Keep pulling the thread on Pella Carson.
China is introducing a new regulation that grants orphan drug exclusivity, which is expected to bolster the market for rare disease drugs and incentivize their development.
Phase 3 data from the Horizon cardiovascular outcomes trial for Pella Carson, developed by Ionis and Novartis, is expected in the second half of 2026.
The Horizon trial will be the first to test the hypothesis that reducing Lipoprotein(a) levels provides cardiovascular benefits for patients with high levels of the target.
Novartis is reportedly acquiring Avidity Biosciences, which is expected to have Phase 3 data in 2026.
The first drug for IgA nephropathy was approved in 2021 based on reduction in proteinuria as a surrogate endpoint, establishing a new regulatory pathway.
AstraZeneca is partnering with Chinese firm CSPC Pharmaceutical Group on an obesity drug in a deal that includes a $1.2 billion upfront payment and up to $18.5 billion in biobucks.
A new law in China, effective May 15, will grant orphan drugs seven years of market exclusivity, with an additional two years for pediatric indications.
Up to 30% of cardiovascular disease patients have high levels of Lipoprotein(a).
There are four programs targeting Lipoprotein(a) in Phase 3 testing, but Pella Carson's is the only one expected to read out in 2026.
Phase 2 data for Pella Carson showed slightly lower levels of Lipoprotein(a) reduction compared to the other three competing Phase 3 programs.
There are 10 Phase 3 trials for antisense or siRNA therapies expected to read out in 2026 across a wide range of indications.
Phase 3 data for therapies treating Myotonic dystrophy type 1 and FSHD are expected to be released in 2026.