FDA approves drug for infantile hemangioma

April 29, 2014

French pharmaceutical company Pierre Fabre has received marketing authorization from the Food and Drug Administration for the oral drug Hemangeol (propranolol hydrochloride) for the treatment of infantile hemangioma.

French pharmaceutical company Pierre Fabre has received marketing authorization from the Food and Drug Administration for the oral drug Hemangeol (propranolol hydrochloride) for the treatment of infantile hemangioma.

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The drug is the first - and, so far, only - approved treatment for proliferating infantile hemangioma requiring systemic therapy, according to a company news release. The oral solution will be available in the United States in June.

Pierre Fabre applied for authorization to both the FDA and the Committee for Medicinal Products for Human Use of the European Medicines Agency in May 2013. It is reported that European marketing authorization for the drug is imminent.

“Dr. Léauté-Labreze’s serendipitous discovery and Pierre Fabre’s support of the development of propranolol in an optimized oral solution vehicle with a specific indication for infantile hemangioma is the most significant therapeutic innovation of my almost 30-year career,” pediatric dermatologist Elaine Siegfried, M.D., tells Dermatology Times. “For neonates with hemangiomas at risk for ulceration, disfigurement or functional sequelae, this drug has revolutionized potential for successful treatment. We no longer struggle with the need for complex woundcare or narcotic analgesia, and can confidently reassure parents that their child’s hemangioma will one day be gone, without surgery or scarring.”

Dr. Siegfried, who is a professor in the division of dermatology at Saint Louis University School of Medicine, St. Louis, says the drug’s development has also “provided insight into the pathophysiology of hemangiomas and allowed for development of topical treatment with related compounds.”

Propranolol was first used as an off-label treatment for infantile hemangiomas by dermatologists at the Bordeaux University hospital in 2007. It had been used in cardiology, but not properly studied for use among infants suffering from hemangiomas. Testing began in 2009 to demonstrate the drug’s safety and efficacy as a prime treatment for the condition.

The drug was studied among infants ranging from ages 5 weeks to 5 months and compared four different treatments against placebo. Researchers noted a 60 percent success rate in resolving target hemangioma. Placebo success rate was less than 4 percent.