Rare diseases
Inclusion of the patient perspective is currently an “optional” part of the rare disease drug development process, CBER Director Karim Mikhail told BioSpace. Mikhail, along with his CDER counterpart Michael Davis and other agency rare disease leaders, is trying to change that.
After the FDA delayed a decision for Agios Pharmaceuticals’ sickle cell disease candidate, the biopharma is letting go of more than 35% of its R&D employees.
AstraZeneca has apparently made a $10 billion bid to acquire Rhythm Pharmaceuticals, an offer William Blair suspects will not be the last for the hot ticket metabolic biotech.
The majority of the large pharmas have now reported third quarter earnings—spoiler alert: Eli Lilly made more money than anyone expected. Plus, the FDA dropped another initiative aimed at accelerating rare disease treatments; HHS greenlights the compounding of six unapproved peptides; and uniQure’s embattled gene therapy for Huntington’s disease has finally been accepted for review.
Neurocrine Biosciences will conduct a new study for the rare disease drug Vykat to explore dosing and safety in patients with Prader-Willi syndrome who have complex health issues.
After a year of controversial regulatory reversals, the FDA has accepted uniQure’s application for ifezuntirgene inilparvovec for priority review, with a May 1 action date. If approved, the therapy—also called AMT-130—would be the first disease-modifying treatment for Huntington’s disease.
Former biologics boss Peter Marks had a vision. With the FDA’s latest draft guidance, it’s coming to fruition. Speaking with BioSpace, current CBER leader Karim Mikhail said the “adapted pathway” is designed to develop personalized gene therapies more quickly.
If approved, BridgeBio Pharma’s BBP-418—which, according to one Jefferies analyst, boasts “pretty darn compelling” data—would be the first therapy for limb-girdle muscular dystrophy. The FDA has an action date of Nov. 27.
Setara Biosciences has formed around a handful of assets from Actio Biosciences, which was acquired by Jazz Pharmaceuticals earlier this year.
After Sanofi paid $600 million in cash to acquire Dren Bio’s bispecific last year, the biotech has secured a hefty financing to fund late-stage development and commercial preparations for its lead asset.
PRESS RELEASES