FDA Grants Orphan Drug Designation to BIOX-101 for Intracerebral Hemorrhagic Stroke
Currently, there is a gap in treatment for this patient population and intracerebral hemorrhagic stroke is considered an orphan disease.
The FDA granted orphan drug designation for BIOX-101, a therapeutic candidate, to treat intracerebral hemorrhagic (ICH) stroke. BIOX-101 inhibits the harmful secondary effects of hemorrhagic stroke, which can include secondary brain ischemia, neuroinflammation, and neuronal damage.1
Hemorrhagic stroke is caused by a ruptured blood vessel in the brain and can be categorized as either ICH or subarachnoid hemorrhage. | Image Credit: peterschreiber.media | stock.adobe.com
“The granting of orphan drug designation in these 2 jurisdictions confirms that we are on a relatively rapid and cost-effective path to market for BIOX-101. We are working hard to offer a disruptive new therapy for patients suffering from intracranial hemorrhagic stroke, an often deadly condition with a poor prognosis for which no therapeutic is currently available,” Marc Dechamps, CEO at Bioxodes, said in a news release.1
Hemorrhagic stroke is caused by a ruptured blood vessel in the brain and can be categorized as either ICH or subarachnoid hemorrhage (SAH). ICH is bleeding into the brain parenchyma, and SAH is bleeding in the subarachnoid space, according to StatPearls. Early diagnosis and treatment are essential as the disease progresses rapidly. Current treatment can include blood pressure management, management of raised intracranial pressure, hemostatic therapy, antiepileptic therapy, and surgery.2
Health care teams can also improve outcomes, which can include physicians, critical care nurses, and pharmacists. When surgery is necessary, it should be done in a standardized manner, and cognitive rehabilitation therapy should be given to survivors. Currently, there is a gap in treatment for this patient population, and ICH is considered an orphan disease.2
The company plans to release interim results from the first 16 patients in the phase 2a BIRCH trial. Investigators conducted the study at 10 stroke units in Belgium, and the trial will enroll a total of 32 patients. Twenty-four patients will receive the study drug, and 8 will receive the standard of care. This will be an open-label study.1,3
“We’ve reached a crucial point in the development of BIOX-101, which we believe can save lives and improve quality of life by preventing the harmful secondary damage in patients hit by intracerebral hemorrhagic stroke, a devastating condition for which there is no currently available treatment," Dechamps said in a news release.3
The company will also file for fast track approval with the FDA and will also plan a registration international phase 2b trial, with plans to start recruitment in the first quarter of 2027.1
BIOX-101 prevents blood clot formation without increasing the risk of further bleeding and can prevent acute neuroinflammatory events associated with ICH. It also inhibits the activation of neutrophils, which are responders of the inflammatory system.3
