The US Food and Drug Administration (FDA) has approved a pediatric formulation of propranolol hydrochloride (Hemangeol, Pierre Fabre Dermatologie) for treatment of proliferating infantile hemangioma requiring systemic therapy, the company said.
Hemangeol "fulfills an unmet medical need and thousands of American children may now benefit from this new therapy each year," Dr. Jean-Jacques Voisard, dermatologist and general manager of Pierre Fabre Dermatologie, said in a statement.
Hemangeol is an oral solution specially developed for safe and effective use in children, the company said.
The drug was studied in infants aged 5 weeks to 5 months (at therapy initiation) in a randomized study comparing 4 propranolol treatment protocols (1 or 3 mg/kg/day for 3 or 6 months) versus placebo.
The 3 mg/kg/day dose for 6 months had a 60.4% success rate in complete or nearly complete resolution of the target hemangioma, compared with 3.6% in the placebo group (P < .0001), the company said. After stopping treatment, 11.4% of patients needed to be retreated.
The most frequently reported adverse reactions (> 10%) in infants treated with Hemangeol were sleep disorders, aggravated respiratory tract infections such as bronchitis and bronchiolitis associated with cough and fever, diarrhea, and vomiting. Adverse reactions led to treatment discontinuation in fewer than 2% of treated patients, according to the company.
The company also notes that Hemangeol can cause serious side effects including hypoglycemia, bradycardia, hypotension, and bronchospasm, worsen congestive heart failure, and may increase the risk for stroke in children with PHACE syndrome (Posterior fossa, Hemangioma, Arterial lesions, Cardiac abnormalities/aortic coarctation, Eye abnormalities).
Hemangeol is contraindicated in premature infants with corrected age < 5 weeks; infants weighing less than 2 kg; infants with known hypersensitivity to propranolol or any of the excipients; infants with 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 mm Hg; or pheochromocytoma.
Hemangeol will be available in the US beginning in June, the company said.
The European Medicines Agency's Committee for Medicinal Products for Human Use gave a positive opinion on the pediatric formulation of propranolol hydrochloride in February, with marketing authorization expected in late April 2014, the company said.
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Cite this: FDA OKs Propranolol Hydrochloride for Infantile Hemangioma - Medscape - Mar 17, 2014.