BioMarin seeks label expansion for achondroplasia drug; FDA lifts Inhibikase full clinical hold
BioMarin is looking to press its advantage in the achondroplasia, or dwarfism, drug space.
The California company said Tuesday afternoon the FDA will take up its application to expand the Voxzogo label into children younger than 5 years of age. Regulators will make their decision by Oct. 21.
BioMarin’s move came a day after competitor BridgeBio touted Phase II data for its own achondroplasia program and subsequently sought a $150 million stock offering. Voxzogo was originally approved in November 2021 for patients 5 years and older.
Both companies have faced questions about developing a drug some patients and advocacy groups say reinforces the stigma around the condition and tries to solve a problem that doesn’t exist.
FDA lifts Inhibikase full clinical hold
The FDA lifted a full clinical hold on Inhibikase Therapeutics’ experimental drug for multiple system atrophy, the company announced Wednesday.
Lifting the hold will allow Inhibikase to move the drug, known as IkT-148009, into a Phase II study, per a company press release. Regulators imposed the hold last November, pausing research in both MSA and Parkinson’s disease. The hold for Parkinson’s was lifted in January.
“We are grateful for the expeditious review by the FDA,” CEO Milton Werner said in a statement.
According to earlier company statements, the FDA paused the program to more closely scrutinize the reasoning for Inhibikase’s selected Phase II dose, how the company would monitor side effects and additional safety data.
It wasn’t immediately clear Wednesday morning when the biotech planned to launch the Phase II MSA trial.