Regulatory

FDA has granted accelerated approval to Avlayah, the first therapy engineered to cross the blood-brain barrier and treat the neurological manifestations of Hunter syndrome.

While the space has traditionally been dominated by smaller biotechs, that may be changing.

This is the agency's first approved treatment for acquired hypothalamic obesity.

FDA has approved Wegovy HD, a higher-dose 7.2 mg injectable semaglutide, based on Phase III data showing a mean weight loss of 20.7% in adults with obesity.

In the final part of his conversation with Pharmaceutical Executive, Aravo CCO Dave Rusher discusses the various regulatory updates around the world in the pharma and AI space.

FDA has approved Icotyde as the first oral targeted therapy for moderate-to-severe plaque psoriasis, offering adults and adolescents a once-daily pill that blocks the IL-23 receptor.

The ruling places a hold on any decisions made by the council, although the government does plan to appeal the ruling.

Arexvy is now approved for adults aged 18 to 49 with underlying conditions that elevate their risk of severe RSV disease.

FDA cites systemic failures in how the company tracked and reported adverse drug events across several of its medicines, including semaglutide.

Europe’s Joint Clinical Assessment promises harmonization, but it introduces a new layer of strategic complexity that manufacturers must navigate at both EU and national levels.

Ron Wyden led 7 Democrats demanding details from Pfizer, AstraZeneca, Novo Nordisk, and Eli Lilly.

FDA approved GSK's Wellcovorin as the first treatment for cerebral folate deficiency caused by a rare genetic variant.

The application is based on Phase III trial data showing the drug reduced the risk of invasive disease recurrence or death.

Ron Lanton, Senior Partner & Global Strategist, Lanton, Lanton & Sosa Law PLLC, discusses FDA’s removal of the two-trial requirement marking a significant policy shift and prompting sponsors to consolidate into a single robust pivotal study.

The vaccine chief briefly left the role last summer before almost immediately returning to it.

Ron Lanton, Senior Partner & Global Strategist, Lanton, Lanton & Sosa Law PLLC, notes shifting regulatory signals for Moderna’s mRNA flu vaccine review and raises broader questions about the stability of the approval pathway.

FDA informed uniQure it will not accept external control data as the primary basis for approving AMT-130, instead demanding a prospective, randomized, sham surgery-controlled trial before moving forward.

In the second part of his conversation with Pharmaceutical Executive, Ajna Biosciences CEO Joel Stanley explains the complexities of getting a botanical drug approved.

Alice Valder Curran discusses how the industry continues to strategize around this complicated order.

Ajna Biosciences CEO Joel Stanley discusses the differences between botanical drugs and botanically-derived drugs.

Defining the “active ingredient” is the central challenge of biologic PTE, because biologics are large, complex molecules that do not fit neatly into a statutory framework built for small molecule drugs.

FDA approves a supplemental BLA for BioMarin Pharmaceutical Inc.’s Palynziq, expanding its use to adolescents 12 and older with PKU.

FDA expands approval of Novo Nordisk’s once-weekly Sogroya to include three new pediatric growth disorder indications, offering children as young as 2.5 an alternative to daily growth hormone injections backed by Phase III non-inferiority data.

FDA grants accelerated approval to Boehringer Ingelheim’s Hernexeos (zongertinib) for treatment-naïve HER2-mutant advanced NSCLC.

The approval makes Dupixent the first FDA-approved therapy for allergic fungal rhinosinusitis, supported by Phase III data showing significant improvements in sinus opacification, nasal symptoms, and reductions in surgery and systemic corticosteroid use.