ClinicaSpace

The Hunter syndrome space suffered a setback in February when the FDA turned down REGENXBIO’s investigational gene therapy, raising urgent questions about whether competitor Denali Therapeutics can clear the agency’s bar next month.

A “significant number of patients” could see a cure for multiple myeloma within the next two decades, one expert told BioSpace. Here are five therapies that could change the treatment paradigm.

Center for Biologics Evaluation and Research Director Vinay Prasad will exit the FDA for the second time after a controversial run that saw the oncologist butt heads with colleagues and ruffle feathers in biopharma after presiding over the rejection of several rare disease therapies.

While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be considered for approval, experts believe there is an alternate path forward for the therapy, perhaps even based on precedent from the recent drama surrounding Moderna’s mRNA flu vaccine.

Last week, the FDA made its one pivotal trial policy official, sparking myriad questions from industry leaders, including around specific evidence required for the single study and why it hasn’t been implemented across all therapeutic areas before now.

Regulators overseeing rare disease treatments need better tools to weigh competing risks in real time. Sarepta Therapeutics’ Elevidys is a prime example of why.

The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.

After last year’s ‘stampede’ for FGF21 assets, the focus for the metabolic dysfunction-associated steatohepatitis space has shifted toward differentiated approaches, such as THR-β agonists and combination treatments, that seek to mirror the commercial success of Madrigal’s Rezdiffra.

Following the FDA’s refusal to review Moderna’s investigational mRNA flu vaccine last week, Commissioner Marty Makary faced questions from the U.S. president about the agency’s handling of vaccines. It’s a clear signal that the tension long brewing at the drug regulator has now gone all the way to the top.

The FDA’s refusal to review Moderna’s mRNA-based flu vaccine is “part of a disturbing pattern” of moving regulatory goalposts, according to Clay Alspach, executive director of the Alliance for mRNA Medicines. Meanwhile, streamlined communications with regulators in other countries pave the way for rapid uptake of novel modalities.