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Infigratinib topped “even the most optimistic expectations” for efficacy and safety in the late-stage PROPEL 3 study in achondroplasia, Truist Securities analysts said Thursday.
FEATURED STORIES
The rare disease drugmaker is facing potential competitors for achondroplasia drug Voxzogo. Is a big M&A deal with two approved assets enough to maintain investor interest?
The FDA issued a rare Refusal-to-File letter to Moderna over its mRNA-based influenza vaccine application, in an unusual move that sent the biotech’s shares tumbling.
A rapturous response to data published last year for Pelage’s hair loss candidate overwhelmed the biotech. Now, the company is ready to show the world the science behind the breakthrough.
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Read our takes on the biggest stories happening in the industry.
Novo Nordisk and Eli Lilly have been battling head-to-head in an exploding obesity market. They should never have been compared apples to apples.
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Makary is a pancreatic surgeon at Johns Hopkins who became known for battling medical mistakes and in recent years has been an outspoken critic of COVID-19 policies.
Chris Boshoff will take over as Pfizer’s next chief scientific officer as of January 1, replacing Mikael Dolsten who led the pharma through the COVID-19 pandemic. The move comes weeks after Pfizer faced pressure from activist investor Starboard Value.
After previously failing studies in Parkinson’s and Alzheimer’s, dalzanemdor’s latest stumble in Huntington’s disease has pushed Sage Therapeutics to pull the plug on the NMDA receptor modulator.
Readouts from Novo Nordisk and Viking Therapeutics at AASLD 2024 strengthen the argument for GLP-1 therapies as an emerging backbone of MASH treatment, with the potential to combine it with other drug classes to achieve deeper responses, according to BMO Capital Markets analyst Evan Seigerman.
The selection of controversial TV personality Mehmet Oz to run the agency overseeing Medicare and Medicaid follows Trump’s pick of RFK Jr. to run its parent department, HHS.
Trump fingers Robert F. Kennedy Jr. to lead the HHS, lupus and ATTR-CM dominate headlines this week, bluebird bio has a cash gap to leap and RegenxBio eyes Sarepta in Duchenne muscular dystrophy.
A judge in the U.K. last month sided with Pfizer over GSK in a respiratory syncytial virus vaccine patent lawsuit, positioning both companies to compete for that market and laying down a marker for ongoing legal clashes in other parts of the world.
While the full impact of the Supreme Court decision remains unknown, the new regulatory landscape could be a net positive for drug developers.
A handful of billion-dollar deals in the rare disease space highlights the uptick in Big Pharma’s investment, but it’s still extremely low compared to the money flowing to more common indications.
After the unexpected success of their PHOENYCS GO study for dapirolizumab pegol in lupus earlier this fall, Biogen and UCB are planning a second late-stage trial by the end of the year to support a drug application.