US

Justices question what Amgen’s invention covers in its dispute with Sanofi. They appear inclined to uphold Federal Circuit’s standard for enablement despite potential negative impact on genus claims.

Pink Sheet infographic show US regulatory forecast for the many new vaccines facing the twin tests of FDA approval and CDC recommendation, which are both needed to effectively access the US commercial marketplace.

Early parent involvement in drug development and clinical trial design could improve recruitment and outcomes, stakeholders argue.

Agency’s presentations on Biogen/Ionis’ ALS drug included differing viewpoints from the statistical and clinical pharmacology teams; the transparency and emphasis on a multidisciplinary review approach may reflect lessons learned from missteps at the Aduhelm panel review in 2020.

A November 2022 refuse-to-file letter cited clinical and statistical issues for the stem cell therapy, which failed its Phase III clinical efficacy endpoint. However, BrainStorm has consistently asserted that NurOwn demonstrated benefit in patients with less advanced disease at baseline, and it is encouraged by regulatory flexibility FDA recently has shown for the neurodegenerative disease.

Concern about disrupting the implementation of the negotiation program could presage a tough road for the legislation in the Democrat-controlled Senate.

Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered. 

Advisory committee to discuss proposed changes to isotretinoin REMS to minimize burden and treatment delays while preventing risk of fetal exposure. Agency recommends against at-home pregnancy tests.

Pink Sheet reporter and editors discuss Moderna CEO’s Stéphane Bancel’s defense of his company and COVID-19 vaccine pricing at the Senate Health, Education, Labor and Pensions Committee, the NIH denying a petition to use government march-in rights for Xtandi, and Tofersen receiving a positive US FDA advisory committee recommendation for use in ALS.

Moderna’s time in the hot seat of the US drug pricing debate went about as well as it could for the company. The firm astutely set a tone of an All-American success story – but the hearing also showcased other ‘only in America’ themes.

Draft guidance gives recommendations for conducting one randomized controlled trial to generate the evidence for accelerated approval and confirm clinical benefit.

The Biden administration decision not to pursue ‘march in’ proceedings for Pfizer/Astellas’ Xtandi sounds like good news. The reality is that the prostate cancer therapy is likely to face a more certain federal pricing intervention in the first year of the new Medicare ‘negotiation’ process.