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This week’s Capitol Hill meetings come on the heels of rejections of ultra-rare disease drugs developed by Biohaven and Saol Therapeutics. Physicians and patient groups implored the FDA to expedite these treatments.
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After a rocky 2025, Sarepta Therapeutics’ executives admit they have work to do to bring patients back into the fold as sales of Duchenne muscular dystrophy gene therapy Elevidys continue to decline.
Last week, the FDA made its one pivotal trial policy official, sparking myriad questions from industry leaders, including around specific evidence required for the single study and why it hasn’t been implemented across all therapeutic areas before now.
Alternatives to opioids are desperately needed to better treat moderate to severe acute pain, but to date, we’ve seen few novel analgesics hit the market.
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Eli Lilly’s win in a head-to-head trial drove Novo Nordisk’s market cap to pre-Wegovy levels not long after the victor became the first pharma company to top a $1 trillion valuation. It seems one company can do no right, while the other can do no wrong.
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Former president and CEO of CytoDyn Nader Pourhassan along with Kazem Kazempour, former CEO of the CRO running CytoDyn’s trials, are awaiting sentencing but could face up to 20 years in prison for each count of securities and wire fraud and insider trading.
The EPIC Act has been proposed with bipartisan and industry support to give small molecule drugs the same protection against price negotiation as biologics, but concerns over how to balance the federal budget could prevent a short-term fix to the IRA.
The discontinuation caps off a turbulent development path for izokibep, which in September 2023 produced disappointing results from Phase IIb/III study in hidradenitis suppurativa that was found to have had dosing errors.
Around 25 companies have gone public this year, most of them in the early months. Most have tumbled from their original offer price.
By far, the largest acquisition of 2024 was Novo Holdings’ yet-to-be-closed buyout of manufacturer Catalent at $16.5 billion. Outside of that, the leading pharmaceutical companies kept to less than $5 billion per deal.
Ending the diagnostic journey for patients, particularly those with rare diseases, improves patients’ quality of life while reducing costs to healthcare systems.
With nearly 90% of patients showing no detectable cancer cells after treatment, J&J and Legend’s Carvykti could stave off competition from emerging CAR T therapies such as Gilead and Arcellx’s anito-cel.
BNT327, now in early-phase trials, is part of a class of drugs that could one day challenge Keytruda’s dominance. BioNTech obtained the candidate when it bought Biotheus last month in an acquisition deal that could reach up to $950 million.
GSK, Gilead and Arcellx, Vertex and more present new data at the American Society of Hematology annual meeting just as sickle cell therapies Casgevy and Lyfgenia have a new outcomes-based payment model; Eli Lilly and Novo Nordisk pump new funds into manufacturing; and AbbVie makes a Cerevel comeback while uniQure clears a path toward accelerated approval in Huntington’s disease.
Carisma’s second workforce reduction this year likely leaves the company with 44 full-time employees as turns its focus to developing therapies for fibrosis, oncology and autoimmune diseases.