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Respiratory Critical Care Spectrum
Acute respiratory failure (ARF) is a condition that occurs along a wide disease spectrum from respiratory compromise through acute respiratory distress to death.
ARF is precipitated either directly by an injury to the lung (e.g. pneumonia), or by an indirect insult elsewhere in the body that affects the lung (e.g. sepsis).
Patients may progress along the disease pathway from less severe to more severe in a short period of time and acute intensive critical care is often required.
Two main stages of the disease have been defined as acute lung injury (ALI) and the more severe acute respiratory distress syndrome (ARDS).
The initial insult to the lung causes fluid and inflammation to accumulate and leads to a functional loss of the natural surfactant pool within the respiratory tract.
Restoring surfactant function in these patients can potentially stabilize the airways and lead to improved gas exchange within the lung.
There are a significant number of patients at risk annually in the United States for ALI, and the unmet needs for this population are quite high, with mortality rates
estimated to be as high as 30% to 40%. Discovery Labs estimates the global market opportunity for ALI prevention is greater
than $1 billion.
There have been several clinical studies evaluating the therapeutic role of surfactants and other pharmaceutical agents to treat this patient population; however,
there are currently no approved drug therapies. We believe many of these attempts may have failed because the patient populations treated were too heterogeneous or too
far along the disease spectrum for the therapies to show a consistent treatment effect.
KL4 surfactant may potentially be effective for treating ALI patients or preventing a further progression along the disease
spectrum. After an initial experience treating severe ARDS patients in a small phase 2 pilot study, we initiated a proof-of-concept phase 2 clinical trial to determine
whether administration of a bolus of intratracheal SURFAXIN® improves lung function and reduces the duration of mechanical
ventilation in children up to two years of age diagnosed with ARF. ARF in young children represents a more homogenous patient population, making them more conducive
for a proof-of-concept study in this disease area. Results of the study suggested that bolus intratracheal SURFAXIN was
generally safe and well tolerated in this patient population and further supported the concept that intervention earlier in the disease spectrum may enhance the treatment
effect.
SURFAXIN (lucinactant) is an investigational drug, and is under review by the U.S. Food & Drug Administration. Several published and presented
pre-clinical and clinical studies are referenced throughout this website; however, approved and final prescribing information for SURFAXIN is not available at this time.
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