Pink Sheet is part of Pharma Intelligence UK Limited

This site is operated by Pharma Intelligence UK Limited, a company registered in England and Wales with company number 13787459 whose registered office is 5 Howick Place, London SW1P 1WG. The Pharma Intelligence group is owned by Caerus Topco S.à r.l. and all copyright resides with the group.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By

UsernamePublicRestriction

US

Set Alert for US

Supreme Court Seems Ready To Uphold Ruling Against Amgen’s Anti-PCSK9 Antibody Patents

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.

Biologics Intellectual Property

Vaccine Decisions: US FDA And CDC Weigh New Vaccines For Diseases From Dengue To RSV

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.

US FDA Performance Tracker Vaccines

Neonatal Drug Development Could Benefit From Rare Disease Experience

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

Pediatrics Research & Development

At Tofersen Panel Review, US FDA Shined A Light On Internal Differences Of Opinion

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.

Advisory Committees Review Pathway

BrainStorm’s ALS Treatment NurOwn, Filed Over Protest, Will Get US FDA Panel Review

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.

Advisory Committees Rare Diseases

House QALY Bill Overreach Threatens Medicare Price Negotiation Program, Democrats Warn

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

Health Technology Assessment Medicare

Recent And Upcoming FDA Advisory Committee Meetings

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

Advisory Committees Drug Review

iPLEDGE REMS: FDA Panel To Consider Changing Pregnancy Testing, Counseling Requirements

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.

Advisory Committees Risk Management

Pink Sheet Podcast: Moderna CEO On Capitol Hill, Xtandi March-In Rights, Tofersen Adcomm In ALS

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.

Pricing Debate Intellectual Property

Moderna And The All-American Drug Pricing Hearing

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.

Vaccines Pricing Debate

US FDA Formalizes ‘One-Trial’ Approach For Oncology Accelerated Approval

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

Cancer Research & Development

Xtandi Still Faces Likely US Price Cut Even As It Avoids ‘March In’ Proceeding

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.

Reimbursement Medicare
See All
UsernamePublicRestriction

Register