Editors from the Pink Sheet and Medtech Insight discuss the retirement of Jeff Shuren, PBM executives again appear unscathed after another Capitol Hill appearance, and the growing push to ensure the FDA’s rare pediatric disease priority review voucher program is renewed.

But the agency should explain why different meeting types are granted than requested or denied entirely, industry representatives said during session on meeting management best practices.

The EMA says the benefits of treatment with Leqembi are not large enough to outweigh its risks. Meanwhile, Alzheimer Europe is hoping that real-world findings from the US-mandated patient registry or from ongoing trials will help persuade the EU regulator to reconsider its position.

Two orphan drugs are among a host of products that the European Medicines Agency’s human medicines committee has OKd for pan-EU approval.

The Alliance for Safe Biologic Medicines urged restraint when considering FDA findings suggesting switching studies may not be as vital to support interchangeability.

Relyvrio/Albrioza, Amylyx’s ill-fated treatment for amyotrophic lateral sclerosis, was approved for marketing by the US Food and Drug Administration but rejected by the European Medicines Agency. The Pink Sheet explores how the two agencies applied the available regulatory flexibilities and the impact of other factors, such as regulatory precedence and patient influence, on decision-making.

Eisai/Biogen could this week learn whether the European Medicines Agency will give the thumbs up to market lecanemab across the EU for early Alzheimer’s disease.

The US FDA surprised some observers when it advised Agenus against filing for accelerated approval of the CTLA-4/PD-1 combination, but the agency has been cracking down on confirmatory trial requirements recently to better ensure the studies will be finished.

Many platform designation requests have been from sponsors eager to cite other sponsors’ products, but CBER Director Peter Marks said in an interview with the Pink Sheet his office likely is years away from accepting those applications.

Pink Sheet reporter and editors discuss the impact of a US and EU drug approvals analysis that found the US FDA still clears many novel products first, including most new cell and gene therapies and cancer treatments.

A prominent rare disease researcher has praised the UK’s Department for Health and Social Care for its transparency and willingness to work with experts, but said that regulatory systems needed to speed up to facilitate patient access to innovative treatments.

Zanidatamab, Jazz and BeiGene’s investigational treatment for biliary tract cancer, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

The Oncologic Drugs Advisory Committee urged the FDA not to delay Imfiniz’s perioperative NSCLC indication, while also unanimously pushing for better trial designs in lung cancer and all resectable tumors.

The FDA’s experience with product-specific guidances for generic drugs may impact biosimilar industry decision-making, but the program has improved in recent years.

Relyvrio/Albrioza, Amylyx’s ill-fated treatment for amyotrophic lateral sclerosis, was approved for marketing by the US Food and Drug Administration but rejected by the European Medicines Agency. The Pink Sheet explores how the two agencies applied the available regulatory flexibilities and the impact of other factors, such as regulatory precedence and patient influence, on decision-making.

The Oncologic Drugs Advisory Committee will vote on a product agnostic question related to perioperative trial designs during a 25 July meeting after discussing whether AstraZeneca needs an additional trial for its Imfinzi perioperative regimen in non-small cell lung cancer.

Senior leadership in the FDA’s drugs, biologics and devices centers want reviewers to feel more comfortable taking risks in product approvals, but may need more concrete examples of regulatory flexibility's success to convince them.

CDER real world evidence policy head John Concato said the FDA could approve a new labeled use based only on real-world evidence, but made clear the approach is challenging. 

Novel agents in immuno-oncology headline the drug center’s upcoming goal dates, while gene therapies make up most of the biologic center’s workload.

So far this year, when the agency has taken action on NMEs that went before advisory committees, the FDA decision has matched the committee vote.

Half of the FDA’s upcoming 2024 user fee goals target rare diseases, but highly prevalent conditions like uncomplicated urinary tract infections and atopic dermatitis are also up for agency action in the second half of the year.

Firms can be more forceful in disputes with FDA now that Supreme Court has eliminated Chevron deference. Questions around whether an evidentiary standard has been met may be ripe for challenge, legal experts said, but they also caution that sponsors will face more uncertainty.

The European Medicines Agency says that in 2022, the average time taken up by clock-stops exceeded the average time of drug assessment.

Clene leadership spoke with the Pink Sheet about its push for accelerated approval for its ALS nanoparticle treatment. A new nudge from Congress asking the US FDA to consider survival data in accelerated approval could help.