Rare diseases
The FDA turned away Ebvallo in January, taking issue with the design of the registrational trial. In a recent meeting, however, the agency agreed that the study could in fact support the cell therapy’s approval. The news comes a week after the departure of controversial biologics Director Vinay Prasad.
Catalyst Pharmaceuticals comes with a trio of approved drugs as Angelini Pharma expands its neuroscience offerings.
While AstraZeneca has discontinued work on four assets—including one in asthma and another in acromegaly—the pharma has also elected to take forward a bispecific antibody that destroys the EGFR protein.
Regeneron hauled in $3.6 billion during the first quarter of 2026, as analysts homed in on a slight Eylea HD miss and key upcoming readouts, including for LAG3 candidate fianlimab in metastatic melanoma.
Chiesi Group is taking KalVista Pharmaceuticals under its wing, paying $1.9 billion for the biotech’s oral therapy Ekterly to treat severe swelling episodes caused by the rare genetic disorder hereditary angioedema.
Staff at Salt Lake City-based techbio company Recursion recently heard from Jenny and Tim Jones about their challenging family history of familial adenomatous polyposis.
The FDA has renewed calls for Amgen’s Tavneos to be pulled from the market, saying it has discovered new evidence that study personnel doctored the results of the drug’s pivotal study in order to make it look effective.
With Phase 3 data in hand, Intellia Therapeutics is seeking approval for its in vivo CRISPR gene editing therapy for hereditary angioedema.
Approved Thursday via the FDA’s Commissioner’s National Priority Voucher program, Otarmeni is the first gene therapy for hearing loss—and the first treatment to target an underlying cause of the condition.
The pivotal study of zilganersen in Alexander disease missed a secondary endpoint, but analysts expect the FDA to approve the asset given the unmet need and overall data.
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