Ultragenyx Pharmaceutical Inc.
NEWS
Speaking at BIO2025, rare disease leaders from Ultragenyx, Amylyx and Yale questioned the need for the new regulatory pathway proposed by FDA Commissioner Marty Makary. They acknowledged, however, that creative thinking is required to enable more treatments for patients with ultrarare diseases.
While sparking excitement among biopharma companies focused on rare and ultrarare indications, experts say FDA Commissioner Marty Makary’s proposal is light on details and raises potential concerns about safety, access and liability.
Ionis and Ultragenyx are competing to develop oligonucleotide treatments for Angelman syndrome, but will Neuren’s peptide catch up?
By speeding lifesaving drugs’ way to market and focusing on the underlying causes of disease, the pathway has helped save many lives.
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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