Rare diseases
This is the third indication for Fabhalta after Novartis won FDA approval of the small molecule in paroxysmal nocturnal hemoglobinuria and primary immunoglobulin A nephropathy.
After Sarepta reported the death of a patient who had recently taken the gene therapy Elevidys, patient advocacy group Parent Project Muscular Dystrophy stepped up—as they always do.
The gene therapy world is in turmoil, but Arbor, armed with more than $1 billion in partnerships and raises, is going forward.
Ionis and Ultragenyx are competing to develop oligonucleotide treatments for Angelman syndrome, but will Neuren’s peptide catch up?
Ionis will receive $280 million upfront and could get up to $660 million in future milestone payments. Ono will take charge of late-stage development as well as regulatory and commercialization activities.
BEAM-302 “has set the bar for efficacy in this space,” William Blair analysts wrote in an investor note on Monday.
On the agenda for the FDA this month are two RNA-based treatments for rare diseases.
While Congress is renewing the priority review voucher program for rare pediatric diseases, the FDA should be required to keep public records of the passes changing hands, too.
Analysts at Jefferies see Makary as a positive for the rare disease space, given his support for accelerated approvals and openness to “customizing regulatory pathways for rare diseases.”
A BioSpace analysis of all 80 priority review vouchers that have been handed out across the three FDA programs that offer them found that 2024 was the busiest year yet. Companies have disclosed spending $513 million on vouchers that were earned in 2024 so far.
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