The biotech plans a pivotal trial that would support accelerated approval of the Takeda-partnered antisense oligonucleotide, WVE-003, and will discuss it with regulators before the end of the year.

The biotech said it plans to engage with regulators before the end of the year for the Takeda-partnered antisense oligonucleotide, WVE-003, to discuss a pivotal trial that would support accelerated approval.

With no approved therapies globally for Stargardt's, competition in the indication is increasing in Japan, where Kubota Vision is seeking for conditional approval for emixustat, while Belite Bio’s contender tinlarebant has received Sakigake designation as a local Phase III program continues.

The notice of opportunity for hearing on the US FDA’s proposal not to approve an insomnia sNDA for Vanda’s Hetlioz opens another front in the company’s aggressive campaign against complete response letters and generic competition to the sleep disorder drug.