American scientists are close to solving the problem of the blood-brain barrier (BBB). This barrier, which protects the brain from viruses and bacteria, also repels an estimated 98% of drugs. Even medications approved to treat neurodegenerative diseases have trouble getting through. For instance, some studies suggest that less than 0.1% of a dose of the Alzheimer’s drug Leqembi (lecanemab) makes it into the brain after intravenous administration, writes PharmaVoice.
However, this could soon change. Biotech company Denali Therapeutics has filed an approval application for a drug that uses new technology to transport medicines directly into the brain. The U.S. Food and Drug Administration (FDA) has granted the application for tividenofusp alfa to treat Hunter syndrome a fast track designation. A regulatory decision on the application is expected by April 5.
If approved, tividenofusp alfa would be the first medication to utilize the modular Transferrin Receptor-enabled TransportVehicle platform. This platform is designed to carry enzymes, oligonucleotides, and antibodies across the blood-brain barrier.
Patients in a mid-stage clinical trial showed a 91% reduction in heparan sulfate, a biomarker for Hunter syndrome. They also showed early signs of improvement in hearing and on key measures of adaptive behavior and cognition at 24 weeks.
Denali CEO Ryan Watts emphasized that the technology could become a versatile platform for treating a wide range of central nervous system (CNS) pathologies—from lysosomal storage disorders to common neurodegenerative diseases. “In addition to evaluating our TransportVehicle to enable enzyme replacement across lysosomal storage disorders and neurodegenerative diseases, we are pursuing the potential of the platform to deliver antibodies and oligonucleotides for diseases that impact the brain,” Watts said in a statement.
