Rare diseases
The Senate failed to pass a massive spending bill on Thursday—which includes the rare pediatric PRV program but also funding for the Immigration and Customs Enforcement’s large-scale crackdown in Minnesota and other states.
Moderna will continue to lead clinical development and manufacturing of the asset, while Recordati will handle commercialization of mRNA-3927, which is under development for the rare metabolic disorder propionic acidemia.
After years of contraction, investors see biotech reentering a growth cycle driven by scientific progress, asset quality and renewed conviction in oncology, obesity and neuroscience innovation.
The discovery of a tumor in a patient who received REGENXBIO’s gene therapy for Hurler syndrome prompted the FDA to place a hold on that program along with the company’s Hunter syndrome program, which is awaiting an FDA decision on or before Feb. 8.
After a patient safety signal and then death, the FDA in October 2025 placed holds on two of the company’s CRISPR programs for hereditary transthyretin amyloidosis.
Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.
Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging field.
The $1.2 trillion budget package will now move to the Senate, which is expected to hold a vote next week.
The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
After winning a surprise approval for its hereditary angioedema drug Ekterly, KalVista is confident the oral offering will capture the lion’s share of the market for on-demand use.
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