FDA
The FDA in October 2025 granted bitopertin its Commissioner’s National Priority Voucher but after a shortened review time has decided the data did not support regulatory approval for treating erythropoietic protoporphyria.
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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The mesothelioma approval for the Keytruda combination regimen potentially unlocks a $12 billion market opportunity, according to a recent report from research firm IMARC Group.
The FDA’s approval of Kisqali in combination with an aromatase inhibitor allows Novartis to target patients with earlier breast cancer who are at risk of recurrence.
The newly approved filling line will be able to provide both 50-mg and 100-mg doses of the respiratory syncytial virus antibody Beyfortus to help meet demand ahead of the 2024/2025 RSV season.
Friday’s approval comes after a previous rejection in October 2023 due to manufacturing concerns.
The FDA has six target action dates ahead to round out September as drugs for gastroparesis, Niemann-Pick disease type C and more await decisions.
Tecentriq Hybreza, which combines Roche’s Tecentriq with Halozyme Therapeutics’ Enhanze drug delivery technology, is being touted as the first and only subcutaneous anti-PD-(L)1 cancer immunotherapy.
One patient developed elevated liver blood tests after receiving Astellas Pharma’s non-hormonal small molecule blocker, leading the regulator to add a warning to the drug’s label.
Tremfya’s ulcerative colitis approval on Wednesday comes as Johnson & Johnson’s blockbuster immunotherapy Stelara continues to face growing competition from biosimilars.
With a Friday advisory committee meeting looming, the sole indication for Intercept Pharmaceuticals’ Ocaliva appears to be at risk as the regulator flags issues regarding its post-marketing results.
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