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Under the temporary reign of top food executive Kyle Diamantas, the FDA will sustain programs initiated by former Commissioner Marty Makary, including the Commissioner’s National Priority Voucher initiative.
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Partners Summit Therapeutics and Akeso are expected to steal the show at the American Society of Clinical Oncology’s annual conference with data from their potential Keytruda rival, alongside Revolution Medicine’s groundbreaking pancreatic cancer candidate and other assets that could reshape patient care.
The tragic tale of TIGIT is well known. However, RIPK1, myc, STING and alpha-synuclein have also left a trail of failed clinical trials, canceled partnerships and sunk investments in their wake.
Analysts homed in on Duchenne muscular dystrophy and myotonic dystrophy type 1 assets during first quarter earnings as major players like REGENXBIO and Novartis as well as Dyne, Wave, Solid and Sarepta near the regulatory finish line.
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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.
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The Massachusetts-based startup’s goal is to develop RNA medicines that can treat diseases anywhere in the body using an “all-in-one” platform.
The Swiss pharma has canceled a second mid-stage trial for its investigational schizophrenia drug ralmitaront, as the competition shows promise in the space.
With the FDA’s approval on Monday, Ayvakit is the first and only treatment for adults with indolent systemic mastocytosis, a rare hematologic disorder, according to Blueprint Medicines.
Indivior’s Opvee is an emergency nasal spray medication to reverse opioid overdose approved for patients aged 12 years and above with signs of respiratory or central nervous system depression.
Ex-FDA Neuroscience Director Billy Dunn was appointed to Prothena’s board of directors last week. Industry representatives and regulatory experts weigh in on the potential ethical implications.
Data from a Phase III trial of apraglutide for a type of short bowel syndrome with intestinal failure is expected by the end of the year. It is also in a Phase II study for acute graft-versus-host disease.
The 61-patient study met the primary endpoint of improved physiologic liver function and secondary endpoints, which included established non-alcoholic steatohepatitis biomarkers, Hepion reported Monday.
Sanofi and Regeneron reported positive results for the anti-inflammatory drug in a chronic obstructive pulmonary disease trial. The companies hope to see approval before a 2024 read-out of a second trial.
The topical treatment Vyjuvek got the FDA’s greenlight, making it the first redosable gene therapy and the first therapeutic for the rare skin disease dystrophic epidermolysis bullosa.
With Friday’s approval, Epkinly edges out Roche’s bi-specific antibody glofitamab, which is also being proposed for diffuse large B-cell lymphoma.