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After a patient safety signal and then death, the FDA in October 2025 placed holds on two of the company’s CRISPR programs for hereditary transthyretin amyloidosis.
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Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.
Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging field.
From opening new therapeutic mechanisms to repairing neuronal damage, investigational molecules from Ventyx Therapeutics, AC Immune, Gain Therapeutics and more could shape the future of Parkinson’s disease treatment.
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The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
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The Chinese biopharma company will be closing its U.S. headquarters and wet lab in the D.C. suburbs, according to scientists who recently lost their jobs.
With Monday’s FDA rejection of their long-acting glatiramer acetate formulation, Viatris and Mapi Pharma continue the biopharma industry’s recent losing streak in multiple sclerosis.
Early Phase IIb data from 66 patients showed Novartis’ BTK inhibitor remibrutinib has potential outside chronic spontaneous urticaria, leading to symptomatic relief in patients with hidradenitis suppurativa.
Fresh off its $259 million Series C funding round, Alumis unveiled mid-stage data Saturday for its TYK2 inhibitor ESK-001 demonstrating strong symptomatic improvement in moderate-to-severe plaque psoriasis.
Johnson & Johnson’s Protagonist-partnered oral psoriasis candidate was able to sustain its therapeutic benefit through one year, according to data presented on Saturday at the American Academy of Dermatology annual meeting.
Novo Nordisk’s blockbuster weight-loss drug Wegovy was approved on Friday by the FDA to reduce the risk of cardiovascular death, heart attack and stroke in adults who have cardiovascular disease and are obese or overweight.
In this deep dive, BioSpace examines what’s next for Leqembi, the true cost of anti-amyloid antibodies, and what other Alzheimer’s treatments are coming down the pipeline.
The approval, which Bristol Myers Squibb reported on Thursday, positions the company to compete with Astellas and Pfizer’s Padcev.
According to Fresenius Kabi, Tyenne is the first biosimilar to Genentech’s Acterma which has both IV and subcutaneous formulations approved by the FDA.
The company announced Thursday it terminated its BTK inhibitor evobrutinib for multiple sclerosis, which did not meet the primary endpoints in two late-stage trials.