre.com

Press contacts: USA: +1 212 614 4170/+ 1 646 209 9913 diana.levine@bm.com France : +33 (0)1 49 10 83 84 valerie.roucoules@pierre-fabre.com

References:

1. Frieden IJ et al. Guidelines of Care for Hemangiomas of Infancy. American Academy of Dermatology Guidelines /Outcomes Commitee. Journal of the American Academy of Dermatology. 1997 oct ; 37 (4) :631-537

2. Kilcline C., Frieden IJ. Infantile hemangiomas: how common are they? A systematic review of the medical literature. PediatrDermatol. 2008;25: 168-73

3. Hoornweg MJ., Smeudlers MJ., Van der Horst CM. Prevalence and characteristics of hemangiomas in young children. Ned TujdschrGeneeskd. 2005;149: 2455-8

4. Hartzell Larry D., Buckmiller Lisa M. Current management of infantile hemangiomas and their common associated conditions. OtolaryngolClin N Am 45. 2012;545--56

5. Leaute-Labreze C et al. Propanolol for severe hemangiomas of infancy. N Engl J Med 2008 ; 358(24) :264

6.

C. Leaute-Labreze et al. A Randomized, Controlled Trial of Oral Propranolol in Infantile Hemangioma, N Engl J Med 2015;372:735-46.

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