News
The FDA plans to hold an advisory committee meeting to discuss Capricor Therapeutics’ application for deramiocel, which the agency rejected last July. The news surprised CEO Linda Marbán, who told BioSpace the FDA has not communicated any issues of concern with the company’s resubmitted application.
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Dual and even triple or quadruple track processes have come roaring back in 2026 thanks to a glut of M&A that has refilled investors’ wallets. Big Pharma is being put on notice that time is critical if they want to acquire.
Policymaking at FDA has been anything but business as usual under the Trump administration, but former regulators cite the agency’s new investigational new drug pilot program as a sign of normalcy.
The FDA’s recently altered outlook on the evidence required for approval of rare disease drugs could have immediate benefits for companies including Skyhawk Therapeutics, Capricor Therapeutics and Biohaven.
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If cell and gene therapy makers are going to achieve their mission to improve patients’ lives, the industry must come together to share information across stakeholders, from regulators to manufacturers to payers.
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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.
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.
The deal is the latest in a series of Sanofi investments in its immunology portfolio. According to Sanfoi, DR-0201 can achieve deep B cell depletion, giving it the potential to reset the immune system.