EMA Highlights: EMA gives green light for infant tumour drug

by Gary Finnegan – The European drug regulator has given a paediatric-use marketing authorisation (PUMA) for Hemangiol to treat proliferation of infantile haemangioma.

This is only the second PUMA the European Medicines Agency (EMA) has issued since their introduction in 2007. The first ever PUMA was granted in September 2011 to Buccolam for the treatment of seizures in children.

As an incentive to stimulate research, medicines granted a PUMA benefit from ten years of market protection. However, the regulator said the industry and academia had yet to fully embrace the facility.

Hemangiol is an oral solution of propranolol, a well-known medicine which has been used since the 1960s in adults for cardiovascular therapies and is commonly prescribed in several clinical situations, including control of hypertension.

Infantile haemangioma are benign tumours involving blood vessels that develop in the skin or internal organs. They typically appear during the first four to six weeks of life, characteristically exhibit early rapid growth and then usually start to shrink spontaneously. Around 3% to 10% of children are affected.

Although usually uncomplicated, haemangioma can produce painful ulceration and scarring, and some are associated with life-threatening complications, such as breathing difficulties and heart problems.

The EMA said treatment with Hemangiol is to be used in patients with serious complications requirement systemic therapy and should be initiated in infants aged five weeks to five months. At present, most EU countries do not have any approved treatment options for proliferating infantile haemangioma.

Meanwhile, EMA’s Committee for Medicinal Products for Human Use (CHMP) has given the green light for 12 medicines for rare diseases over the past 12 months. These include medicines for the treatment of rare cancers (3), multidrug resistant tuberculosis (3) and pulmonary arterial hypertension (2).

There has been a year-on-year increase in the number of marketing-authorisation recommendations for medicines for rare diseases over the last few years: 11 in 2013, eight in 2012 and four in 2011.