French orphan specialist developed new assay for primary endpoint to show non-inferiority of its room temperature-stable salt of trientine, earning a broader FDA approval than generically available second-line trientine products.
Orphalan’s Cuvrior (trientine tetrahydrochloride) will join the US market five years after European Union approval, but with an indication for an earlier line of therapy for patients with Wilson’s disease, a rare inherited disorder of copper transport.
The FDA approved Cuvrior, a new salt of the copper chelating agent trientine, on 28 April 2022 for treatment of...
Clinical trial sponsors in the rare disease space face “big safety demands” from regulators, which can be challenging for those with limited resources. A CRO founder makes the case for “embedding automation” into the clinical trial process to keep pace with regulatory requirements.
The termination of the HHS Secretary’s Committee on Human Research Protections closed an important forum for discussing pediatrics and pregnancy in clinical trial design, a former SACHRP chair said.
FDA regulation was painted as an obstacle to US dominance in the cell and gene therapy space even as panelists at an agency event praised the Office of Therapeutic Products' track record under Nicole Verdun.
Expanded funding for cost recovery could dovetail with FDA Commissioner Martin Makary’s idea for a “conditional approval” pathway based on a plausible mechanism of action.
Clinical trial sponsors in the rare disease space face “big safety demands” from regulators, which can be challenging for those with limited resources. A CRO founder makes the case for “embedding automation” into the clinical trial process to keep pace with regulatory requirements.
The developers of the ChAdOx1 NipahB vaccine will be able to benefit from the early and enhanced support that the European Medicines Agency offers through its priority medicines scheme.
The termination of the HHS Secretary’s Committee on Human Research Protections closed an important forum for discussing pediatrics and pregnancy in clinical trial design, a former SACHRP chair said.
