Rare diseases
The gene therapy uses an AAV vector to restore healthy levels of the alpha-galactosidase enzyme, which is rendered dysfunctional in patients with Fabry disease, leading to the toxic build-up of lipids in cells.
The FDA in July last year declined to approve UX111 for Sanfilippo syndrome, a rare neurodegenerative disorder, citing manufacturing issues. Ultragenyx resubmitted its application ten days ago, expecting a six month review time.
After a series of deaths in patients taking Sarepta Therapeutics’ gene therapies, doubt has crept into investor sentiments around the long-time Wall Street darling, and patients may soon begin looking elsewhere.
After review, Amgen is certain that Tavneos is effective and has a favorable benefit-risk profile. The company informed the FDA on January 28 that they would not pull the drug.
Pfizer announces the first data from its Metsera-acquired pipeline just ahead of its earnings call, where analysts pressed execs for more details; Merck and Roche also released Q4 and full year earnings, with Eli Lilly, Novo Nordisk and others reporting Wednesday; REGENXBIO hits a regulatory snag ahead of its upcoming PDUFA; more.
Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like MPS II moving into the spotlight.
U.S. President Donald Trump signed a spending package into law Tuesday that reauthorizes the FDA’s previously stalled rare pediatric disease priority review voucher program, among other initiatives, while ending a three-day partial government shutdown.
While GSK did not provide a specific reason for returning Wave Life Sciences’ WVE-006, the decision comes after the asset in September 2025 came below analyst expectations in a Phase Ib/IIa AATD study.
Sanofi will take venglustat to regulators for Gaucher disease but an application for Fabry disease is less clear after the failure of a Phase III trial.
In what is shaping up to be a back-loaded month, the FDA is set to release a slew of regulatory decisions in February, including two that would expand the labels of blockbuster drugs.
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