Cystic Fibrosis (CF) is an autosomal recessive disease characterized by the secretion of thick, sticky mucus which clogs the lungs and leads to life-threatening lung infections; and obstructs pancreatic enzyme secretions that are essential for the body to break down and absorb nutrients. CF patients have a limited number of available treatment options and significant unmet needs still exist. Opportunities are significant for new therapies that will improve symptoms, change the course of this disease, as well as increase therapy options. The introduction of the first disease modifying therapy, Kalydeco (ivacaftor), by Vertex in 2012, paved the way for a new class of therapies known as cystic fibrosis transmembrane conductance regulator (CFTR) modulators. On June 24, 2014, Vertex announced positive Phase III clinical trial data for lumacaftor in combination with Kalydeco in patients with CF who have two copies of the F508del mutation. This novel combination therapy of CFTR modulators has the potential to treat the underlying cause of the disease for approximately 50% of all CF patients and drive rapid growth in the CF market.
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Scope
Overview of CF, including epidemiology, etiology, pathophysiology, symptoms and current treatment optionsAnnualized CF therapeutics market revenue, annual cost of therapies and forecasts for five years to 2018.Key topics covered include strategic product assessment, market characterization, unmet needs, R&D strategies, clinical trial design and implications for the CF therapeutics market.Pipeline analysis: comprehensive data split across different phases, emerging trends and mechanisms of action under development, including inhaled antibiotics, CFTR modulators and pancreatic enzyme products.Analysis of the current and future market competition in the US and five major EU CF therapeutics market. Clinical and commercial benchmarking of promising pipeline products versus standard of care treatments and competitive assessment of all therapies. Insightful review of the key industry drivers, restraints and challenges.
Reasons to buy
Identify the unmet needs and remaining opportunities in the CF therapeutics market.Develop business strategies by understanding the trends shaping and driving the US and five major EU CF therapeutics market.Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.Assess the clinical and commercial viability of promising pipeline products.Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies.Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various emerging therapies.Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.Drive revenues by understanding the key trends, innovative products and technologies, market and segments likely to impact the US and five major EU CF therapeutics market in future.
Highlights
Key Questions Answered
What are the pressing unmet needs and remaining opportunities in the CF market?What R&D strategies are drug developers pursuing in the CF therapeutics space?What is the significance of CFTR modulators in the CF market?What is the clinical and commercial positioning of key pipeline products in the CF market?What are the innovative early-stage treatment approaches in the CF market?
Key Findings
Rapid growth in the CF market is expected from 2012 to 2018.Emerging market players are employing diverse R&D strategies to gain entry in the CF market.A curative therapy is the most pressing unmet need in CF.Significant opportunities exist for novel disease modifying drugs.
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