Pierre Fabre Dermatologie has obtained marketing authorization from the FDA for the pediatric drug Hemangeol(propranolol hydrochloride), which is the first and only approved treatment for “proliferating infantile hemangioma requiring systemic therapy”. Hemangeolis an oral solution specially developed for safe and effective use in children. Hemangeol will be available June 2014.

This marketing authorization comes after the new drug application for Hemangeol was submitted to the FDA in May 2013. The application was also submitted to the European Medicines Agency, receiving positive opinion on February 21st, 2014 from the CHMP, with marketing authorization expected for April 28th, 2014.

The efficacy of propranolol in the treatment of infantile hemangioma (IH) was first discovered in 2007 by Dr. Christine Léauté-Labreze, a dermatologist at the Bordeaux University Hospital. Subsequently, the off-label use of this molecule became the first-line treatment for IH. While propranolol has long been known and used in cardiology, its use in infants with IH had never been properly studied and there was no pharmaceutical form approved for pediatric use. In 2009, Pierre Fabre Dermatologie undertook the pharmaceutical and clinical development required to make the Bordeaux University Hospital discovery accessible to infants with IH, with proven clinical safety and efficacy.

Hemangeol (propranolol hydrochloride) formulation was specifically developed for the use in pediatric population following the guidelines of health regulatory agencies. Hemangeol was studied in infants five weeks to five months old (at therapy initiation) with a proliferative infantile hemangioma requiring systemic treatment in a randomized, double blind placebo controlled, multi-dose and multi-center adaptive Phase II/III trial, which compared four propranolol treatment protocols (1 or 3 mg/kg/day for three or six months) versus placebo. The treatment protocol of 3 mg/kg/day dose for the duration of six months had a 60.4 percent success rate versus 3.6 percent in the placebo group (p< 0.0001) reaching the primary endpoint: complete or nearly-complete resolution of the target hemangioma. In the study, 11.4 percent of patients needed to be retreated after stopping the treatment.

“This collaboration has endowed pediatric dermatology with a new therapy that fulfills an unmet medical need and thousands of American children may now benefit from this new therapy each year,”declared Dr. Jean-Jacques Voisard, Dermatologist, General Manager of Pierre Fabre Dermatologie.

“As Pierre Fabre US representatives we are proud to be part of a Group able to develop children dedicated medicines and to be the first Pierre Fabre subsidiary to obtain marketing approval for Hemangeol,”added Laurent-Emmanuel Saffré, General Manager of Pierre Fabre Pharmaceuticals, Inc. (USA).

“The marketing authorization granted by the FDA rewards a public–private partnership developed over the last six years by Pierre Fabre Laboratories and the Bordeaux University Hospital, with the support from Aquitaine Science Transfert,”stated Eric Ducournau, CEO of Pierre Fabre Dermo-cosmetics SAS, parent company of Pierre Fabre Dermatologie.