The U.S Food and Drug Administration (FDA) advisory panel gave a recommendation for a drug that treats the Morquio A Syndrome. The agency has not made its final decision yet but will most likely approve it for public use.
The Morquio A Syndrome, which is usually chronic and progressive. This uncommon hereditary disease causes malformation of the skeletal system and affects various organs such as the eyes, ears, heart and lungs. Symptoms which may include loss of hearing, vision impairment and heart problems manifest as early, before a child reaches five years old.
The experimental drug elosulfase alfa, named Vimizim, is manufactured by General BioMarin Pharmaceutical Inc. and is seen to reach about $532 million sales by 2018.
If finally approved, Vimizim will be tagged as an"orphan drug" that allows the pharmaceutical company a market exclusivity spanning seven years. Typically, orphan drugs treat those diseases that are contracted by less than 200,000 patients.
The experiment was conducted by allowing young patients to do 6-minute walks and 3-minute stair climb exercises to measure their improvement. In a span of 24 weeks, the scientists noticed that the Vimizim drug had improved the walking distance of the patients by 22.5 meters, compared to those patients who were given a placebo drug. This is a "modest" gain according to the FDA advisory panel.
The Stair Climb exercise, however, did not give any substantial improvement, statistics-wise.
According to family interviewed by the advisory panel, the adolescents who took Vimizim appeared to have increased energy and better stamina that have paved the way for them to socialize better and move by themselves.
The panel that approved of the drug said there are more advantages to gain from Vimizim compared to the accompanying risks, such as allergies and anaphylaxis reactions.
"We are thrilled to have achieved this important milestone in our mission to bring the first approved therapy to treat Morquio A patients," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin in a press release. "I am very pleased with the outcome of today's panel vote and look forward to continuing to work with the FDA to bring this much-needed therapy to these patients."
