Rare diseases
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.
AbbVie and Gilead are going back to their roots and leaning on their established areas of expertise to set themselves up for sustainable success in 2025.
Along with its gene editing therapy Casgevy, Vertex is offering fertility preservation support for its patients—a program that the HHS claims violates anti-kickback statutes.
In exchange for its investigational gene therapies, Regenxbio will receive $110 million upfront and up to $700 million in milestones. After hitting an all-time low of $6.95 at close of business yesterday, the stock surged on the news by nearly 20% before markets opened Tuesday.
Sarepta Therapeutics’ Duchenne muscular dystrophy therapy Elevidys handily beat analysts’ expectations in the fourth quarter of 2024, reflecting the biotech’s “world-class” execution, according to BMO Capital Markets analysts.
Benefiting from technological and conceptual groundwork and positive early data, gene therapies are advancing in the clinic for cardiovascular diseases including congestive heart failure, chronic refractory angina and cardiomyopathy.
PRESS RELEASES