Friday 28 August 2015, Amsterdam
The report recognizes the following companies as the key players in the Global Duchenne Muscular Dystrophy Drugs Market: BioMarin Pharmaceutical, Eli Lilly, PTC Therapeutics, Santhera Pharmaceuticals and Sarepta Therapeutics
Other Prominent Vendors in the market are: Acceleron Pharma, Akashi Therapeutics, Asklepios BioPharmaceuticals, Bristol-Myers Squibb, Capricor Therapeutics, Catabasis Pharmaceuticals, Janssen Pharmaceuticals, Lexicon Pharmaceuticals, Marathon Pharmaceuticals, Nippon Shinyaku, Nobelpharma, Pfizer, Summit Therapeutics and Taiho Pharmaceutical
Commenting on the report, an analyst said: “Vendors are focusing on developing drugs that work on novel molecular targets to treat Duchenne muscular dystrophy. For instance, vendors are developing drug candidates based on premature stop codon read-through mechanism such as exon skipping products that target exons such as exon 51, exon 53, exon 45, exon 50, and exon 44, and also PDE5 inhibitors. Gene therapy is also being tested to treat this condition. For instance, Asklepios BioPharmaceutical’s pipeline candidate Biostrophin is a gene therapy in the Phase I trials; drisapersen by BioMarin Pharmaceutical and eteplirsen by Sarepta Therapeutics are exon-skipping products; and NPC-14 by Nobelpharma and PTC Therapeutics’ ataluren (Translarna) are premature stop codon read-through products. These drugs are expected to have a better safety and efficacy parameter over corticosteroids, which are used as off-label drugs, because they target the underlying cause of the disease and are expected to produce better outcomes in terms of improving the patient’s condition.”
According to the report, duchenne muscular dystrophy has no known cure. There is only one approved pharmacotherapy to treat this condition. However, many off-label drugs are available in the market such as corticosteroids but these have safety and efficacy issues. There is only one marketed product in the EU, ataluren, which can be used for the treatment of the disorder. However, this drug has not received an approval in the US or rest of the world. In addition, the disease is associated with high treatment and management costs. This provides an opportunity for vendors to invest in R&D to come up with an affordable cure.
Further, the report states that the cost of the treatment of a patient with Duchenne muscular dystrophy is high, making it difficult for many patients to afford the treatment.
The study was conducted using an objective combination of primary and secondary information including inputs from key participants in the industry. The report contains a comprehensive market and vendor landscape in addition to a SWOT analysis of the key vendors.
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