Rare diseases
The rare disease space is awaiting two FDA verdicts in February, one for a genetic disease and another for a non-malignant tumor.
Several companies—including JCR Pharmaceuticals, Denali Therapeutics and Regenxbio—have products in the pipeline that could improve treatment options for this rare disease.
Novartis was among the most prolific pharma dealmakers in 2024, a trend that it expects to continue with more bolt-on deals this year to set up for sustainable long-term growth.
Achondroplasia, which affects around one in 20,000 babies, has just one approved treatment: BioMarin’s Voxzogo. However, new investigational treatments are vying to compete in the area.
Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and more aim to deliver the next wave of progress with near-term data and regulatory milestones.
In a good-news-bad-news week for Biogen, the company will cut an undisclosed number of employees, just as a higher dose of its Ionis-partnered therapy Spinraza for spinal muscular atrophy will be considered by the FDA and EMA.
At J.P. Morgan, most biopharma executives expressed a neutral stance on the incoming administration, but just days later, President Trump issued multiple executive orders that concern the industry.
The FDA is putting Atara’s active Investigational New Drug applications on hold due to manufacturing concerns at a third-party provider while releasing Amylyx’s investigational ALS therapy from a previous pause.
While some analysts expect the 2025 IPO market to be relatively cool in the near-term, others anticipate more bids than in 2024.
After a series of strongly worded letters—one of which was addressed directly to Commissioner Robert Califf—the FDA has publicly laid out its reasoning for rejecting Vanda Pharmaceuticals’ gastroparesis drug candidate tradipitant.
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