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.
FEATURED STORIES
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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The monthly maintenance regimen, which offers a more convenient frequency than the initially approved treatment schedule for patients with Alzheimer’s, could help with Leqembi’s thus far disappointing uptake and sales.
From March 2020 through September 2022, Biohaven wielded meals at high-end restaurants and paid speaking opportunities to induce healthcare providers to prescribe its migraine therapy Nurtec ODT, according to the Department of Justice.
Bristol Myers Squibb’s Opdivo plus Yervoy, as well as Pfizer’s Braftovi, have each shown strong Phase III performances that could position them as new standards of care in certain subtypes of metastatic colorectal cancer.
Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and more aim to deliver the next wave of progress with near-term data and regulatory milestones.
Achondroplasia, which affects around one in 20,000 babies, has just one approved treatment: BioMarin’s Voxzogo. However, new investigational treatments are vying to compete in the area.
The Japanese pharma had one asset rejected by the FDA and withdrew a regulatory application for another, but already this month the company has secured an approval for AstraZeneca-partnered Dato-DXd, to be marketed as Datroway.
While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several companies including Wave, Dyne and Avidity are looking to answer the call with investigational therapies targeting greater efficacy and broader reach.
Less than a day into his second term, President Donald Trump ordered a freeze on communications at major public health agencies, among other moves that have sent waves through the biopharma industry.
The readout comes on the heels of CagriSema’s disappointing Phase III performance, where it missed Novo’s projection of 25% weight reduction.
The unsuccessful Phase III results are the latest to suggest that the blockbuster cancer drug is finally bumping up against its limits after racking up around 50 approvals since getting its first FDA nod in September 2014.