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Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like MPS II moving into the spotlight.
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After advancing in lockstep through the pandemic, the fortunes of the biotechs have diverged as their use of COVID-19 windfalls has taken shape.
After suffering in the wake of expired tax incentives for pharmas, the island is trying to take advantage of geopolitics to grow its drug manufacturing sector.
AstraZeneca’s $15 billion pledge to its China operations highlights the country’s advantages. But other regions are also hoping to host more clinical studies.
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Phacilitate’s annual event dawns as cell and gene therapies reach a new tipping point: the science has hit new heights just as regulatory and government policies spark momentum and frustration.
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Biosimilars are essential healthcare equalizers, but their regulation is overly complicated due to lobbying by makers of branded biologics looking to maintain blockbuster revenue.
Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several companies—including Novartis, Scholar Rock and Biogen—progressing novel candidates through clinical trials.
Johnson & Johnson follows Eli Lilly in spending billions on U.S. manufacturing after President Donald Trump threatened major tariffs on pharmaceutical products. Pfizer has also promised a similar commitment.
This is the third indication for Fabhalta after Novartis won FDA approval of the small molecule in paroxysmal nocturnal hemoglobinuria and primary immunoglobulin A nephropathy.
The label expansion could help J&J establish Tremfya as a successor to Stelara, which is now facing a growing biosimilar challenge.
After withdrawing its nomination for Dave Weldon last week, the Trump administration is now reportedly considering Texas Republican Michael Burgess to head the Centers for Disease Control and Prevention.
Adaptimmune is rolling out its T cell therapy Tecelra for synovial sarcoma, recording $1.2 million in sales since its approval in August 2024. Nevertheless, it is pausing development of two oncology assets to save money.
The FDA approval of Alnylam’s Amvuttra sets up a three-way race with Pfizer and BridgeBio, which both market transthyretin stabilizers for transthyretin amyloid cardiomyopathy.
Paratek Pharmaceuticals is betting that OptiNose’s chronic rhinosinusitis treatment will be a partner to its antibiotic treatment Nuzyra.
After Sarepta reported the death of a patient who had recently taken the gene therapy Elevidys, patient advocacy group Parent Project Muscular Dystrophy stepped up—as they always do.