FDA Offers Support for Use of Drisapersen


drisapersenDrisapersen recently received a positive nod from the FDA. After several recent productive meetings with the FDA Prosensa announced that they will submit Drisapersen for an approval in 2014. Already deemed a breakthrough designated drug, Drisapersen has provided a viable option for treating Duchenne muscular dystrophy. While initial Phase III studies showed Drisapersen to be comparable to a placebo for improving walking distance in the boys with Duchenne muscular dystrophy, extension studies have been encouraging. A new profile of study data and analysis has revealed the benefits of Drisapersen over a placebo. Recent information disseminated from Prosensa demonstrates that the use of Drisapersen in male patients with Duchenne muscular dystrophy {DMD} proves to delay the disease progression when administered early and for a longer period of time. The lengthened treatment plan, along with administration of Drisapersen sooner rather than later is exciting news for patients with this debilitating disease.

Prosensa hopes that the latest mid-stage data gleaned will prove to be meaningful and comprehensive enough for an FDA approval. Prosensa has also announced their plan to execute two post approval studies of Drisapersen, to offer even more information to prove the efficacy, safety and tolerability of this formidable drug. The news touting the benefits of Drisapersen for patients with DMD propelled shares of Prosensa in the stock market and offered hope to this patient population in desperate need of treatments to manage their rare and devastating condition. As a neuromuscular disease that affects 1 in every 3,500 male births DMD is caused by a mutation in the dystrophin gene. This mutation causes an absence or deficiency in the dystrophin protein, leading to detrimental losses of muscle strength. This extreme loss of muscle strength unfortunately affects the heart muscle, ultimately leading to cardiac and respiratory failure. However, the use of Drisapersen offers hope to not only extend the lives of patients with Duchenne muscular dystrophy, but also to provide a lead toward a cure.

LGM Pharma is a provider of the API Drisapersen, CAS # 1251830-50-8 for research and development purposes. Clients can be assured of quality API products and continuous support throughout the R&D process.

Products currently covered by valid US Patents are offered for R&D use in accordance with 35 USC 271(e)+A13(1). Any patent infringement and resulting liability is solely at buyer risk.

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