News
BioMarin’s investigational therapy failed to elicit clinical improvements in patients with ENPP1 deficiency, while also missing key secondary endpoints of rickets severity and growth.
FEATURED STORIES
Nusano will bring a massive new radioisotope facility in Salt Lake City online by the end of the year, establishing a supply of starting materials for the next generation of radiopharmaceuticals.
Last month, Revolution Medicines’ RAS inhibitor doubled survival in a Phase 3 pancreatic cancer trial. On the biotech’s heels are Immuneering, Actuate Therapeutics, Erasca and more, looking to improve on that result with increased tolerability—and more time for patients.
The recent approval of Regeneron’s Otarmeni underscores the maturation of gene therapies across a range of diseases. Here, BioSpace reviews genetic medicines in development for the central nervous system, retinal, cardiac and neuromuscular diseases.
FROM OUR EDITORS
Read our takes on the biggest stories happening in the industry.
The Department of Health and Human Services is spinning its wheels, unable to establish steady leadership at three major divisions—the CDC and the FDA’s two primary review units.
THE LATEST
FDA decisions lack majority consensus, experts agree, possibly leading to less nuanced verdicts on new drug applications. This type of “fiat” decision-making, as multiple regulatory experts have called it, is also bleeding into the agency’s policymaking.
Less than a year after cutting roughly 30% of its employees, BioAtla is letting go of an even larger chunk of its workforce as it considers its future, which could include strategic partnerships and selling off assets.
Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed to the biotech that a sham surgery–controlled study is needed before submitting the gene therapy for approval.
As Novo Nordisk continues to lose ground in the obesity market to rival Eli Lilly, the Danish company has started construction projects to establish the ex-Alkermes plant as a hub for supplying oral GLP-1 products to global markets.
Aardvark Therapeutics is down 54% since Friday after the biotech said it detected “reversible cardiac observations” in a healthy volunteer study of its drug to treat extreme hunger in patients with the rare genetic disease.
Generate:Biomedicines has hit the public markets as the world begins to question the usefulness of AI technology. CEO Mike Nally says biology is the key to unlocking the technology’s full potential.
Yuviwel will compete with BioMarin’s Voxzogo. Meanwhile, BridgeBio is working to bring its own achondroplasia drug, the FGFR3 blocker infigratinib, to the market.
One of the two new members of the CDC’s Advisory Committee on Immunization Practices questioned the safety of COVID-19 vaccines before the Texas Senate in 2021.
A combination of Merck’s Keytruda and Pfizer’s Padcev could offer a chemotherapy-free treatment alternative for patients with muscle-invasive bladder cancer, even those eligible for cisplatin treatment.
On the FDA’s docket this month are two decisions pushed back from 2025, including one for a rare form of obesity and another for dry eye disease.