The US Food and Drug Administration has given its backing for the first ever therapy to treat the inherited muscle disorder Pompe disease, Genzyme’s Myozyme. The often-fatal disorder affects less than 10,000 people worldwide.
The US Food and Drug Administration has given its backing for the first ever therapy to treat the inherited muscle disorder Pompe disease, Genzyme’s Myozyme (alglucosidase alfa). The often-fatal disorder, caused by a deficiency in the enzyme acid alpha glucosidase, affects less than 10,000 people worldwide.
The approval was based on data from two clinical trials involving 39 infants aged 1 month to 3.5 years with Pompe disease; the condition usually results in death from respiratory failure, but patient survival amongst Myozyme recipients was substantially greater than would have been expected historically, with no need for ventilation. Symptoms may either display during the first few months of life, when it is usually rapidly fatal, or during childhood, adolescence or adulthood, in which case patients may experience progressive debilitation and premature death.
Myozyme, which was cleared under the FDA’s orphan drug programme and via a fast-track review, is now entitled to seven years’ market exclusivity, as well as10 years in Europe where it won a green light for marketing in early April. Despite the small patient numbers, analysts have suggested Myozyme could become a $1 billion dollar drug, following in the footsteps of Genzyme's top-selling offering, Cerezyme (imiglucerase) for Gaucher disease. The illness affects just 4,500 patients, but Genzyme still earned $932 million from the drug in 2005.
Myozyme will be launched in the USA within the next two weeks.
New labels for Nexium and Diovan
Meanwhile, the FDA has given the thumbs up to new labeling for AstraZeneca’s antiulcerant Nexium (esomeprazole) and Novartis’ blood pressure drug Diovan (valsartan). Nexium will now be made available in delayed-release capsules for children aged 2-17 requiring short-term treatment for acid reflux. Believed to affect some 8% - or 3 million - children in the USA, the approval looks set to significantly expand Nexium’s marketing potential. The drug, AZ’s biggest seller, reaped almost $1.2 billion during the first quarter of this year alone – but saw its growth rate drop off to 13%, disappointing investors, so this latest nod should please on all counts.
Finally, Novartis will be cheered after the FDA granted the approval of two higher-dose formulations of its star turn, the hypertension drug Diovan HCT (valsartan/hydrochlorothiazide). The new formulations, which will ensure Diovan has the most flexible range of dosing options in the angiotensin receptor blocker class of drugs, will be made available early June. Novartis’ Diovan/Co-Diovan franchise was up 16% at $939 million during the first quarter of the year.
