FDA grants priority review for Eagle Pharmaceuticals’ Ryanodex NDA for the treatment of exertional heat stroke.
Eagle Pharmaceuticals announced today that their 505(b)(2) New Drug Application (NDA) for Ryanodex (dantrolene sodium) for the treatment of exertional heat stroke (EHS) has been accepted for filing and granted a priority review designation by the U.S. Food and Drug Administration (FDA).
The FDA grants priority review to medicines that may offer major advances in care or provide a treatment option where no adequate therapy exists. Under the Prescription Drug User Fee Act (PDUFA), the FDA will aim to complete its review within six months of the NDA submission; the PDUFA date for the NDA has been set for July 23, 2017.
“There is currently no approved pharmacological treatment for EHS. If Ryanodex is approved, Eagle will be the first to market with a potentially transformational therapy. EHS can strike anyone, but athletes, our military and outdoor workers are especially vulnerable. We look forward to working with the FDA throughout the review process and to their expedited decision in July 2017,” said Scott Tarriff, Chief Executive Officer of Eagle.
“The number of injuries associated with exertional heat illness in the U.S. increased over 130 percent between 1997 and 2006. And, we believe the incidence of EHS is significantly under-reported. There may be approximately 75,000 cases of EHS annually. Ryanodex will represent Eagle’s most significant self-launched commercial product and we are continuing to build our commercial capabilities to serve the healthcare profession upon approval,” added David Pernock, President and Chief Commercial Officer of Eagle Pharmaceuticals.
The NDA is supported by safety and efficacy data from a controlled clinical trial in EHS patients, and preclinical data from animal studies conducted under the ‘Animal Rule’ to further support the efficacy of Ryanodex.
The clinical study supported the known and well-characterized safety profile of Ryanodex and demonstrated that administration of Ryanodex in addition to body cooling showed substantial evidence of increased clinically meaningful effectiveness in treating patients with EHS, compared to body cooling alone. In addition, animal data provided further evidence supporting the efficacy of Ryanodex in the treatment of this rare and life-threatening condition.
“We evaluated Ryanodex in our clinical study conducted in a real-world acute care setting, and in a well-established animal model. Clinical data showed that Ryanodex offered a clinically meaningful benefit to EHS patients when combined with traditional cooling methods, which is further supported by statistically significant results from our animal work,” said Adrian Hepner, M.D., Ph.D., Chief Medical Officer of Eagle Pharmaceuticals.
Information regarding Eagle’s pivotal animal study can be found in Eagle’s press release dated December 13, 2016. Additional information regarding Eagle’s clinical study can be found in Eagle’s press release dated December 17, 2015.
EHS is a sudden and unpredictable disorder that constitutes a medical emergency, which may result in severe multi-organ dysfunction and death. EHS is more commonly seen in young people undergoing exertional physical activity in a hot weather environment, and is one of the leading causes of death in young athletes and military personnel in non-combat situations. EHS cases are also observed in outdoor workers, firefighters, and people randomly performing intense recreational physical activity.
Currently, there is no approved drug product for the treatment of EHS, one of the most severe forms of heat-related illness, characterized by core body temperature of 104° F (40° C) or greater and significant neurological dysfunction. EHS carries high rates of morbidity and mortality. The central nervous system is particularly sensitive to hyperthermia, which may lead to severe neurologic complications and permanent brain damage.
(Source: Business Wire)
Filed Under: Drug Discovery
