Ultragenyx Pharmaceutical Inc.
NEWS
A handful of billion-dollar deals in the rare disease space highlights the uptick in Big Pharma’s investment, but it’s still extremely low compared to the money flowing to more common indications.
As therapies for rare and neurological diseases earn accelerated approval, experts laud the program’s intent while remaining concerned about confirmatory trials and clinical efficacy, especially as products greenlit under this pathway are pulled from the market.
In a Phase I/II study, Ultragenyx’s antisense oligonucleotide GTX-102 led to significant cognitive, behavioral, sleep and gross motor improvements in patients with the rare genetic disorder Angelman syndrome.
Last year, the FDA declined to approve a drug that appears to reverse a rare and debilitating enzyme deficiency. Some experts say it’s emblematic of a need for more flexibility around therapeutics targeting rare diseases.
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After reaching a settlement with Thermo Fisher earlier this month, the family of Henrietta Lacks is now suing Ultragenyx for its “unjust enrichment” using their matriarch’s immortal cell line.
The FDA’s Center for Biologics Evaluation and Research is aiming to recreate the success achieved with the rapid development of COVID-19 vaccines under a program of the same name.
BioSpace spoke with several leaders to gather their thoughts and predictions for the new year. Here is the first installment of this two-part series.
The buyout comes on the heels of promising Phase I/II results from GTX-102, an antisense oligonucleotide candidate being developed to treat Angelman syndrome.
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