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FDA Commissioner Marty Makary called these changes “common-sense reforms” that could expedite the development of cell and gene therapies.
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After years stuck in the “doldrums,” the biopharma sector is in a “very good place” heading into the new year, analysts told BioSpace, with both rare and chronic diseases headlining investor and R&D interest as JPM26 kicks off.
Pharmas will need to provide their latest stance on the Most Favored Nation drug pricing plans, while biotech finally gets a break after a few tough years.
From biotech veterans to embattled modalities to a new wave of RNAi therapeutics, BioSpace’s NextGen Class of 2026 emerged during a tough fundraising environment in 2025. Check out the 15 battle-tested companies that caught our eye.
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With five CDER leaders in one year and regulatory proposals coming “by fiat,” the FDA is only making it more difficult to bring therapies to patients.
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Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a non-profit sponsor to win FDA approval.
The FDA has informed Merck, Sanofi and AstraZeneca of the safety investigation into their RSV antibodies, though it remains unclear what regulatory action, if any, will be taken.
The Commissioner’s National Priority Voucher program was launched in June to shorten review times for companies that align with various national priorities, such as improving domestic drug production and supply.
Pfizer deals again in obesity space as Wave and Structure drop splashy weight loss results; what CDER Director Richard Pazdur’s sudden retirement means for biopharma; neuro diseases take center stage at CTAD; and more.
With $6 billion left in firepower, Pfizer is planning transactions in the hundreds of millions to the low-billions range, particularly in internal medicine and immunology and inflammation, Guggenheim reported.
Long a quieter, locally focused industry, Japanese pharma giants are increasingly looking to the rest of the world for deals.
After a series of unfortunate regulatory rejections and manufacturing issues surfaced, Regeneron’s shares dipped to $483 this summer—the lowest they’d been since early 2021. But they now sit higher than they did at the start of the year.
Saol Therapeutics received a complete response letter for its pyruvate dehydrogenase complex deficiency treatment a week after the FDA unveiled its Rare Disease Evidence Principles program. On Dec. 18, in a Type A meeting, the biotech will attempt to convince the agency that its drug fits perfectly into the framework.
TERN-701 more than doubled the response rate of Novartis’ rival approved therapy in an early-stage trial, sending the biotech’s shares flying.
Pfizer apparently had more in the tank after the high-profile battle to acquire Metsera earlier this fall. The company has licensed a new GLP-1 from YaoPharma.