FDA
The FDA’s refusal to review Moderna’s mRNA-based flu vaccine is part of a larger communications crisis unfolding at the agency over the past nine months that has also ensnarled Sarepta, Capricor, uniQure and many more.
FEATURED STORIES
The Senate failed to pass a massive spending bill on Thursday—which includes the rare pediatric PRV program but also funding for the Immigration and Customs Enforcement’s large-scale crackdown in Minnesota and other states.
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.
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Continuing our SCOPE 2025 coverage, Rohit Nambisan, CEO at Lokavant addresses not only current challenges, but the life sciences industry’s responsibility to maintain scientific integrity.
Evrysdi is the first, and so far only, noninvasive disease-modifying treatment for spinal muscular atrophy.
Leaked data showed that Pfizer’s mevrometostat has strong therapeutic potential in metastatic castration-resistant prostate cancer, while recent readouts also position the pharma as a strong contender in colorectal cancer and bladder cancer.
The approval comes days after Germany’s Merck KGaA confirmed it was in advanced talks to acquire SpringWorks.
Compounding pharmacies aren’t the only makers of off-brand versions of Novo Nordisk’s Wegovy and Eli Lilly’s Zepbound. The situation is causing the FDA regulatory headaches and, more seriously, posing potential risks to the public.
The intravenous antibiotic Emblaveo, a combination of aztreonam and avibactam approved in Europe last year, was developed in partnership with Pfizer. AbbVie acquired its share of the asset as part of its $63 billion buyout of Allergan.
Before garnering approval on Tuesday, Onapgo had been rejected twice by the FDA.
The rare disease space is awaiting two FDA verdicts in February, one for a genetic disease and another for a non-malignant tumor.
Robert F. Kennedy, Jr.—Trump’s pick for HHS secretary who endured confirmation hearings last week—has repeatedly criticized industry ties to the FDA, particularly financial links between the two, which could indicate trouble for the user-fee model.
The approval of Axsome Therapeutics’ Symbravo for migraine with or without aura came alongside the greenlight for Vertex’s non-opioid treatment Journavx.