User login
The US Food and Drug Administration (FDA) has approved the first recombinant C1-esterase inhibitor product (Ruconest) for the treatment of acute attacks in adults and adolescents with hereditary angioedema (HAE).
HAE, which is caused by insufficient amounts of a plasma protein called C1-esterase inhibitor, affects approximately 6000 to 10,000 people in the US.
People with HAE can develop rapid swelling of the hands, feet, limbs, face, intestinal tract, or airway. These acute attacks can occur spontaneously or may be triggered by stress, surgery, or infection.
“Hereditary angioedema is a rare and potentially life-threatening disease,” said Karen Midthun, MD, director of the FDA’s Center for Biologics Evaluation and Research. “[The approval of Ruconest] provides an important treatment option for these patients.”
Ruconest is a human recombinant C1-esterase inhibitor purified from the milk of genetically modified rabbits. The product is intended to restore the level of functional C1-esterase inhibitor in a patient’s plasma, thereby treating the acute attack of swelling.
Trial results have suggested Ruconest is superior to placebo in treating most HAE attacks. However, due to the limited number of patients with laryngeal attacks, Ruconest has not been established as an effective treatment for these attacks.
The FDA approval of Ruconest to treat HAE is based on results of a phase 3, randomized, controlled trial (RCT), which included an open-label extension (OLE) phase, and is supported by the results of 2 additional RCTs and 2 additional OLE studies.
The pivotal RCT and OLE studies included 44 subjects who experienced 170 HAE attacks. The primary efficacy endpoint was the time to the beginning of symptom relief, assessed using patient-reported responses to 2 questions about the change in overall severity of their HAE attack symptoms after the start of treatment.
The researchers assessed these responses at regular time points for each of the affected anatomical locations for up to 24 hours. To achieve the primary endpoint, a patient had to have a positive response to both questions, along with persistence of improvement at the next assessment time (ie, the same or a better response).
There was a statistically significant difference in the time to the beginning of symptom relief in the intent-to-treat population (n=75) between the Ruconest and placebo arms (P=0.031).
The median time to the beginning of symptom relief was 90 minutes for Ruconest-treated patients (n=44) and 152 minutes for placebo-treated patients (n=31).
The most common adverse events, reported in at least 2% of patients receiving Ruconest, were headache, nausea, and diarrhea.
Serious adverse events associated with the treatment include anaphylaxis and arterial and venous thromboembolic events in patients with risk factors, such as an indwelling venous catheter/access device, a prior history of thrombosis, underlying atherosclerosis, the use of oral contraceptives or certain androgens, morbid obesity, and immobility.
Ruconest is manufactured by Pharming Group NV, located in Leiden, the Netherlands, and will be distributed in the US by Santarus Inc., a wholly owned subsidiary of Salix Pharmaceuticals Inc., which is located in Raleigh, North Carolina.
Salix is planning to make Ruconest available to patients later this year. 
The US Food and Drug Administration (FDA) has approved the first recombinant C1-esterase inhibitor product (Ruconest) for the treatment of acute attacks in adults and adolescents with hereditary angioedema (HAE).
HAE, which is caused by insufficient amounts of a plasma protein called C1-esterase inhibitor, affects approximately 6000 to 10,000 people in the US.
People with HAE can develop rapid swelling of the hands, feet, limbs, face, intestinal tract, or airway. These acute attacks can occur spontaneously or may be triggered by stress, surgery, or infection.
“Hereditary angioedema is a rare and potentially life-threatening disease,” said Karen Midthun, MD, director of the FDA’s Center for Biologics Evaluation and Research. “[The approval of Ruconest] provides an important treatment option for these patients.”
Ruconest is a human recombinant C1-esterase inhibitor purified from the milk of genetically modified rabbits. The product is intended to restore the level of functional C1-esterase inhibitor in a patient’s plasma, thereby treating the acute attack of swelling.
Trial results have suggested Ruconest is superior to placebo in treating most HAE attacks. However, due to the limited number of patients with laryngeal attacks, Ruconest has not been established as an effective treatment for these attacks.
The FDA approval of Ruconest to treat HAE is based on results of a phase 3, randomized, controlled trial (RCT), which included an open-label extension (OLE) phase, and is supported by the results of 2 additional RCTs and 2 additional OLE studies.
The pivotal RCT and OLE studies included 44 subjects who experienced 170 HAE attacks. The primary efficacy endpoint was the time to the beginning of symptom relief, assessed using patient-reported responses to 2 questions about the change in overall severity of their HAE attack symptoms after the start of treatment.
The researchers assessed these responses at regular time points for each of the affected anatomical locations for up to 24 hours. To achieve the primary endpoint, a patient had to have a positive response to both questions, along with persistence of improvement at the next assessment time (ie, the same or a better response).
There was a statistically significant difference in the time to the beginning of symptom relief in the intent-to-treat population (n=75) between the Ruconest and placebo arms (P=0.031).
The median time to the beginning of symptom relief was 90 minutes for Ruconest-treated patients (n=44) and 152 minutes for placebo-treated patients (n=31).
The most common adverse events, reported in at least 2% of patients receiving Ruconest, were headache, nausea, and diarrhea.
Serious adverse events associated with the treatment include anaphylaxis and arterial and venous thromboembolic events in patients with risk factors, such as an indwelling venous catheter/access device, a prior history of thrombosis, underlying atherosclerosis, the use of oral contraceptives or certain androgens, morbid obesity, and immobility.
Ruconest is manufactured by Pharming Group NV, located in Leiden, the Netherlands, and will be distributed in the US by Santarus Inc., a wholly owned subsidiary of Salix Pharmaceuticals Inc., which is located in Raleigh, North Carolina.
Salix is planning to make Ruconest available to patients later this year.
The US Food and Drug Administration (FDA) has approved the first recombinant C1-esterase inhibitor product (Ruconest) for the treatment of acute attacks in adults and adolescents with hereditary angioedema (HAE).
HAE, which is caused by insufficient amounts of a plasma protein called C1-esterase inhibitor, affects approximately 6000 to 10,000 people in the US.
People with HAE can develop rapid swelling of the hands, feet, limbs, face, intestinal tract, or airway. These acute attacks can occur spontaneously or may be triggered by stress, surgery, or infection.
“Hereditary angioedema is a rare and potentially life-threatening disease,” said Karen Midthun, MD, director of the FDA’s Center for Biologics Evaluation and Research. “[The approval of Ruconest] provides an important treatment option for these patients.”
Ruconest is a human recombinant C1-esterase inhibitor purified from the milk of genetically modified rabbits. The product is intended to restore the level of functional C1-esterase inhibitor in a patient’s plasma, thereby treating the acute attack of swelling.
Trial results have suggested Ruconest is superior to placebo in treating most HAE attacks. However, due to the limited number of patients with laryngeal attacks, Ruconest has not been established as an effective treatment for these attacks.
The FDA approval of Ruconest to treat HAE is based on results of a phase 3, randomized, controlled trial (RCT), which included an open-label extension (OLE) phase, and is supported by the results of 2 additional RCTs and 2 additional OLE studies.
The pivotal RCT and OLE studies included 44 subjects who experienced 170 HAE attacks. The primary efficacy endpoint was the time to the beginning of symptom relief, assessed using patient-reported responses to 2 questions about the change in overall severity of their HAE attack symptoms after the start of treatment.
The researchers assessed these responses at regular time points for each of the affected anatomical locations for up to 24 hours. To achieve the primary endpoint, a patient had to have a positive response to both questions, along with persistence of improvement at the next assessment time (ie, the same or a better response).
There was a statistically significant difference in the time to the beginning of symptom relief in the intent-to-treat population (n=75) between the Ruconest and placebo arms (P=0.031).
The median time to the beginning of symptom relief was 90 minutes for Ruconest-treated patients (n=44) and 152 minutes for placebo-treated patients (n=31).
The most common adverse events, reported in at least 2% of patients receiving Ruconest, were headache, nausea, and diarrhea.
Serious adverse events associated with the treatment include anaphylaxis and arterial and venous thromboembolic events in patients with risk factors, such as an indwelling venous catheter/access device, a prior history of thrombosis, underlying atherosclerosis, the use of oral contraceptives or certain androgens, morbid obesity, and immobility.
Ruconest is manufactured by Pharming Group NV, located in Leiden, the Netherlands, and will be distributed in the US by Santarus Inc., a wholly owned subsidiary of Salix Pharmaceuticals Inc., which is located in Raleigh, North Carolina.
Salix is planning to make Ruconest available to patients later this year.